[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"als-amyotrophic-lateral-sclerosis\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:als-amyotrophic-lateral-sclerosis":29},{"pageToken":4,"total":5,"offset":6,"count":7,"results":8},null,30,0,25,[9,45,80,106,128,152,178,222,250,279,317,351,373,397,420,448,480,504,523,550,577,607,631,653,673],{"id":10,"slug":11,"hasResults":12,"nctId":13,"briefTitle":14,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":26,"conditions":27,"keywords":30,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":33,"lastUpdatePostDateStruct":34,"startDateStruct":37,"completionDateStruct":39,"leadSponsor":41,"locationsCount":44},"100054293","evaluation-of-the-feasibility-and-efficacy-of-a-chronic-brain-computer-interface-for-speech-rehabilitation-in-patients-with-locked-in-syndrome-lis-100054293",false,"NCT07698496","Evaluation of the Feasibility and Efficacy of a Chronic Brain-computer Interface for Speech Rehabilitation in Patients With Locked-in-syndrome (LIS)","SpeechBCI","Inclusion Criteria:\n\na. Male or female between 18 and 65 years old b. French speaking person c. Person in locked in syndrome with a severe speech disorder following either: i. A subcortical stroke ii. Amyotrophic Lateral Sclerosis. d. Person with stable clinical state (esp. regarding respiratory state) e. Person able to read on a screen (sufficient eye movement control) f. Negative plasma pregnancy test for women of childbearing potential\\* g. Highly effective or at least acceptable\\*\\* contraception for women of childbearing potential.\n\nh. Persistence of localizing signals of language function proven by functional MRI.\n\ni. Person able to perform a simple BCI task with MEG j. Person with a neuropsychological profile evaluated by psychiatrist as compatible with sustain BCI training k. Informed consent to participate in the obtained using their usual means and code of communication (residual vocalizations, eye-blinking code, pictograms, eye tracking, alphabet chart, etc.), in the presence of their trusted person or curator who is accustomed to communicating with them using this means.\n\nl. Person affiliated to the French social security system or beneficiary of such a system\n\nExclusion Criteria:\n\nPatients with any of the following criteria cannot be included in this investigation:\n\n1. Severe cognitive disorders assessed after a neuropsychological evaluation\n2. Deafness\n3. Continuous assisted ventilation\n4. Contraindication to intracranial surgery\n5. Contra-indication to MRI (1.5T), CT-scan or their related contrast agent injection or MEG examinations.\n6. Anatomical brain MRI showing structural abnormalities in the cortical areas of language\n7. Functional impairment of language cortical areas on MRI\n8. Auditory evoked potential showing hearing impairment\n9. Person with a skull shape incompatible with one WIMAGINE implant on each hemisphere\n10. Persons referred to in Articles L1125-7 of the Public Health Code and article 64 of MDR(corresponding to all protected persons: pregnant women, women in labor, breastfeeding mothers, minors, persons deprived of liberty by judicial or administrative decision.","ALL","18 Years","65 Years",{"count":21,"type":22},3,"ESTIMATED","INTERVENTIONAL",[25],"NA","Eighteen million people worldwide are affected by speech disorders. Locked-in syndrome (LIS) represents the most extreme form of communication disability resulting from motor impairment. In France, the Association du Locked-In Syndrome (ALIS) reports approximately 500 individuals with LIS, most of whom live at home. The quality of life of people with LIS depends strongly on their ability to communicate, and speech synthesis is the mode of communication restoration most desired by individuals with LIS. Several surveys have shown that improving communication abilities in these individuals leads to a significant improvement in their quality of life as well as that of their caregivers.\n\nNatural speech allows the production of an average of 150 words per minute. Non-invasive communication methods, whether based on residual motor function (eye-blink code) or on brain-machine or brain-computer interfaces (Brain-Computer Interface, BCI) using scalp electroencephalographic (EEG) signals, involve a high cognitive load and have low efficiency (spelling only a few letters per minute). Invasive BCIs for speech rehabilitation aim to overcome the limitations of non-invasive devices (cognitive overload and slow speech rate). The intention to act (the act of speaking) is predicted by an algorithm based on the direct decoding of neuronal activity from the sensorimotor cortex (the area where articulatory muscles are represented).\n\nTo date, studies testing speech rehabilitation BCIs in humans remain rare. A subdural electrocorticographic (ECoG) invasive BCI enabled speech decoding (words and sentences from a limited repertoire) for chronic use (2 years). Real-time control of an on-screen cursor allowing spelling of up to 90 letters per minute (equivalent to text messaging) has also been achieved using an intracortical invasive BCI (Utah Array). Very recently, up to 60 words per minute were produced using an intracortical invasive BCI (Utah Array) implanted in the ventral premotor cortex, although with a connector potentially contaminated by acoustic audio feedback. Furthermore, these devices still rely on transcutaneous connectors, which may be sources of infection and prevent routine daily-life use.\n\nIn summary, there is currently no fully implantable, wireless invasive \"speech BCI\" with real-time speech synthesis suitable for long-term home use. The present study will use an intracranial extradural invasive BCI combined with a speech synthesizer, with the aim of developing a communication tool suitable for everyday use. More specifically, the SpeechBCI protocol will propose two complementary BCI approaches in the same subject: a speech BCI (primary objective, BCI\\_PAROLE device) and a cursor BCI (secondary objective). Both BCIs will use the WIMAGINE intracranial epidural system, enabling ECoG signal acquisition with a very limited risk of infection and brain injury and providing signals that are more stable over time compared with intracortical or subdural ECoG devices that retain transcutaneous connectors. These systems will allow long-term and ecological use, as the WIMAGINE implant is wireless and offers excellent long-term signal stability. The WIMAGINE implant has already been successfully tested for controlling an exoskeleton in a tetraplegic subject (operational for over 6 years) and very recently for controlling walking in real-life conditions via a spinal cord stimulator in a paraplegic individual.\n\nThe BCI\\_PAROLE device will integrate a speech synthesizer providing real-time auditory feedback to the speaker. The BCI-CURSEUR device will allow the subject to control an on-screen cursor to access various communication functionalities (web access, emails, chats, etc.). This will provide a complementary communication solution to real-time speech production.\n\nThe hypothesis of this stydy is that the intention to speak (attempted speech) will be decoded by the BCI\\_PAROLE device in individuals with LIS because, as with limb paralysis, paralysis of articulatory and phonatory muscles does not prevent the corresponding cortical map from producing specific signals. Similarly, the BCI-CURSEUR device will decode the intention to move a cursor and perform actions on a computer screen.\n\nNeuronal electrical signals will be recorded bilaterally from the ventral motor cortex (representation of lips, cheeks, tongue, palate, and larynx-the vocal tract) and from the dorsal part of the motor cortex (larynx and hand, the latter for controlling a two-degree-of-freedom cursor), using a total of 128 electrodes (64 on each cerebral hemisphere). The implant will be optimally positioned over speech motor areas using preoperative functional imaging in order to optimize speech decoding by the BCI\\_PAROLE device.",[28,29],"Locked in Syndrome","ALS (Amyotrophic Lateral Sclerosis)",[31],"Locked in syndrom, Speech disorders, Brain-computer interface, BCI, speech rehabilitation","NOT_YET_RECRUITING","2026-07-07",{"date":35,"type":36},"2026-07-13","ACTUAL",{"date":38,"type":22},"2026-10-10",{"date":40,"type":22},"2031-09-30",{"name":42,"class":43},"University Hospital, Grenoble","OTHER",4,{"id":46,"slug":47,"hasResults":12,"nctId":48,"briefTitle":49,"officialTitle":50,"acronym":51,"eligibilityCriteria":52,"healthyVolunteers":53,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":54,"targetDuration":4,"studyType":23,"phases":56,"briefSummary":57,"conditions":58,"keywords":61,"overallStatus":70,"whyStopped":4,"lastUpdateSubmitDate":71,"lastUpdatePostDateStruct":72,"startDateStruct":74,"completionDateStruct":76,"leadSponsor":78,"locationsCount":21},"100520020","effects-of-probiotics-in-amyotrophic-lateral-sclerosis-frontotemporal-dementia-spectrum-disorder-als-ftdsd-patients-100520020","NCT06051123","Effects of Probiotics in Amyotrophic Lateral Sclerosis-Frontotemporal Dementia Spectrum Disorder (ALS-FTDSD) Patients","Effects of Probiotics on Lipidomic Profile and Disease Evolution in ALS-FTDSD Patients: A Randomized Multicenter, Double-blind, Phase II, Placebo-controlled, Parallel Trial.","PROBIOTIC","Inclusion criteria:\n\nParticipants must meet all of the following inclusion criteria to be eligible for enrolment into the study:ALS-FTDSD participants\n\n1. Aged 18 years old or greater.\n2. Diagnosis of ALS by El Escorial Criteria revised (possible, probable, probable with lab support and definite).\n3. Onset of weakness or speech impairment no more than 24 months before randomization.\n4. ALSFRS-R equal or superior to 24\u002F48 at screening, with no more than one subscore under 2\u002F4.\n5. SVC greater than or equal to 60% predicted for sex, age and height at screening.\n6. Note on FTD Symptoms: The presence of FTD symptoms is not a requirement for inclusion in this study. Participants with a diagnosis of ALS, whether or not accompanied by FTD symptoms, are eligible for inclusion. No prior or screening diagnosis of FTDSD is required.\n7. Subject has an informant\u002Fcaregiver who has frequent and sufficient contact to provide accurate information about the patient's cognitive abilities and behaviors to complete the ALS-CBS.\n8. Participants apt to comprehend and sign the ICF.\n9. Participants of child-bearing potential must have a negative serum pregnancy test at screening and agree to use a medically approved method of birth control for the duration of the study. All hormonal birth control must have been in use for a minimum of three months. Acceptable methods of birth control include:\n\n   * Abstinence or agrees to use contraception if planning to become sexually active.\n   * Hormonal contraceptives including oral contraceptives, hormone birth control patch, vaginal contraceptive ring, injectable contraceptives, or hormone implant\n   * Double-barrier method\n   * Intrauterine devices\n   * Non-heterosexual lifestyle or agrees to use contraception if planning on changing to heterosexual partner(s)\n   * Vasectomy of partner at least 6 months prior to screening\n10. Willing to maintain eating habits throughout the study.\n11. Willing to refrain from consuming probiotic supplements and food containing added probiotics and\u002For prebiotics (e.g., yogurts with live, active cultures or supplements) from the moment of screening until the end of the study.\n\nHealthy controls:\n\n1. Aged 18 years old or greater.\n2. Able to comprehend and willing to sign ICF.\n3. Participants of child-bearing potential must have a negative serum pregnancy test at screening and agree to use a medically approved method of birth control for the duration of the study. All hormonal birth control must have been in use for a minimum of three months. Acceptable methods of birth control include:\n\n   * Abstinence or agrees to use contraception if planning to become sexually active.\n   * Hormonal contraceptives including oral contraceptives, hormone birth control patch, vaginal contraceptive ring, injectable contraceptives, or hormone implant\n   * Double-barrier method\n   * Intrauterine devices\n   * Non-heterosexual lifestyle or agrees to use contraception if planning on changing to heterosexual partner(s)\n   * Vasectomy of partner at least 6 months prior to screening\n4. Willing to maintain eating habits throughout the study.\n5. Willing to refrain from consuming probiotic supplements and food containing added probiotics and\u002For prebiotics (e.g., yogurts with live, active cultures or supplements) from the moment of screening until the end of the study.\n\nInformant\u002Fcaregiver\n\n1. Aged 18 years old or greater.\n2. Has frequent and sufficient contact to provide accurate information about the patient's cognitive abilities and behaviors to complete the ALS-CBS.\n3. Able to comprehend and willing to sign ICF\n\nExclusion criteria:\n\nSubjects with any of the following characteristics\u002Fconditions will not be included in the study:\n\nALS-FTDSD participants\n\n1. Use of respiratory support (non-invasive ventilation or mechanical respiratory support) at screening.\n2. Significant medical condition or behavioral issues that could interfere with participation in the clinical trial in the principal investigator's opinion.\n3. Use of a feeding tube at randomization.\n4. Use of lipid-lowering drugs for less than 3 months before randomization.\n5. Introduction of lipid-lowering drug unless it is due to the event of acute coronary syndrome or stroke as per Canadian guidelines.\n6. Use of edaravone with stable dosage for less than 2 months before randomization.\n7. Use of riluzole with stable dosage for less than 1 month before randomization.\n8. Introduction of edaravone or riluzole during the clinical trial.\n9. Immunodeficiency (immune-compromised and immune-suppressed participants, e.g., AIDS, lymphoma, participants undergoing long-term corticosteroid treatment, chemotherapy and allograft participants).\n10. Pregnancy (as per serum HCG pregnancy test at screening), planning to be pregnant or currently breastfeeding.\n11. Use of probiotics other than the study medication in the month prior to randomization. Note: participants could be eligible to participate after a 4-week washout period.\n12. Use of any antibiotic drug in the month prior to randomization. Note: participants could be eligible to participate after a 4-week washout period.\n13. Milk and soy allergy, or severe lactose intolerance.\n14. Currently enrolled in another clinical trial.\n\nHealthy controls:\n\n1. Use of lipid-lowering drugs for less than 3 months before randomization.\n2. Introduction of lipid-lowering drug unless it is due to the event of acute coronary syndrome or stroke as per Canadian guidelines.\n3. Pregnancy (as per serum HCG pregnancy test at screening), planning to be pregnant or currently breastfeeding. Note: participants will not take the IP or placebo, but pregnancy is a major factor that could affect lipidomic profiling.\n4. Use of probiotics in the month prior to day 0 of the study (visit 2). Note: participants could be eligible to participate after a 4-week washout period. Although participants will not consume any study product, we aim to maintain environmental factors comparable to the ALS-FTDSD group.\n5. Use of any antibiotic drug in the month prior to day 0 of the study (visit 2). Note: participants could be eligible to participate after a 4-week washout period. Although participants will not consume any study product, we aim to maintain environmental factors comparable to the ALS-FTDSD group.\n6. Milk and soy allergy, or severe lactose intolerance. Note: Although participants will not consume any study product, we aim to maintain environmental factors comparable to the ALS-FTDSD group. We aim to exclude allergies, so participants are maintained on standard diet.\n7. Currently enrolled in another clinical trial.",true,{"count":55,"type":22},150,[25],"The aim of this study is to assess the impact of a probiotic formulation on participants with ALS-FTDSD. It is hypothesized that participants given the probiotics will have different lipid profiles compared to participants receiving the placebo at different time points.",[59,29,60],"ALSFTD","Frontal Temporal Dementia (FTD)",[62,63,64,65,66,67,68,69],"ALS","FTD","motor function","probiotics","lipidomics","metabolites","neurofilament","microbiome","RECRUITING","2026-06-26",{"date":73,"type":36},"2026-06-30",{"date":75,"type":36},"2024-01-01",{"date":77,"type":22},"2027-02",{"name":79,"class":43},"Centre hospitalier de l'Université de Montréal (CHUM)",{"id":81,"slug":82,"hasResults":12,"nctId":83,"briefTitle":84,"officialTitle":85,"acronym":4,"eligibilityCriteria":86,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":87,"targetDuration":4,"studyType":23,"phases":88,"briefSummary":90,"conditions":91,"keywords":92,"overallStatus":70,"whyStopped":4,"lastUpdateSubmitDate":97,"lastUpdatePostDateStruct":98,"startDateStruct":100,"completionDateStruct":102,"leadSponsor":104,"locationsCount":21},"100615556","phase-2-tofersen-in-non-sod1-als-100615556","NCT07294144","Tofersen in Non-SOD1 ALS","A Study to Evaluate the Biological Effect of Tofersen in Adults With Amyotrophic Lateral Sclerosis Without Mutations in SOD1","Inclusion Criteria:\n\n* Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use PHI in accordance with national and local participant privacy regulations.\n* Aged 18 years or older at the time of informed consent.\n* Confirmed diagnosis of ALS.\n* Time since onset of weakness due to ALS ≤ 24 months at the time of the screening visit.\n* Prior confirmed genetic testing negative for SOD1 and FUS mutations. Participants with mutations in genes other than SOD1 and FUS may be enrolled at the discretion of the Site Investigator.\n* SVC ≥ 50% of predicted value as adjusted for sex, age, and height (from the sitting position).\n* Medically able to undergo the study procedures and to adhere to the visit schedule at the time of study entry, as determined by the Investigator.\n* All participants must agree to practice effective contraception during the study and be willing and able to continue contraception for 5 months after their last dose of study treatment.\n* If taking riluzole, participant must be on a stable dose for ≥ 30 days prior to Day 1 and expected to remain at that dose until the final study visit.\n* If taking edaravone, participant must have initiated edaravone ≥ 60 days (2 treatment cycles) prior to Day 1 and expected to remain at that dose until the final study visit, unless the Investigator determines that edaravone should be discontinued for medical reasons, in which case it may not be restarted during the study.\n\nExclusion Criteria:\n\n* Treatment with another investigational drug (including investigational drugs for ALS through compassionate use or expanded access programs), biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer. Specifically, no prior treatment with small interfering RNA, stem cell therapy, or gene therapy is allowed.\n* Current enrollment in any other interventional study.\n* History of drug abuse or alcoholism within ≤ 6 months of study enrollment that would limit participation in the study, as determined by the Investigator.\n* Presence of an untreated or inadequately treated active infection requiring systemic antiviral or antimicrobial therapy at any time during the screening period.\n* Ongoing medical condition (e.g., wasting or cachexia, severe anemia) that according to the Investigator would interfere with the conduct or assessments of the study.\n* History of confounding neuromuscular or neurological disorder that is expected to have a progressive (i.e., worsening) course during the study, and\u002For is expected to be associated with elevations in neurofilament, in the opinion of the Investigator.\n* Female participants who are pregnant or currently breastfeeding.\n* Significant cognitive impairment, clinical dementia, or unstable psychiatric illness, including psychosis, suicidal ideation, suicide attempt, or untreated major depression ≤ 90 days, as determined by the Investigator.\n* History of allergies to a broad range of anesthetics.\n* Tracheostomy.\n* Presence of risk for increased or uncontrolled bleeding and\u002For risk of bleeding that is not managed optimally could place a participant at an increased risk for intraoperative or postoperative bleeding. These could include, but are not limited to, anatomical factors at or near the LP site (e.g., vascular abnormalities, neoplasms, or other abnormalities) and underlying disorders of the coagulation cascade, platelet function, or platelet count (e.g., hemophilia, Von Willebrand's disease, liver disease).\n* Anticipated need, in the opinion of the Investigator, for administration of any antiplatelet or anticoagulant medication that cannot be safely held before and\u002For after an LP procedure according to local or institutional guidelines and\u002For Investigator determination.\n* Presence of an implanted shunt for the drainage of CSF or an implanted CNS catheter.\n* Clinically significant abnormalities in hematology or clinical chemistry parameters, as determined by the Investigator, which would render the participant unsuitable for enrollment.\n* Inability to comply with study requirements.\n* Other unspecified reasons that, in the opinion of the Investigator, make the participant unsuitable for enrollment.",{"count":5,"type":22},[89],"PHASE2","The goal of this clinical trial is to evaluate whether tofersen is safe and effective in adults with non-SOD1 ALS. Tofersen is currently approved by the U.S. Food and Drug Administration to treat SOD1-ALS. The main questions it aims to answer are:\n\n* Does tofersen lower the levels of neurofilament light chain (NfL) in the blood and CSF of adult participants with non-SOD1 ALS?\n* Is tofersen safe and tolerable for adult participants with non-SOD1 ALS?\n* Does tofersen affect other measurements such as clinical outcomes and quality-of-life measures in participants with non-SOD1 ALS?\n\nParticipants will :\n\n* Receive 100mg tofersen via lumbar puncture for 24 weeks. The doses are at the following time points: Weeks 0, 2, 4, 8, 12, 16, 20, and 24.\n* Complete 2 follow-up visits following the end of the dosing period at Weeks 28 and 32.\n* Complete a variety of questionnaires and outcome measurements such as strength and breathing testing.",[29],[93,94,95,96],"als","amyotrophic lateral sclerosis","tofersen","qalsody","2026-06-23",{"date":99,"type":36},"2026-06-25",{"date":101,"type":36},"2025-12-29",{"date":103,"type":22},"2028-05",{"name":105,"class":43},"Washington University School of Medicine",{"id":107,"slug":108,"hasResults":12,"nctId":109,"briefTitle":110,"officialTitle":111,"acronym":4,"eligibilityCriteria":112,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":113,"enrollmentInfo":4,"targetDuration":4,"studyType":114,"phases":4,"briefSummary":115,"conditions":116,"keywords":117,"overallStatus":122,"whyStopped":4,"lastUpdateSubmitDate":97,"lastUpdatePostDateStruct":123,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":124,"locationsCount":127},"100599720","intermediate-size-patient-population-expanded-access-protocol-100599720","NCT07088159","Intermediate-size Patient Population Expanded Access Protocol","Intermediate-size Patient Population Expanded Access Protocol for Tazbentetol in Adult Patients With Amyotrophic Lateral Sclerosis (ALS)","Inclusion Criteria:\n\n* ALS diagnosis\n* Age 18 -85 years at time of signing informed consent form\n* Ineligible for other interventional ALS clinical research participation\n* Vital Capacity greater than 35% of predicted capacity for age, height, and sex\n* If currently taking standard of care treatment for ALS, must be on stable dose for at least 30 days prior to taking tazbentetol.\n* Life expectancy of at least 6 months, according to Investigator's judgement\n\nExclusion Criteria:\n\n* Clinically significant and\u002For unstable medical condition (other than ALS) that would pose a risk to the patient\n* Known ongoing or clinically uncontrolled cardiac disease\n* Clinically significant liver disease\n* Clinical significant cognitive impairment or neurological disorder, as determined by Investigator judgement\n* Concomitant use of another investigational medical product for treatment of ALS\n* Unable to reliably and regularly swallow whole oral medications on a daily basis","85 Years","EXPANDED_ACCESS","The purpose of this Expanded Access Program is to provide tazbentetol to ALS patients who are not eligible to enroll in an ALS clinical trial. This Expanded Access Program will assess safety and tolerability, and clinical efficacy of tazbentelol.",[29],[118,119,29,120,121],"Expanded Access Protocol","synapse","neural connectivity","tazbentelol","AVAILABLE",{"date":99,"type":36},{"name":125,"class":126},"Spinogenix","INDUSTRY",15,{"id":129,"slug":130,"hasResults":12,"nctId":131,"briefTitle":132,"officialTitle":132,"acronym":4,"eligibilityCriteria":133,"healthyVolunteers":12,"sex":17,"minAge":134,"maxAge":4,"enrollmentInfo":135,"targetDuration":4,"studyType":23,"phases":137,"briefSummary":132,"conditions":138,"keywords":4,"overallStatus":70,"whyStopped":4,"lastUpdateSubmitDate":142,"lastUpdatePostDateStruct":143,"startDateStruct":145,"completionDateStruct":147,"leadSponsor":149,"locationsCount":151},"100634721","independence-through-endovascular-neuroprosthetic-technology-intent-an-early-feasibility-study-100634721","NCT07543367","INdependence Through Endovascular Neuroprosthetic Technology (INTENT): an Early Feasibility Study","Inclusion Criteria:\n\n* Able to provide informed consent to participate in the study.\n* Diagnosis of ALS, with bilateral upper-limb paresis.\n* ALS must be refractory to treatment and have been present for a minimum of six months.\n* Aged 22 years or older.\n* Life expectancy greater than 12 months post-implantation.\n* Preserved precentral gyrus assessed using CT.\n* Suitable vascular anatomy assessed using CT venography.\n* Suitable anatomy for subcutaneous pocket creation.\n* Able to undergo anesthesia.\n* Willing and able to comply with all investigational requirements, including clinical testing visits and training visits in the home.\n* Caregiver(s) willing and able to facilitate study visits, including visits to the study site and in the home, and BCI use outside of study visits (e.g., device charging).\n* Patient and caregiver fluent in English.\n* Suitable home environment for BCI training.\n\nExclusion Criteria:\n\n* Active infection or unexplained fever in the 48 hours prior to informed consent.\n* Major psychiatric disorder that may adversely impact the participant's safety or study compliance, including severe depression, psychotic features, personality disorder, severe emotional lability, or substance abuse.\n* Diagnosis of ALS-FTD or another dementia.\n* Active implanted device (e.g., deep brain stimulator, cardiac defibrillator, pacemaker, vagal nerve stimulator, spinal cord stimulator, diaphragmatic pacer, etc.).\n* Known allergy to patient-contacting materials included in the implanted device.\n* Contraindication to angiographic imaging or iodine contrast media.\n* History of central venous sinus thrombosis.\n* Recent history of new venous thromboembolic event (in the 6 months prior to implant), recurrent history of venous thromboembolic disease, or hypercoagulable state.\n* Contraindication to antithrombotic therapy.\n* Participant is at substantially increased risk of infection, including immunocompromised status, recurrent or chronic infection, or poorly controlled diabetes mellitus.\n* Significant risk of non-healing of the subcutaneous pocket incision, including history of chronic non-healing surgical wounds or poorly controlled diabetes mellitus.\n* Pregnant or breast feeding.\n* Patients who are currently enrolled in any other clinical trial that would confound interpretation of safety or effectiveness data or may interfere with the ability to meet study requirements.\n* Any other disease or disorder that could significantly affect participation in the study. Examples may include corrected vision insufficient for viewing computer screens or hearing insufficient for following verbal instructions, which might impact the participant's ability to participate in BCI training and testing.","22 Years",{"count":136,"type":22},10,[25],[139,29,140,141,62],"Neurological Disorder","Motor Neuron Disease","ALS - Amyotrophic Lateral Sclerosis","2026-05-07",{"date":144,"type":36},"2026-05-11",{"date":146,"type":22},"2026-04",{"date":148,"type":22},"2029-12",{"name":150,"class":126},"Synchron, Inc.",5,{"id":153,"slug":154,"hasResults":12,"nctId":155,"briefTitle":156,"officialTitle":157,"acronym":4,"eligibilityCriteria":158,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":159,"enrollmentInfo":160,"targetDuration":4,"studyType":23,"phases":162,"briefSummary":163,"conditions":164,"keywords":165,"overallStatus":70,"whyStopped":4,"lastUpdateSubmitDate":169,"lastUpdatePostDateStruct":170,"startDateStruct":171,"completionDateStruct":173,"leadSponsor":175,"locationsCount":177},"100615698","tongue-strengthening-exercises-in-people-with-als-100615698","NCT07295990","Tongue-strengthening Exercises in People With ALS.","The Impact of Dysphagia Exercise on Oropharyngeal Swallowing Function in Patients With ALS","Inclusion Criteria:\n\n* Diagnosis of possible, probable, or definite ALS (El-Escorial Revisited)\n* Lingual exercises naïve\n* Impaired lingual strength generation compared to normative data\n* EAT-10 score \\\u003C3\n\nExclusion Criteria:\n\n* Stroke\n* Head injury\n* Head and neck cancer\n* Tracheostomy\n* Other concomitant neurogenic disorder\n* Recent oral surgery other than routine dental surgery\n* Unable to generate isometric lingual pressure on lingual manometer\n* Participation in another clinical trial intervention that may confound results\n* NPO (nothing by mouth)\n* Anarthric","99 Years",{"count":161,"type":22},20,[25],"This study is testing a tongue exercise program for people living with ALS to see if it can help support speech and swallowing. All participants will receive the treatment, and researchers will measure changes over time by comparing each person's results to their own earlier results.\n\nPeople who join the study will have two in-person visits and four weekly telehealth sessions with a speech-language pathologist. During these sessions, participants will practice tongue resistance exercises, complete speech and swallowing tasks, and answer surveys about their experience. They will also use a small device at home to measure tongue strength and swallowing.\n\nThe exercise program involves pressing the tongue against a device several times a day, five days per week, for five weeks. Researchers want to learn if this program is safe, practical, and helpful for people with ALS.",[29,141],[62,166,167,168],"Amyotrophic Lateral Sclerosis","Dysphagia","Dysarthria","2026-05-05",{"date":144,"type":36},{"date":172,"type":22},"2026-05-01",{"date":174,"type":22},"2029-01",{"name":176,"class":43},"Nova Southeastern University",1,{"id":179,"slug":180,"hasResults":12,"nctId":181,"briefTitle":182,"officialTitle":183,"acronym":184,"eligibilityCriteria":185,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":186,"targetDuration":4,"studyType":23,"phases":188,"briefSummary":189,"conditions":190,"keywords":195,"overallStatus":70,"whyStopped":4,"lastUpdateSubmitDate":213,"lastUpdatePostDateStruct":214,"startDateStruct":216,"completionDateStruct":218,"leadSponsor":220,"locationsCount":44},"100576240","phase-2-psilocybin-therapy-for-psychological-distress-in-palliative-patients-100576240","NCT06782724","Psilocybin Therapy for Psychological Distress in Palliative Patients","The Safety and Efficacy of Psilocybin Therapy Compared to Low-dose Control in Reducing Depressive Symptoms in Patients With COPD, ALS, MS, or APD.","PsyPal","Inclusion Criteria:\n\n1. Patient has to be diagnosed with one of the following four conditions, defined as:\n\n   COPD i) Diagnosis by medical specialist ii) Postbronchodilator FEV1\u002FFVC \\\u003C 0,7 and FEV1 \\\u003C80% pred iii) ≥ 40 years old iv) ≥ 10 years smoking\n\n   ALS i) ALS according to Goldcoast criteria (Shefner et al, Clin Neurophysiol, 2020) ii) ALS-FRS-R subscores of minimum 1 in item 2, 3 and 8, subscore of minimum 2 in item 1, 4 and 10 and a subscore of minimum 3 in item 11 and 12\n\n   MS i) Fulfilled diagnostic revised McDonald criteria for MS from 2017 (Thompson et al., 2018) ii) EDSS ≥ 1,0\n\n   APD i) Advanced to Late-Stage Parkinson's Disease - patients with a diagnosis of Parkinson's Disease per the MDS clinical diagnosis criteria with evidence of motor and non-motor fluctuations ii) Diseases in the spectrum of Progressive supranuclear palsy (PSP), fulfilling possible and probable criteria, according to the MDS diagnostic criteria iii) Clinically Established and Clinically Probable Multiple System Atrophy (MSA) according to the MDS diagnostic criteria\n2. Patient meets ICD-10 criteria for major depressive disorder documented through the com-pletion of the mood section of the Mini International Neuropsychiatric Interview by a screen-ing psychologist or physician.\n3. Patient has a MADRS score of \\> 19.\n4. Patient should have a life expectancy of at least 6 months (assessed by study physician).\n5. Patient is at least 18 years of age.\n6. Patient has an identified caregiver\u002Fsupport person. See specific conditions for Czechia in Appendix 5.\n7. Patient is able to read and understand the informed consent and all scales used in a local language. For those with ALS, MS, or APD, competency is ensured via neurologist assessment, cognitive screening, caregiver support during screening and interactive approaches where the screening clinician ask the patient to explain their understanding of consent elements, re-explaining potentially misunderstood information.\n8. Patient is able to and willing to adhere to study requirements, including attending all study visits, preparatory and follow-up sessions, and completing all study evaluations.\n9. Patient is able to ingest capsules.\n\nExclusion Criteria:\n\n1. Patient has used a psychedelic substance in the past 6 months (e.g., psilocybin, LSD, 5-MeO-DMT, DMT, ayahuasca or mescaline).\n2. Patient is in active treatments for other psychiatric disorders, judged by the screening clinician to be a more significant clinical problem than depression \u002F distress.\n3. Patient meets ICD-10 criteria for schizophrenia spectrum or other psychotic disorders, including major depressive disorder with psychotic features (except substance\u002Fmedication-induced or due to another medical condition) or bipolar I\u002FII disorder.\n4. Patients with any lifetime diagnosis of schizophrenia spectrum or other psychotic disorders.\n5. Patient has a first-degree relative with schizophrenia spectrum, bipolar I disorder or other psychotic disorders (expect substance\u002Fmedication-induced or due to another medical condition).\n6. Patients with a pre-existing psychiatric condition judged to be incompatible with safe exposure to psilocybin therapy.\n7. Significant suicide risk as defined by (1) suicidal ideation with intent to act (defined as ≥ 5 on MADRS item 10), (2) suicidal attempts within the past year, or (3) clinical assessment of significant suicidal risk during patient interview.\n8. Patient meets ICD-10 criteria for active\u002Fcurrent alcohol or drug use disorder.\n9. Patient has ongoing treatment with antipsychotic drugs. Any prohibited agents must have been stopped at least 5x the elimination half-life of the specific drug at the time of baseline (see Appendix 1a for Prohibited medications).\n10. Patient is unwilling or unable to pause formal psychotherapy (days 0-42).\n11. Patient has neurological conditions (e.g., intracranial tumour, epilepsy, brain injuries, or other neurological disorders) expected by the PIs to conflict with the treatment \u002F study protocol.\n12. Disease-specific exclusion criteria:\n\n    COPD: Unresolved exacerbation or pulmonary infection within last 4 weeks. ALS: Significant cognitive deficits (MoCa, see below). MS: Significant cognitive deficits (MoCa, see below), epilepsy or radiologically isolated syndrome.\n\n    APD: Dementia (MoCa, see below), or Schwab and England ADL scale with scores \\> 80% in the best functional state.\n13. Cardiovascular conditions: recent stroke (\\\u003C 1 year from signing of ICF), recent myocardial infarction (\\\u003C 1 year from signing of ICF), uncontrolled hypertension (blood pressure \\> 140\u002F90 mmHg), clinically significant arrhythmia within 1 year of signing the ICF, or QTc prolongation exceeding 450ms (males) \u002F 470ms (females).\n14. Patient has moderate to severe hepatic impairment (Child-Pugh score ≥ 7).\n15. Patient has insulin-dependent diabetes or who are taking oral hypoglycaemic agents and have a current risk of hypoglycaemia that would require medical intervention.\n16. Patient has any physical or psychological symptoms, medications, blood test results or clinically significant findings at Screening or Baseline (based on the clinical judgement of clinical\u002Fmedical study personnel) that would make a patient unsuitable for the study.\n17. Patient has an allergy or intolerance to any of the materials contained in either drug product.\n18. Cognitive and Neuropsychological assessment: Patients will be excluded if they score below mean minus 1.5 Standard Deviation according to normative age and scholarity adjusted data on the Montreal Cognitive Assessment (MoCA) assessment.\n19. Recent (2 weeks) change or planned change in antidepressant medication during the intervention.\n20. Women who are pregnant, intend to become pregnant during the study or who are currently nursing, or are unwilling to use Highly Effective Contraceptive Methods",{"count":187,"type":22},108,[89],"The goal of this clinical trial is to evaluate whether psilocybin therapy can effectively treat depression and psychological distress in adult patients with COPD, ALS, MS, or APD who have at least 6 months life expectancy. The main questions it aims to answer are:\n\n* Can psilocybin therapy safely reduce depressive symptoms compared to low-dose control?\n* Will the therapeutic effects be rapid and sustained over a 6-month period?\n\nResearchers will compare patients receiving two escalating doses of psilocybin (15mg followed by 25mg) against those receiving two low doses (1mg) to see if the higher doses lead to greater improvements in depression, anxiety, demoralization, and quality of life.\n\nParticipants will:\n\n* Attend three preparation sessions with psychotherapists (1-2 hours each)\n* Undergo two supervised psilocybin dosing sessions (6-8 hours each)\n* Complete five integration therapy sessions following the dosing sessions\n* Participate in follow-up assessments at 6 weeks, 3 months, and 6 months\n* Have access to a digital care platform and peer support groups during the 6-month follow-up period\n* Optional: Control group participants may receive one high-dose psilocybin session (25mg) after the initial study period",[191,29,192,193,194],"COPD (Chronic Obstructive Pulmonary Disease)","MS (Multiple Sclerosis)","Major Depressive Disorder (MDD)","Atypical Parkinson Disease",[196,197,198,199,200,201,202,203,204,205,93,206,207,208,94,209,210,211,212],"psilocybin","therapy","palliative","care","palliative care","end-of-life distress","depression","psychological distress","psypal","copd","ms","apd","chronic obstructive pulmonary disorder","multiple sclerosis","major depressive disorder","atypical parkinson disease","existential distress","2026-05-04",{"date":215,"type":36},"2026-05-08",{"date":217,"type":36},"2025-07-01",{"date":219,"type":22},"2028-01-01",{"name":221,"class":43},"University Medical Center Groningen",{"id":223,"slug":224,"hasResults":12,"nctId":225,"briefTitle":226,"officialTitle":227,"acronym":4,"eligibilityCriteria":228,"healthyVolunteers":53,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":229,"targetDuration":4,"studyType":23,"phases":231,"briefSummary":232,"conditions":233,"keywords":235,"overallStatus":70,"whyStopped":4,"lastUpdateSubmitDate":242,"lastUpdatePostDateStruct":243,"startDateStruct":244,"completionDateStruct":246,"leadSponsor":248,"locationsCount":177},"100624291","clinical-outcome-assessment-for-at--bci-100624291","NCT07407725","Clinical Outcome Assessment for AT & BCI","Development of a Clinical Outcome Assessment for Assistive Technologies and Brain-Computer-Interfaces","Enrollment will involve three different cohorts, namely a cohort of healthy participant, a cohort of ALS\u002FSCI participant who underwent the implant of an invasive BCI device, and a cohort of ALS\u002FSCI participants without any implanted BCI device.\n\nBelow are listed the inclusion and exclusion criteria for each diagnostic group.\n\nSpinal Cord Injury (SCI):\n\nInclusion Criteria:\n\n* Age at or above 18 years old;\n* Diagnosis of spinal cord injury, at the level of T1 or above levels (between C1 and T1);\n* Ability to communicate independently or with a support device, or with a legal representative;\n* Ability to participate in a study session for about 3 hours (e.g., endurance, fatigue), which may include breaks as needed.\n\nExclusion Criteria:\n\n* Participation in another trial that would conflict with the current study or clinical endpoint interference may occur.\n\nAmyotrophic Lateral Sclerosis (ALS):\n\nInclusion criteria:\n\n* Age at or above 18 years old;\n* Diagnosis of amyotrophic lateral sclerosis;\n* Ability to communicate independently, with a support device, or with a legal representative;\n* Ability to participate in a study session for about 3 hours (e.g., endurance, fatigue), which may include breaks as needed.\n\nExclusion criteria:\n\n● Participation in another trial that would conflict with the current study or clinical endpoint interference may occur.\n\nHealthy Controls:\n\nInclusion criteria:\n\n* Age at or above 18 years old;\n* No history of neurological or psychiatric disorders;\n* Ability to provide written informed consent;\n* Ability to participate in a study session for about 3 hours (e.g., endurance, fatigue), which may include breaks as needed.\n\nExclusion criteria:\n\n* Participation in another trial that would conflict with the current study or clinical endpoint interference may occur;\n* Cognitive, visual, or auditory deficits that would interfere with study participation;\n* Current or prior diagnosis or condition that could confound study assessments.",{"count":230,"type":22},60,[25],"Many individuals with severe motor impairments rely on Assistive Technologies (ATs) or Brain-Computer Interfaces (BCIs) to interact with digital devices such as their computers. Clinicians and researchers currently lack a common framework to objectively quantify how much a given AT or BCI improves real-world function or to compare across tools. This project seeks to address this gap by developing a standardized method to objectively assess or compare the functional benefit of these tools on digital independence, i.e., the ability to independently operate computers, phones, and other digital systems, by creating a unique Digital Assessment Interface (DAI).\n\nThis assessment will be a simulation of online and digital activities that prior work has determined is important to functional daily living in the digital domain. Participants will complete this assessment with various ATs and BCIs, and these scores will be used to create an index, which will be comprised of performance outcomes, clinician-reported outcomes, and patient-reported outcomes.\n\nThe tool aims to quantify and compare digital task performance across devices and user populations. The primary objective of this study is to develop an index. The index will quantify functional performance of individuals using various ATs and BCIs. The secondary objectives are to extensively evaluate the psychometric properties of the index, such as the validity, responsiveness, reliability, and floor\u002Fceiling effects both globally and across different devices and impairment levels, ensuring that it can reliably measure the impact of an AT or BCI on a user's ability to independently operate digital systems; and to characterize the familiarization and use of specific BCI and AT systems with reference to a normative healthy control population.",[234,29],"Spinal Cord Injury",[236,237,238,239,240,241],"iBCI","Brain Computer Interface","Assistive Technology","Clinical Outcome Assessment","dADL","digital activities of daily living","2026-04-29",{"date":169,"type":36},{"date":245,"type":36},"2026-01-08",{"date":247,"type":22},"2027-06",{"name":249,"class":43},"Shirley Ryan AbilityLab",{"id":251,"slug":252,"hasResults":12,"nctId":253,"briefTitle":254,"officialTitle":255,"acronym":256,"eligibilityCriteria":257,"healthyVolunteers":12,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":258,"targetDuration":4,"studyType":260,"phases":4,"briefSummary":261,"conditions":262,"keywords":4,"overallStatus":70,"whyStopped":4,"lastUpdateSubmitDate":270,"lastUpdatePostDateStruct":271,"startDateStruct":273,"completionDateStruct":275,"leadSponsor":277,"locationsCount":177},"100628864","invasive-home-ventilation-in-denmark-100628864","NCT07467187","Invasive Home Ventilation in Denmark","The Evolution of Invasive Home Mechanical Ventilation in Denmark","HOMEVENT DK","Inclusion Criteria:\n\n* Patients are included if they have or have had a respiratory certified personale care assistent during the period 2016-2025.\n\nExclusion Criteria:\n\n* Not tracheostomized\n* Tracheostomized but on spontaneous breathing throughout 1 January 2016 - 31 December 2025\n* Not discharged to home, assisted living, nursing home, or rehabilitation during 1 January 2016 - 31 December 2025",{"count":259,"type":22},450,"OBSERVATIONAL","The aim of this study is to describe national trends over the past 10 years in patients receiving invasive home mechanical ventilation (HMV) in Denmark. This includes indications for invasive HMV, diagnostic groups, and one-year mortality.",[263,29,264,265,266,267,268,269],"Neuromuscular Diseases (NMD)","Spinal Cord Injuries (SCI)","Duchenne Muscular Dystrophy (DMD)","SMA - Spinal Muscular Atrophy","MSA - Multiple System Atrophy","Tracheostomized Patients","Tracheostomy","2026-04-15",{"date":272,"type":36},"2026-04-20",{"date":274,"type":36},"2026-04-13",{"date":276,"type":22},"2028-09-01",{"name":278,"class":43},"Rigshospitalet, Denmark",{"id":280,"slug":281,"hasResults":12,"nctId":282,"briefTitle":283,"officialTitle":283,"acronym":284,"eligibilityCriteria":285,"healthyVolunteers":53,"sex":17,"minAge":286,"maxAge":287,"enrollmentInfo":288,"targetDuration":4,"studyType":23,"phases":290,"briefSummary":291,"conditions":292,"keywords":305,"overallStatus":70,"whyStopped":4,"lastUpdateSubmitDate":307,"lastUpdatePostDateStruct":308,"startDateStruct":310,"completionDateStruct":312,"leadSponsor":314,"locationsCount":316},"100299199","the-swedish-biofinder-2-study-100299199","NCT03174938","The Swedish BioFINDER 2 Study","BioFINDER2","COHORT A: Cognitively healthy younger individuals (40-65 years of age) INCLUSION CRITERIA\n\n* Age 40-65 years\n* Absence of cognitive symptoms as assessed by a physician with special interest in cognitive disorders.\n* MMSE score 27-30 at screening visit.\n* Do not fulfill the criteria for MCI or any dementia according to DSM-V.\n* Speaks and understands Swedish to the extent that an interpreter is not necessary for the patient to fully understand the study information and cognitive tests.\n\nEXCLUSION CRITERIA\n\n* Significant unstable systemic illness or organ failure, such as terminal cancer, that makes it difficult to participate in the study.\n* Current significant alcohol or substance misuse.\n* Significant neurological or psychiatric illness.\n* Refusing lumbar puncture, MRI or PET.\n\nCOHORT B: Cognitively healthy elderly individuals (66-100 years of age) INCLUSION CRITERIA\n\n* Age 66-100 years\n* Absence of cognitive symptoms as assessed by a physician with special interest in cognitive disorders.\n* MMSE score 26-30 at screening visit.\n* Do not fulfill the criteria for MCI or any dementia according to DSM-V.\n* Speaks and understands Swedish to the extent that an interpreter is not necessary for the patient to fully understand the study information and cognitive tests.\n\nEXCLUSION CRITERIA\n\n* Significant unstable systemic illness or organ failure, such as terminal cancer, that makes it difficult to participate in the study.\n* Current significant alcohol or substance misuse.\n* Significant neurological or psychiatric illness.\n* Refusing lumbar puncture, MRI or PET.\n\nCOHORT C: Subjective cognitive decline and mild cognitive impairment INCLUSION CRITERIA\n\n* Age 40-100 years.\n* Referred to the memory clinics due to cognitive symptoms experienced by the patient and\u002For informant. These symptoms do not have to be memory complaints, but could also be executive, visuospatial, language, praxis, psychomotor or social cognitive complaints.\n* MMSE score of 24 - 30 points.\n* Do not fulfill the criteria for any dementia (major neurocognitive disorder) according to DSM-V.\n* The medical doctor (after clinical assessments, cognitive testing, CSF analyses and structural brain imaging) believes the cognitive complaints are caused by an incipient neurocognitive disorder of any sort. This is defined as any case fulfilling the criteria above (i.e. both SCD and MCI) with an abnormal CSF Aβ42\u002F40 ratio, which is strongly associated with brain Aβ pathology and prodromal Alzheimer's disease. Further, cases with MCI (=minor neurocognitive impairment) due to either Parkinson's disease, Lewy body disease, vascular neurocognitive disorder or frontotemporal dementia (please see Appendix below for clinical criteria and references) can also be included.\n* Speaks and understands Swedish to the extent that an interpreter is not necessary for the patient to fully understand the study information and cognitive tests.\n\nEXCLUSION CRITERIA\n\n* Significant unstable systemic illness or organ failure, such as terminal cancer, that makes it difficult to participate in the study.\n* Current significant alcohol or substance misuse.\n* Refusing lumbar puncture, MRI or PET.\n\nCOHORT D: Dementia due to Alzheimer's disease INCLUSION CRITERIA\n\n* Age 40-100 years.\n* Referred to the memory clinics due to cognitive symptoms experienced by the patient and\u002For informant. These symptoms do not have to be memory complaints, but could also be executive, visuospatial, language, praxis or psychomotor complaints.\n* MMSE score of 12-26 points.\n* Fulfill the criteria for dementia (major neurocognitive disorder) due to Alzheimer's disease (DSM-V).\n* Speaks and understands Swedish to the extent that an interpreter was not necessary for the patient to fully understand the study information and cognitive tests.\n\nEXCLUSION CRITERIA\n\n* Significant unstable systemic illness or organ failure, such as terminal cancer, that makes it difficult to participate in the study.\n* Current significant alcohol or substance misuse.\n* Refusing lumbar puncture, MRI or PET.\n\nCOHORT E: Other dementias INCLUSION CRITERIA\n\n* Age 40-100 years.\n* Fulfill the criteria for dementia (major neurocognitive disorder) due to FTD, PDD, DLB or subcortical VaD alternatively the criteria for PD, PSP, MSA, CBS or ALS.\n* Speaks and understands Swedish to the extent that an interpreter was not necessary for the patient to fully understand the study information and cognitive tests.\n\nEXCLUSION CRITERIA\n\n* Significant unstable systemic illness or organ failure, such as terminal cancer, that makes it difficult to participate in the study.\n* Current significant alcohol or substance misuse.\n* Refusing lumbar puncture, MRI or PET.","20 Years","100 Years",{"count":289,"type":22},2950,[25],"The Swedish BioFINDER 2 study is a new study that will launch in 2017 and extends the previous cohorts of BioFINDER 1 study (www.biofinder.se). BioFINDER 1 is used e.g. to characterize the role of beta-amyloid pathology in early diagnosis of Alzheimer's disease (AD) using amyloid-PET (18F-Flutemetamol) and Aβ analysis in cerebrospinal fluid samples. The BioFINDER 1 study has resulted in more than 40 publications during the last three years, many in high impact journals, and some the of the results have already had important implications for the diagnostic work-up patients with AD in the clinical routine practice.\n\nThe original BioFINDER 1 cohort started to include participants in 2008. Since then there has been a rapid development of biochemical and neuroimaging technologies which enable novel ways to the study biological processes involved in Alzheimer's disease in living people. There has also been a growing interest in the earliest stages of AD and other neurodegenerative diseases. With the advent of new tau-PET tracers there is now an opportunity to elucidate the role of tau pathology in the pathogenesis of AD and other tauopathies. The Swedish BioFINDER 2 study has been designed to complement the BioFINDER 1 study and to e.g. address issues regarding the role of tau pathology in different dementias and in preclinical stages of different dementia diseases. Further, the clinical assessments and MRI methods have been further optimized compared to BioFINDER 1. Detailed assessments of motor aspects and dual task performance, which is part of a sub-study named Motor-ACT: \"Motor aspects and activities in relation to cognitive decline and brain pathologies, has been added to further optimize assessment of motor function.",[293,294,295,296,297,298,299,300,301,302,303,304,29],"Dementia","Alzheimer Disease","Parkinson Disease","Lewy Body Disease","Parkinson-Dementia Syndrome","Frontotemporal Degeneration","Semantic Dementia","Progressive Nonfluent Aphasia","Progressive Supranuclear Palsy","Corticobasal Degeneration","Multiple System Atrophy","Mild Cognitive Impairment",[306],"Early diagnosis, biomarker, PET, MRI, β-amyloid, tau, CSF, cognitive test","2026-04-01",{"date":309,"type":36},"2026-04-06",{"date":311,"type":36},"2017-05-15",{"date":313,"type":22},"2036-12",{"name":315,"class":43},"Skane University Hospital",2,{"id":318,"slug":319,"hasResults":12,"nctId":320,"briefTitle":321,"officialTitle":321,"acronym":322,"eligibilityCriteria":323,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":324,"targetDuration":4,"studyType":260,"phases":4,"briefSummary":326,"conditions":327,"keywords":332,"overallStatus":70,"whyStopped":4,"lastUpdateSubmitDate":342,"lastUpdatePostDateStruct":343,"startDateStruct":345,"completionDateStruct":347,"leadSponsor":349,"locationsCount":44},"100581941","disease-biosignatures-in-alsftd-spectrum-new-impactful-biological-perspectives-beyond-clinical-approaches-100581941","NCT06856850","Disease Biosignatures in ALS\u002FFTD Spectrum: New Impactful Biological Perspectives Beyond Clinical Approaches","SPECTRALS","Inclusion Criteria:\n\n* Clinical criteria for ALS (Brooks et al., 2000; de Carvalho M., 2008), FTD (GornoTempini et al., 2011; Rascovsky et al., 2011)\n\nExclusion Criteria:\n\n* na",{"count":325,"type":22},230,"Diagnosis of ALS\u002FFTD disease spectrum is challenging because it largely relies on clinical symptoms. Identifying novel biomarkers is essential for a paradigm shift towards a more precise biological-based diagnosis. To achieve this aim, having access to proper specimens and analytical methods is crucial. Our team of experts in neurology, biology, chemistry, physics, and AI will explore ALS\u002FFTD from novel perspectives using transcriptomics, proteomics, genomics and other innovative approaches to analyzing easily accessible tissues. The seed amplification assay (SAA) will be also exploited to detect pathological TDP-43. This project aims to create disease fingerprints useful for patient stratification and monitoring of disease progression, and to evaluate the therapeutic efficacy in clinical trials, thus overcoming the limits of clinical interpretation. Discovering new biomarkers and cellular pathways will improve the diagnosis and treatment of these devastating diseases.",[29,63,328,329,330,331],"Neuropathic","Psychiatric Disorders","Idiopathic Intracranial Hypertension","Frontotemporal Dementia (FTD)",[333,334,335,336,337,338,339,340,341],"Amyotrophic lateral sclerosis","frontotemporal dementia","microbiota","miRNA","protein-NMR","TDP-43","endocytic disfunction","seed amplification assay","peripheral biomarker","2026-03-25",{"date":344,"type":36},"2026-03-30",{"date":346,"type":36},"2025-02-27",{"date":348,"type":22},"2026-08",{"name":350,"class":43},"Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta",{"id":352,"slug":353,"hasResults":12,"nctId":354,"briefTitle":355,"officialTitle":355,"acronym":356,"eligibilityCriteria":357,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":358,"enrollmentInfo":359,"targetDuration":4,"studyType":260,"phases":4,"briefSummary":360,"conditions":361,"keywords":362,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":365,"lastUpdatePostDateStruct":366,"startDateStruct":368,"completionDateStruct":369,"leadSponsor":371,"locationsCount":21},"100629772","characterization-of-platelet-molecular-profiles-in-als-for-the-identification-of-specific-diagnostic-biomarkers---a-pilot-study-100629772","NCT07479017","Characterization of Platelet Molecular Profiles in ALS for the Identification of Specific Diagnostic Biomarkers - A Pilot Study","SLA-PlaQ","Inclusion Criteria:\n\nPatients with ALS:\n\n* Men or women aged 18 to 75\n* ALS diagnosed according to the El Escorial criteria\n* ALS diagnosis less than 3 months ago\n* Onset of symptoms defined as the time when muscle weakness was first observed by the patient less than 2 years ago\n\nControls with another motor neuron disease:\n\n* Men or women aged 18 to 75\n* Diagnosis of motor neuron disease \\\u003C 3 months\n\nExclusion Criteria:\n\n* Genetic variants associated with ALS\n* Pregnant or breastfeeding women\n* Treatment with oral or injectable anticoagulants, antiplatelet agents (EXCEPT aspirin at the maximum authorized dosage of 160 mg per day)\n* Uncontrolled diabetes\n* Persons deprived of their liberty by judicial or administrative decision\n* Persons subject to legal protection measures: guardianship or curatorship\n* Opposition to data processing","75 Years",{"count":230,"type":22},"The search for diagnostic biomarkers that can be used routinely is a major challenge to manage Amyotrophic lateral sclerosis (ALS) in order to characterize the pathophysiology and accelerate the management of the disease. Some non-specific biomarkers have been proposed (Neurofilaments, TDP-43) but their diagnostic value remains controversial. This study aims to identify ALS-specific platelet biomarkers using targeted and untargeted multi-omic approaches, in order to enable differential diagnosis between ALS and other motor neuron diseases.",[29],[363,364],"diagnostic biomarkers","platelet molecular profiles","2026-03-12",{"date":367,"type":36},"2026-03-18",{"date":146,"type":22},{"date":370,"type":22},"2027-08",{"name":372,"class":43},"University Hospital, Tours",{"id":374,"slug":375,"hasResults":12,"nctId":376,"briefTitle":377,"officialTitle":378,"acronym":379,"eligibilityCriteria":380,"healthyVolunteers":12,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":381,"targetDuration":4,"studyType":23,"phases":382,"briefSummary":383,"conditions":384,"keywords":385,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":389,"lastUpdatePostDateStruct":390,"startDateStruct":392,"completionDateStruct":393,"leadSponsor":395,"locationsCount":4},"100629368","virtual-reality-for-anxiety-management-in-persons-with-amyotrophic-lateral-sclerosis-100629368","NCT07473765","Virtual Reality for Anxiety Management in Persons With Amyotrophic Lateral Sclerosis","Just... Breathe: Virtual Reality Guided Breathing Exercise for Anxiety Management in Amyotrophic Lateral Sclerosis","VR ALS","Inclusion Criteria:\n\n* Diagnosed with ALS (gold coast or El-Escorial), or ALS variants of flail limb, progressive muscle atrophy, UMN or LMN predominant\n* Able to use VR device with no negative effects (i.e. headaches, vision changes, dizziness, nausea, disorientation)\n\nExclusion Criteria:\n\n\\- Cognitive impairment that impacts ability to participate fully in assessments",{"count":230,"type":22},[25],"Virtual Reality (VR) is gaining traction as a new and innovative leisure to augment healthcare services. Several benefits of the leisure experience, such as distraction and full sensory immersion, have demonstrated a potential to significantly impact the field of healthcare through pain reduction, anxiety reduction, and is seen as an innovative approach to motor learning. Persons with ALS (pwALS) have a high prevalence of anxiety over the course of their illness, which has a negative impact on their quality of life, and the quality of life of those closest to them. The use of VR for anxiety management and subsequent quality of life improvement has yet to be explored in the ALS population.\n\nFor individuals with ALS, VR can be both (1) an escape from the reality of living day to day with a progressive fatal diagnosis; and (2) the opportunity to potentially improve anxiety, both of which are linked to the quality of life of individuals living with ALS. Our hypothesis is that a simple and accessible home VR-guided relaxation exercise program can improve subjective anxiety symptoms in a person living with ALS and subsequently improve quality of life.",[29],[386,387,388],"Amyotrophic Lateral Sclerosis (ALS)","Virtual Reality","Anxiety Management","2026-03-11",{"date":391,"type":36},"2026-03-16",{"date":307,"type":22},{"date":394,"type":22},"2027-06-30",{"name":396,"class":43},"Horizon Health Network",{"id":398,"slug":399,"hasResults":12,"nctId":400,"briefTitle":401,"officialTitle":402,"acronym":4,"eligibilityCriteria":403,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":358,"enrollmentInfo":404,"targetDuration":4,"studyType":23,"phases":406,"briefSummary":408,"conditions":409,"keywords":4,"overallStatus":70,"whyStopped":4,"lastUpdateSubmitDate":410,"lastUpdatePostDateStruct":411,"startDateStruct":413,"completionDateStruct":415,"leadSponsor":417,"locationsCount":419},"100600640","phase-1-a-study-of-ly4256984-in-participants-with-sporadic-amyotrophic-lateral-sclerosis-100600640","NCT07100119","A Study of LY4256984 in Participants With Sporadic Amyotrophic Lateral Sclerosis","A Multiple Ascending Dose Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered LY4256984 in Participants With Sporadic Amyotrophic Lateral Sclerosis","Inclusion Criteria:\n\n* Have a definite, possible, or probable diagnosis of sporadic amyotrophic lateral sclerosis (ALS) made by a physician experienced with the management of ALS\n* ALS symptom onset as determined by the Investigator within 24 months of Screening\n* Have a body mass index (BMI) within the range of greater than or equal to 18.0 and less than or equal to 35.0 kilogram per square meter (kg\u002Fm²) (inclusive)\n\nExclusion Criteria:\n\n* Have a history or presence of medical illness including, but not limited to, any cardiovascular, renal, hepatic, gastrointestinal, respiratory, hematological, endocrine, psychiatric, or neurological disease, convulsions, or any clinically significant laboratory abnormality\n* Have a history of another neurodegenerative disease or significant dementia\u002Fsevere cognitive problems\n* Have serum alanine aminotransferase (ALT), aspartate transferase (AST), or total bilirubin levels greater than 2 x upper limit of normal.\n* Have a significant renal impairment (estimated glomerular filtration rate \\\u003C60 milliliters per minute \\[mL\u002Fmin\\]\u002F1.73 m²).\n* Have a 12-lead electrocardiogram (ECG) abnormality at screening, in the opinion of the investigator, that increases the risks associated with participating in the study\n* Show clinically significant abnormalities in lumbar spine previously known or determined by screening lumbar X-ray or fluoroscopy (if performed)",{"count":405,"type":22},32,[407],"PHASE1","The purpose of this study is to evaluate how well LY4256984 is tolerated and what side effects may occur in participants with sporadic amyotrophic lateral sclerosis (ALS). The study drug will be administered intrathecally (IT) into the spine. Blood tests will be performed to check how much LY4256984 gets into the bloodstream and how long it takes the body to eliminate it.",[29],"2026-03-05",{"date":412,"type":36},"2026-03-06",{"date":414,"type":36},"2025-08-05",{"date":416,"type":22},"2027-09",{"name":418,"class":126},"Eli Lilly and Company",12,{"id":421,"slug":422,"hasResults":12,"nctId":423,"briefTitle":424,"officialTitle":425,"acronym":426,"eligibilityCriteria":427,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":428,"enrollmentInfo":429,"targetDuration":4,"studyType":23,"phases":430,"briefSummary":431,"conditions":432,"keywords":433,"overallStatus":70,"whyStopped":4,"lastUpdateSubmitDate":439,"lastUpdatePostDateStruct":440,"startDateStruct":442,"completionDateStruct":444,"leadSponsor":446,"locationsCount":177},"100623783","phase-1-safety-and-tolerability-study-of-ctx1000-in-participants-with-amyotrophic-lateral-sclerosis-100623783","NCT07401121","Safety and Tolerability Study of CTx1000 In Participants With Amyotrophic Lateral Sclerosis","Koanewa: A First in Human, Phase 1b, Open-label, Non-randomised, Single Dose Study to Assess the Safety and Tolerability of CTx1000 in Participants Diagnosed With Amyotrophic Lateral Sclerosis","KOANEWA","Inclusion Criteria:\n\n* Diagnosis of ALS in accordance with the revised El Escorial criteria and TRICALS risk score\n* An overall disease duration of ≤ 2 years after the participant's first symptoms\n* No or low circulating anti-AAV9 antibodies (titre ≤ 1:50\n* Stable dosing with a standard of care ALS medication (eg, riluzole and edaravone) and other prescription medications for 30 days prior to Screening\n* Not pregnant or breastfeeding, or willing to cease breastfeeding\n* All participants must use a barrier method of contraception\n\nExclusion Criteria:\n\n* Any participants with genetic forms of ALS, including C9ORF72 repeat carriers, except for TARDBP gene variants, as confirmed by previous clinical history genetic testing\n* Any history of myocardial infarction or stroke within 6 months prior to Screening, or uncontrolled diabetes (HbA1C \\> 9%)\n* Positive test for cytomegalovirus, hepatitis C antibody (HCV), hepatitis B surface antigen (HBsAg), human immunodeficiency virus (HIV) antibody.\n* Inadequate organ function\n* Any participant with a current open tracheostomy","80 Years",{"count":127,"type":22},[407],"This clinical study is in participants with Amyotrophic Lateral Sclerosis and is designed to evaluate the safety and tolerability of the gene therapy CTx1000.",[29],[434,435,436,437,438],"CTx1000","gene therapy","motor neuron disease","genetic medicine","Lou Gehrig disease","2026-02-06",{"date":441,"type":36},"2026-02-10",{"date":443,"type":36},"2026-01-12",{"date":445,"type":22},"2030-12",{"name":447,"class":126},"Celosia Therapeutics Pty Ltd",{"id":449,"slug":450,"hasResults":12,"nctId":451,"briefTitle":452,"officialTitle":453,"acronym":454,"eligibilityCriteria":455,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":456,"targetDuration":458,"studyType":260,"phases":4,"briefSummary":459,"conditions":460,"keywords":461,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":439,"lastUpdatePostDateStruct":474,"startDateStruct":475,"completionDateStruct":476,"leadSponsor":478,"locationsCount":4},"100623727","healey-als-mymatch-common-screening-protocol-100623727","NCT07400393","Healey ALS MyMatch Common Screening Protocol","Healey ALS MyMatch Common Screening Protocol (MCSP) for Determining Preliminary Eligibility for ALS MyMatch Early Phase Clinical Trials","MCSP","Inclusion Criteria:\n\n1. Ability to provide written informed consent.\n2. Adults \\> 18 years of age.\n3. Diagnosis of symptomatic ALS that meets either the revised El Escorial Criteria.(clinically possible, probable, probable lab-supported, or definite) or the Gold Coast Criteria.\n4. Available or pending clinically obtained CLIA ALS genetic panel report.\n5. Time since onset of weakness due to ALS ≤ 24 months at the time of MCSP screening.\n6. Slow VC ≥ 65% of predicted capacity value for gender, height and age at screening.\n7. Clinically unremarkable Complete Blood Counts as per SI's discretion, including but not limited to Hemoglobin ≥ 9 g\u002FdL.\n8. Ability and willingness to complete all study procedures per SI's assessment.\n9. Negative pregnancy test at screening for women of child-bearing potential (WOCB), defined as a sexually mature woman who has not undergone a hysterectomy or who has not been naturally postmenopausal for at least 24 consecutive months (i.e., who has had menses any time in the preceding 24 consecutive months).\n\nExclusion Criteria:\n\n1. Clinically significant unstable medical or surgical condition that would pose a risk to the participant's trial procedural participation or interfere with data collection, per SI's assessment.\n2. Presence of cognitive or mental health disorders impairing ability to provide informed consent for the study per SI assessment.\n3. Active cancer or history of cancer, unless it was successfully treated for durable remission or cure more than 3 years ago. (Note that basal cell carcinoma, squamous cell carcinoma in situ, cervical carcinoma in situ, prostatic carcinoma in situ, or other malignancies that have been curatively excised at any time previously and with no evidence of disease recurrence for at least 3 years are not exclusionary.)\n4. Prior solid organ transplantation.\n5. Use of investigational treatments for ALS (off-label use or active participation in a clinical trial) within 5 half-lives (if known) or 30 days (whichever is longer) prior to the MCSP Screening Visit. (Please refer to the Manual of Procedures (MOP) for current list of experimental therapies)\n6. Screening 12-lead ECG showing QT interval corrected for rate (QTcF) \\> 470 msec for women and \\> 450 msec for men, absence of second degree or higher AV block or other clinically significant cardiac arrythmias.\n7. Clinically significant abnormalities in the Comprehensive Metabolic Panel per SI's assessment, including but not limited to:\n\n   1. Serum alanine aminotransferase or aspartate aminotransferase \\> 3 times the upper limit of normal, or serum bilirubin \\> 1.5 × upper limit of normal\n   2. Estimated GFR (eGFR) of \\\u003C 30 mL\u002Fmin\u002F1.73m2\n8. Other clinically significant electrolyte and metabolic abnormalities\n9. If female, breastfeeding, pregnant, or of child-bearing potential and unwilling to use effective contraception for duration of the trial and after discontinuing treatment as outlined in the ALS MyMatch trial protocol.\n10. Clinically significant unstable medical conditions (other than ALS) that would pose a risk to the participant, per SI's assessment (e.g., cardiovascular instability, systemic infection,), or clinically significant laboratory abnormality or ECG changes.\n11. Exposure at any time to any gene therapies under investigation for the treatment of ALS (off-label use or investigational).\n12. Participants who require Permanent assisted ventilation (PAV). PAV defined as more than 22 hours per day of noninvasive or invasive mechanical ventilation for more than seven consecutive days. The date of onset of PAV is the first day of the seven days.",{"count":457,"type":22},500,"45 Days","The goal of the Healey ALS MyMatch Common Screening Protocol (MCSP), an observational study, is to identify individuals with ALS who may be eligible to be matched to a currently enrolling ALS MyMatch trial. Participants will complete a MCSP Screening Visit and undergo clinical assessments, laboratory testing, and biomarker analyses to determine preliminary trial eligibility. The study also characterizes clinical, genetic, and biofluid biomarker profiles, assesses the prevalence of ALS-associated gene variants, and banks blood samples for future ALS and biomarker research. MCSP enables simultaneous screening for multiple trial-specific biomarkers and uses a targeted medical history form to optimize matching of participants to appropriate MyMatch trials.",[62,29,141],[62,166,462,463,464,454,465,466,467,468,469,470,471,472,473],"MyMatch","ALS MyMatch","MyMatch Program","MyMatch Common Screening Protocol","Early Phase","Biomarker","Phase I","Phase II","Healey","NCRI","ALS Trials","MGB",{"date":441,"type":36},{"date":391,"type":22},{"date":477,"type":22},"2029-03-16",{"name":479,"class":43},"Massachusetts General Hospital",{"id":481,"slug":482,"hasResults":12,"nctId":483,"briefTitle":484,"officialTitle":485,"acronym":4,"eligibilityCriteria":486,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":358,"enrollmentInfo":487,"targetDuration":4,"studyType":23,"phases":489,"briefSummary":490,"conditions":491,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":495,"lastUpdatePostDateStruct":496,"startDateStruct":498,"completionDateStruct":500,"leadSponsor":502,"locationsCount":4},"100623452","phase-1-kamlanoflast-in-amyotrophic-lateral-sclerosis-100623452","NCT07396818","Kamlanoflast In Amyotrophic Lateral Sclerosis","A Trial of Kamlanoflast In Patients With Amyotrophic Lateral Sclerosis","Inclusion Criteria:\n\n1. Diagnosis of definite, probable, laboratory-supported probable, or possible ALS by revised El Escorial research criteria.\n2. Ages 18 to 75 years.\n3. Onset of weakness within three years of study enrollment.\n4. ALS with progression, characterized either by:\n\ni. a reduction of 0.5 points per month or greater on the ALS Functional Rating Scale-Revised (ALSFRS-R), which will be calculated based on (most recent ALSFRS-R at least 12 weeks from screening - ALSFRS-R at screening)\u002Ftime interval; or ii. a calculated progression rate: (48 - ALSFRS-R at \"time of diagnosis\") \u002F duration from onset to diagnosis (month) that is 0.5 points per month or greater.\n\ne) Plasma NfL levels ≥ 2 times the upper limit of the age-specific reference values for normal at the measuring laboratory at screening.\n\nf) Capable of providing informed consent. g) Capable and willing to follow study protocol. h) Ability to swallow pills and liquids at the time of the screening visit and, in the investigator's opinion have the ability to swallow for the duration of the study OR can be fed via a Gastrostomy (G) tube or Percutaneous Endoscopic capacity (PEG) tube.\n\ni) Slow vital capacity (SVC) \\> 65% of predicted value for gender, height, and age (participants perform SVC for three trials and the best SVC will be used).\n\nj) Females of childbearing potential must agree to abstain from sex or use adequate method of contraception for the duration of the study period and for 28 days after the last dose of study drug.\n\nk) Males must agree to abstain from sex or use adequate method of contraception for the duration of the study period and for 28 days after the last dose of study drug.\n\nl) If an approved therapy for ALS is used during the study, a steady dose must be used as follows: i. Participants who do not currently receive riluzole and do not plan to receive riluzole during the study period. Participants receiving riluzole are on a stable dose for at least 4 weeks before enrollment. Participants receiving riluzole are expected to remain on the same dose throughout the duration of the study.\n\nii. Participants who do not currently receive edaravone and do not plan to receive edaravone during the study period. Participants receiving edaravone must have completed at least 1 cycle of treatment before enrollment and are expected to continue edaravone treatment throughout the duration of the study.\n\nExclusion Criteria:\n\n1. Inability to follow the study protocol, based on the investigator's assessment.\n2. Pregnant or nursing women.\n3. Recently（within 28 days）received other experimental treatments.\n4. Presence of any active infections or inflammatory diseases at the time of enrollment that may confound the assessment of levels of inflammatory markers.\n5. Taking any medication or supplements with anti-inflammatory effects, including but not limited to prednisone, colchicine, or curcumin.\n6. Taking Qalsody (tofersen).\n7. Taking any medications containing nucleotide reverse transcriptase inhibitors (NRTIs), including but not limited to Abacavir, Emtricitabine, Lamivudine, or Zidovudine; trade names Atripla, Biktarvy, Cimduo, Combivir, Complera, Delstrigo, Descovy, Dovato, Emtriva, Epivir, Epzicom, Genvoya, Odefsey, Retrovir, Stribild, Symfi, Symtuza, Triumeq, Trizivir, Truvada, Ziagen.\n8. Clinically significant unstable medical condition (other than ALS) that would pose a risk to the participant, according to investigator's judgment (e.g., cardiovascular instability, systemic infection), or clinically significant laboratory abnormality.\n9. Clinically significant abnormal liver or kidney function at baseline (pre-dose). The following values \\[alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \\> 3 times the upper limit of normal (ULN) or estimated Glomerular Filtration Rate (eGFR) \\\u003C 30 mL\u002Fmin\u002F1.73m2\\] are exclusionary regardless of clinical symptoms.\n10. Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair ability of the participant to provide informed consent, in the investigator's opinion.\n11. Active cancer or history of cancer, except for the following: basal cell carcinoma or successfully treated squamous cell carcinoma of the skin, cervical carcinoma in situ, prostatic carcinoma in situ, or other malignancies curatively treated and with no evidence of disease recurrence for at least 3 years.\n12. Non-invasive ventilation, tracheostomy, oxygen supplementation for primary pulmonary pathology.\n13. Current \u002F anticipated need of diaphragm pacing system (DPS).\n14. History of prior AAV gene therapy for any indication;\n15. Presence of any clinically relevant diseases that, in the research team's opinion, would prevent the subject from completing the study, including but not limited to severe cognitive dysfunction or medical conditions other than ALS that affect physical function or life expectancy.\n16. Plan to move away from the study site within the next 6 months.",{"count":488,"type":22},40,[407,89],"This is a study of Kamlanoflast in patients with ALS. Kamlanoflast is orally administered over 24 weeks. Its effects on inflammatory and functional parameters will be studied. Information on safety and tolerability will be collected.",[29,62,492,493,494],"Neuro-Degenerative Disease","Neuro-Degenerative Diseases","Motor Neuron Disease (MND)","2026-02-02",{"date":497,"type":36},"2026-02-09",{"date":499,"type":22},"2026-02",{"date":501,"type":22},"2027-01",{"name":503,"class":126},"Inflammasome Therapeutics",{"id":505,"slug":506,"hasResults":12,"nctId":507,"briefTitle":508,"officialTitle":509,"acronym":4,"eligibilityCriteria":510,"healthyVolunteers":53,"sex":17,"minAge":18,"maxAge":428,"enrollmentInfo":511,"targetDuration":4,"studyType":23,"phases":512,"briefSummary":514,"conditions":515,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":516,"lastUpdatePostDateStruct":517,"startDateStruct":519,"completionDateStruct":520,"leadSponsor":521,"locationsCount":4},"100617687","phase-2-this-study-evaluates-the-safety-target-engagement-and-preliminary-efficacy-of-galunisertib-tgf-r1alk5-inhibitorcombined-with-nerandomilast-pde4-inhibitor-in-grem2-positive-als-a-biomarker-defined-subgroup-hypothesized-to-reflect-heightened-tgf-smad-driven-astrocytic-and-fibrotic-signaling-100617687","NCT07321860","This Study Evaluates the Safety, Target Engagement, and Preliminary Efficacy of Galunisertib (TGF-βR1\u002FALK5 Inhibitor)Combined With Nerandomilast (PDE4 Inhibitor) in GREM2-positive ALS, a Biomarker-defined Subgroup Hypothesized to Reflect Heightened TGF-β\u002FSMAD-driven Astrocytic and Fibrotic Signaling","Randomized, Double-Blind, Placebo-Controlled Phase 2a Study of Partial TGF-βR1 (ALK5) Inhibition With Galunisertib Combined With PDE4 Inhibition With Nerandomilast in GREM2-Positive ALS","Inclusion Criteria\n\nParticipants must meet all of the following criteria:\n\n1. Age:\n\n   * 18 to 80 years, inclusive, at the time of informed consent.\n2. Diagnosis of ALS:\n\n   * Diagnosis of amyotrophic lateral sclerosis according to revised El Escorial criteria or equivalent, confirmed by a qualified neurologist.\n3. GREM2-Positive Status:\n\n   * Evidence of elevated GREM2 at screening, defined as:\n   * CSF GREM2 above a pre-specified threshold OR\n   * Plasma GREM2 above a pre-specified threshold with supportive evidence of astrocytic or TGF-β pathway activation (e.g., elevated GFAP or TGF-β-responsive biomarker).\n   * Biomarker thresholds will be defined prospectively in the protocol and laboratory manual.\n4. Disease Duration:\n\n   * Time from first ALS-related symptom onset ≤ 24 months at screening.\n5. Functional Status:\n\n   * ALS Functional Rating Scale - Revised (ALSFRS-R) total score ≥ a protocol-defined minimum (e.g., ≥ 25) at screening, sufficient to allow detection of functional change.\n6. Respiratory Function:\n\n   * Slow vital capacity (SVC) or forced vital capacity (FVC) ≥ 50% of predicted at screening.\n7. Stable Background ALS Therapy:\n\n   * If receiving riluzole and\u002For edaravone, participants must be on a stable dose for ≥ 30 days prior to screening and willing to maintain the regimen throughout the study.\n8. Ability to Consent:\n\n   * Ability to understand and provide written informed consent personally or via a legally authorized representative, in accordance with local regulations.\n9. Contraception:\n\n   * Women of childbearing potential and men with partners of childbearing potential must agree to use effective contraception during the study and for a defined period after the last dose.\n\nExclusion Criteria\n\nParticipants will be excluded if any of the following apply:\n\n1. Non-ALS Motor Neuron Disease:\n\n   * Diagnosis of primary lateral sclerosis (PLS), progressive muscular atrophy (PMA), or other non-ALS motor neuron disorders.\n2. Advanced Respiratory Insufficiency:\n\n   * Requirement for invasive mechanical ventilation at screening or anticipated need within the immediate study period.\n3. Clinically Significant Hepatic Disease:\n\n   * Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \\> 2.5 × upper limit of normal (ULN) at screening.\n   * Known cirrhosis or active chronic liver disease.\n4. Clinically Significant Cardiac Disease:\n\n   * Uncontrolled arrhythmia, recent myocardial infarction, unstable angina, or clinically significant cardiac dysfunction that may increase risk with study participation.\n5. Active or Uncontrolled Infection:\n\n   * Active systemic infection requiring treatment at screening or known chronic infection that could interfere with immune or biomarker assessments.\n6. Immunocompromised State:\n\n   * History of organ transplantation, active malignancy requiring systemic therapy, or chronic immunosuppressive therapy (excluding stable low-dose corticosteroids, if allowed by protocol).\n7. Prior Exposure to TGF-β Pathway Inhibitors:\n\n   * Previous treatment with galunisertib or other direct TGF-β or ALK5 inhibitors within a protocol-defined washout period.\n8. Recent Investigational Therapy:\n\n   * Participation in another interventional clinical trial or receipt of an investigational drug within 30-60 days prior to screening (exact window defined in protocol).\n9. Concomitant Medications with High Interaction Risk:\n\n   * Use of strong CYP modulators or medications known to significantly interfere with galunisertib or nerandomilast metabolism, unless safely discontinued.\n10. Pregnancy or Breastfeeding:\n\n    * Pregnant or breastfeeding women.\n11. Other Medical Conditions:\n\n    * Any medical, neurological, or psychiatric condition that, in the investigator's judgment, could:\n    * Interfere with study participation or compliance,\n    * Confound interpretation of efficacy or biomarker outcomes,\n    * Increase risk to the participant.",{"count":230,"type":22},[89,513],"PHASE3","Amyotrophic lateral sclerosis (ALS) is a relentlessly progressive neurodegenerative disorder characterized by loss of upper and lower motor neurons, leading to muscle weakness, respiratory decline, and eventual mortality. A growing body of translational and clinical evidence implicates neuroinflammation, reactive astrocytosis, and maladaptive TGF-β signaling as central contributors to disease progression. Elevated levels of Gremlin-2 (GREM2) have been identified as a marker of dysregulated TGF-β-linked astrocytic activity and fibrotic gene programs in some ALS patients, and preclinical data suggest that attenuating these pathways may mitigate glial toxicity and improve neuronal survival.\n\nGalunisertib, a selective ATP-competitive TGF-β receptor type I (TGF-βR1\u002FALK5) inhibitor, has been developed to block SMAD2\u002F3 phosphorylation and TGF-β-mediated transcriptional programs. Meanwhile, nerandomilast, a selective PDE4B inhibitor, elevates intracellular cAMP in immune and glial cells, shifting pro-inflammatory signaling toward resolution and antagonizing secondary fibrotic and inflammatory cascades. Preclinical models show that PDE4 inhibition and TGF-β pathway blockade concurrently reduce maladaptive glial phenotypes and fibrotic mediators.\n\nThis study investigates the combination of galunisertib + nerandomilast in ALS patients with elevated GREM2, hypothesizing that dual targeting of TGF-β-mediated astrocytic reactivity and PDE4B-regulated inflammatory signaling will translate into slowing of disease progression and favorable pharmacodynamic effects on central biomarkers of neuroinflammation and neurodegeneration.",[29],"2026-01-05",{"date":518,"type":36},"2026-01-07",{"date":73,"type":22},{"date":219,"type":22},{"name":522,"class":126},"Gipfel Life Sciences GmbH",{"id":524,"slug":525,"hasResults":12,"nctId":526,"briefTitle":527,"officialTitle":527,"acronym":4,"eligibilityCriteria":528,"healthyVolunteers":12,"sex":17,"minAge":529,"maxAge":4,"enrollmentInfo":530,"targetDuration":4,"studyType":23,"phases":531,"briefSummary":532,"conditions":533,"keywords":4,"overallStatus":70,"whyStopped":4,"lastUpdateSubmitDate":541,"lastUpdatePostDateStruct":542,"startDateStruct":544,"completionDateStruct":546,"leadSponsor":548,"locationsCount":177},"100544013","the-effect-of-a-muscle-mimicking-fabric-type-shoulder-orthosis-on-functional-movements-of-the-upper-limb-in-patients-with-neuromuscular-disorder-100544013","NCT06363357","The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements of the Upper Limb in Patients With Neuromuscular Disorder","Inclusion Criteria\n\n1. Patients with a confirmed diagnosis of a neuromuscular disease (NMD) by genetic testing, muscle biopsy, or electrodiagnostic studies, presenting with prominent upper limb muscle weakness. Examples include:\n\n   1. Muscular Dystrophies: Duchenne\u002FBecker Muscular Dystrophy (DMD\u002FBMD), Limb-Girdle Muscular Dystrophy (LGMD), Facioscapulohumeral Muscular Dystrophy (FSHD), etc.\n   2. Motor Neuron Diseases: Spinal Muscular Atrophy (SMA, Types 2 and 3), Amyotrophic Lateral Sclerosis (ALS, upper limb-dominant), etc.\n   3. Peripheral Neuropathies: Charcot-Marie-Tooth (CMT) disease, etc.\n   4. Other Neuromuscular Conditions: Including but not limited to cervical spinal cord injury.\n2. Aged over 10 years.\n3. A score of 2 to 5 on the Brooke Upper Extremity Functional Rating Scale.\n4. Manual Muscle Test (MMT) grade of less than 3 for shoulder abduction.\n5. Ability to provide written informed consent from the participant and\u002For their legal representative, indicating willingness to participate in the study.\n\nExclusion Criteria\n\n1. Unwillingness or inability to provide informed consent.\n2. A score of 1 or 6 on the Brooke Upper Extremity Functional Rating Scale.\n3. Cognitive impairment severe enough to interfere with the proper use of a shoulder orthosis.\n4. Any other condition which, in the opinion of the investigator, would make study participation inappropriate or unsafe for the patient.","10 Years",{"count":5,"type":22},[25],"The goal of this clinical trial is to investigate the effect of a muscle-mimicking, fabric-type shoulder orthosis on functional movements of the upper limb in patients with neuromuscular disorder.\n\nThe main questions it aims to answer are:\n\n* What is the impact of the muscle-mimicking, fabric-type shoulder orthosis on upper limb functional movements in patients with neuromuscular disorder?\n* Are there observable differences in upper limb function when the shoulder orthosis is worn versus when it is not?\n\nParticipants will:\n\n* Receive education on how to wear and use the shoulder orthosis.\n* Undergo evaluations, including assessment of upper limb performance, shoulder muscle strength testing, active range of motion measurements, assessment of functional workspace, goal attainment scale evaluation, surface electromyography, physiological measurements such as blood pressure and heart rate, fatigue assessment, and assessment for any musculoskeletal or skin-related issues.\n\nResearchers will compare neuromuscular disorder patients before and while wearing and operating the shoulder orthosis to see if there are any significant effects on variables such as upper limb function, range of motion, functional workspace, goal attainment scale, and surface electromyography.",[534,535,536,263,537,538,29,539,540],"Muscular Dystrophy, Duchenne","Orthotic Devices","Upper Extremity","Fascioscapulohumeral Muscular Dystrophy","Spinal Muscular Atrophy (SMA)","LGMD","SCI - Spinal Cord Injury","2025-11-25",{"date":543,"type":36},"2025-12-03",{"date":545,"type":36},"2024-04-20",{"date":547,"type":22},"2025-12-31",{"name":549,"class":43},"Seoul National University Hospital",{"id":551,"slug":552,"hasResults":12,"nctId":553,"briefTitle":554,"officialTitle":554,"acronym":555,"eligibilityCriteria":556,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":557,"targetDuration":4,"studyType":260,"phases":4,"briefSummary":559,"conditions":560,"keywords":563,"overallStatus":70,"whyStopped":4,"lastUpdateSubmitDate":569,"lastUpdatePostDateStruct":570,"startDateStruct":571,"completionDateStruct":573,"leadSponsor":575,"locationsCount":177},"100611017","omics-sciences-for-the-identification-of-pathogenetic-mechanisms-and-biomarkers-in-neurodegenerative-diseases-100611017","NCT07235111","Omics Sciences for the Identification of Pathogenetic Mechanisms and Biomarkers in Neurodegenerative Diseases","NeurOmics","Inclusion Criteria\n\n• Patients suffering from neurodegenerative diseases\n\nExclusion Criteria\n\n• Patients not suffering from neurodegenerative diseases",{"count":558,"type":22},1200,"The study aims to use 'omics' sciences, employing the most advanced technologies currently available, in order to identify pathogenic genomic variants, proteins and\u002For altered molecular pathways in neurodegenerative diseases and to obtain a new and more complete characterisation of subjects affected by the neurodegenerative diseases under study. Thanks to the integration of genomic, gene expression (transcriptomic and epigenomic), protein and metabolic data and clinical data, the study also aims to identify new markers for the diagnosis, prognosis, also in terms of response to therapy, and monitoring of neurodegenerative diseases.\n\nThe study involves the enrolment of at least 1.200 individuals with neurodegenerative disease.",[294,63,561,562,295,29],"Young-onset Dementia","MCI",[564,565,566,567,93,568],"LEWY BODIES DISEASE","young-onset dementia","alzheimer","parkinson","mci","2025-11-20",{"date":541,"type":36},{"date":572,"type":36},"2025-02-28",{"date":574,"type":22},"2039-09-17",{"name":576,"class":43},"Ospedale Policlinico San Martino",{"id":578,"slug":579,"hasResults":12,"nctId":580,"briefTitle":581,"officialTitle":582,"acronym":4,"eligibilityCriteria":583,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":584,"targetDuration":4,"studyType":23,"phases":585,"briefSummary":586,"conditions":587,"keywords":590,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":598,"lastUpdatePostDateStruct":599,"startDateStruct":601,"completionDateStruct":603,"leadSponsor":605,"locationsCount":177},"100609083","augmented-reality-bci-longitudinal-study-for-persons-with-als-stroke-tbi-and-sci-utilizing-cognixion--apple-vision-pro-100609083","NCT07209943","Augmented Reality BCI Longitudinal Study for Persons With ALS, Stroke, TBI and SCI Utilizing Cognixion + Apple Vision Pro","Cognixion Pass Through Study","Inclusion criteria:\n\n* Must have a designated on-site support individual who can be trained on the Cognixion system\n* Fluent in understanding English\n* 18 years or older\n* Must have one of ALS, spinal cord injury or chronic brain injury and need an assistive communication device\n* Must be able to engage in volitional eye opening and sustain eye opening independently for at least 30 minutes.\n* Must have a way to communicate apart from using the Cognixion device such as vocalizations, head nod, eye blinks, eyebrow raises, etc. At a minimum, reliable, independent way of communicating \"Yes\" and \"No\"\n\nExclusion criteria:\n\n* Disruption in English comprehension, either due to lack of fluent proficiency or due to a developmental\u002Facquired language disorder (e.g. aphasia)\n* Severely hearing impaired or deaf\n* Sensitivity to flashing lights\n* Claustrophobia related to the Apple Vision Pro with comfort adapter\n* History of epilepsy and\u002For seizures\n* Vision disorders restricting the visual field such as glaucoma, diplopia (double vision), nystagmus (involuntary eye movements)\n* History of vertigo or other vestibular disorders\n* Scalp that is prone to irritation, inflammation, injury, or infectious process",{"count":136,"type":22},[25],"The goal of this study is refine the usability of a BCI capable communication platform.\n\nThe study will take place in the United States area and will enroll up to 10 participants with late stage ALS, traumatic brain injury (TBI) or spinal cord injury (SCI) that have assistive communication and computer control needs. Each subject will receive an integrated Cognixion + Apple Vision Pro device that includes an augmented reality brain computer interface and associated communication software. The study duration is 3-4 months for each participant.\n\nThe key questions that will be addressed in this study are:\n\n1. Identify the ability of individuals with target indications to use the integrated Cognixion-Apple Vision Pro system to communicate effectively.\n2. Identify the ability of such individuals to learn to use BCI, ET-BCI and other modalities, and to measure their progress over time.\n3. Identify the effectiveness of the different forms of input supported by the combined Cognixion-Apple Vision Pro system (BCI, eye-tracking) in allowing such individuals to communicate and have agency.\n4. Identify how input such as BCI can be optimized to suit the needs of individuals (e.g., specific frequencies that work best for an individual, SNR with different frequencies, number of targets, length of recording for each frequency) and improve overall usability.\n5. Identify the extent to which personalization through a large language model (LLM) affects communication.\n6. Identify the appropriate capabilities to enable through an agentic communication interface.\n\nKey measures include:\n\nITR - information transfer rate SUS - system usability scale",[29,588,540,589],"TBI Traumatic Brain Injury","Stroke",[591,592,593,93,594,595,596,597],"usability study","augmented reality","brain computer interface","generative AI","agentic","spinal cord injury","traumatic brain injury","2025-09-29",{"date":600,"type":36},"2025-10-07",{"date":602,"type":22},"2025-10-16",{"date":604,"type":22},"2026-05-31",{"name":606,"class":126},"Cognixion",{"id":608,"slug":609,"hasResults":12,"nctId":610,"briefTitle":611,"officialTitle":611,"acronym":4,"eligibilityCriteria":612,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":428,"enrollmentInfo":613,"targetDuration":4,"studyType":23,"phases":615,"briefSummary":616,"conditions":617,"keywords":618,"overallStatus":70,"whyStopped":4,"lastUpdateSubmitDate":622,"lastUpdatePostDateStruct":623,"startDateStruct":625,"completionDateStruct":627,"leadSponsor":629,"locationsCount":177},"100598110","non-invasive-brain-stimulation-and-exercise-intervention-for-patients-with-motor-neuron-disease-100598110","NCT07067229","Non-invasive Brain Stimulation and Exercise Intervention for Patients With Motor Neuron Disease","Inclusion Criteria:\n\n* Participants aged between 18 and 80 years\n* Diagnosed with any type of motor neuron disease (MND)\n* Have mild to moderate severity, as assessed by the Sinaki-Mulder scale, with a severity level between 1 and 3\n\nExclusion Criteria:\n\n* History of other neurological disorders, such as stroke\n* Use of ventilatory support\n* Severe dementia",{"count":614,"type":22},100,[25],"Motor neuron disease (MND) is a progressive neurological disorder involving degeneration of motor neurons, leading to muscle weakness, speech and swallowing difficulties, and respiratory failure. This study aims to develop a novel treatment approach combining personalized repetitive transcranial magnetic stimulation (rTMS) with mixed reality (MR) exercise-based games (exergames) to slow disease progression and improve quality of life. In this randomised controlled trial study will compare three groups: (1) rTMS with MR exercise (personalized intervention), (2) rTMS with MR exercise (standard intervention), and (3) sham rTMS with MR exercise. Outcomes will be assessed at baseline, 3 months, and 6 months post intervention. The long-term goal is to implement this approach in clinical settings to enhance care for people with MND.",[29],[166,619,620,621],"Transcranial Magnetic Stimulation","Intervention","Augmented Reality","2025-09-21",{"date":624,"type":36},"2025-09-25",{"date":626,"type":36},"2025-08-01",{"date":628,"type":22},"2027-12-30",{"name":630,"class":43},"Chulalongkorn University",{"id":632,"slug":633,"hasResults":12,"nctId":634,"briefTitle":635,"officialTitle":636,"acronym":637,"eligibilityCriteria":638,"healthyVolunteers":12,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":639,"targetDuration":529,"studyType":260,"phases":4,"briefSummary":640,"conditions":641,"keywords":642,"overallStatus":70,"whyStopped":4,"lastUpdateSubmitDate":645,"lastUpdatePostDateStruct":646,"startDateStruct":648,"completionDateStruct":650,"leadSponsor":652,"locationsCount":177},"100606467","amyotrophic-lateral-sclerosis-registry-in-thailand-100606467","NCT07175935","Amyotrophic Lateral Sclerosis Registry in Thailand","A Prospective, Multicenter Registry Study of Amyotrophic Lateral Sclerosis in Thailand","Thai ALS Regis","Inclusion Criteria:\n\n* Diagnosis of ALS according to El Escorial or Gold Coast criteria\n* Age ≥ 18 years\n* Ability and willingness to provide informed consent\n\nExclusion Criteria:\n\n* Patients unwilling to provide informed consent\n* Patients with alternative diagnoses mimicking ALS",{"count":614,"type":22},"This is a prospective, observational, multicenter registry designed to collect comprehensive clinical, genetic, and outcome data from patients diagnosed with amyotrophic lateral sclerosis (ALS) across Thailand. The registry will establish a national dataset to describe epidemiology, clinical presentation, progression, and treatment outcomes, and will serve as a platform for future clinical and translational research.",[29],[166,643,644],"Epidemiological","Natural History","2025-09-14",{"date":647,"type":36},"2025-09-16",{"date":649,"type":36},"2025-03-01",{"date":651,"type":22},"2030-12-31",{"name":630,"class":43},{"id":654,"slug":655,"hasResults":12,"nctId":656,"briefTitle":657,"officialTitle":658,"acronym":4,"eligibilityCriteria":659,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":660,"targetDuration":4,"studyType":23,"phases":662,"briefSummary":663,"conditions":664,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":665,"lastUpdatePostDateStruct":666,"startDateStruct":668,"completionDateStruct":669,"leadSponsor":671,"locationsCount":4},"100599261","phase-2-a-study-to-evaluate-the-efficacy-and-safety-of-different-doses-of-cb03-154-in-adult-patients-with-amyotrophic-lateral-sclerosis-als-100599261","NCT07082192","A Study to Evaluate the Efficacy and Safety of Different Doses of CB03-154 in Adult Patients With Amyotrophic Lateral Sclerosis (ALS)","A Multicenter, Randomized, Double-blind, Placebo-controlled Phase II\u002FIII Adaptive Clinical Study and Open-label Extension Study to Evaluate the Efficacy and Safety of Different Doses of CB03-154 in Adult Patients With Amyotrophic Lateral Sclerosis (ALS)","Inclusion Criteria:\n\n1. Agree to follow the treatment plan and trial procedures of this study, and sign the written informed consent form.\n2. Male or female, aged 18 to 65 years, inclusive.\n3. The weight of subjects during the screening period must not be less than 45 kg, and the BMI must not be less than 18 kg\u002Fm2.\n4. Diagnosed according to the Revised EI Escorial diagnostic criteria set by the World Federation of Neurology: definite ALS, probable ALS, lab supported probable ALS, or possible ALS.\n5. Less than or equal to 24 months since ALS symptom onset at Screening, and estimated survival time of ≥1 year as per the Investigator's judgement.\n6. Forced vital capacity (FVC) \\>80% of predicted value for gender, height, and age at Screening.\n7. Able to swallow oral medication (tablets) at Screening as judged by the investigator.\n8. For participants taking riluzole: Dose must be stable for at least 4 weeks prior to Screening and participant must be expected to remain on treatment for the duration of the trial. Participants receiving riluzole should maintain the same dose throughout the study.\n9. Currently not receiving edaravone treatment or is in the schedule of edaravone treatment cycle. Participants receiving edaravone treatment must complete at least one cycle of treatment before the screening visit and continue stable dose edaravone treatment throughout the study.\n10. Women of childbearing potential (WOCBP) must use an approved highly effective contraception for at least one menstrual cycle before the first dose of the investigational product and for at least 3 months after the last dose of the investigational product. Similarly, men must start using effective contraception before the first dose of the investigational product and continue for at least 3 months after the last dose of the investigational product, with no plans for procreation. Male participants cannot donate sperm for at least 3 months during the trial and after the last dose of the investigational product and female participants cannot donate or freeze eggs during the trial and for at least 3 months after the last dose of the investigational product.\n\nExclusion Criteria:\n\n1. Significant cognitive impairment, mental disorders (such as schizophrenia, bipolar disorder), other neurodegenerative diseases (such as Parkinson's disease, Alzheimer's disease, frontotemporal dementia, etc.), substance abuse or other causes leading to neuromuscular weakness (such as myasthenia gravis), or other conditions that may interfere with the participants' participation in clinical study or, in the investigator's judgment, may interfere with outcome assessment or affect the completion of the trial.\n2. Serum aspartate aminotransferase (AST), alanine aminotransferase (ALT), or bilirubin\\>2.0× upper limit of normal at screening.\n3. Estimated glomerular filtration rate \\\u003C59 mL\u002Fmin\u002F1.73m2 at Screening (using the Cockcroft-Gault formula to calculate eGFR: eGFR (mL\u002Fmin\u002F1.73m2) = Ccr × 0.84 × 1.73 \u002F BSA; Ccr (mL\u002Fmin) = \\[(140 - age) × weight (kg)\\] \u002F \\[72 × Scr (mg\u002FdL)\\], females multiply the result by 0.85, and Scr is the serum creatinine; BSA (m2) = 0.007184 × weight (kg)\\^0.425 × height (cm)\\^0.725).\n4. D-dimer\\>2.0× upper limit of normal or venous ultrasound of the lower limbs shows deep vein thrombosis at screening or a history of venous thrombosis.\n5. Assistance with ventilation support or tracheostomy or tube feeding status or having a central venous catheter is required at screening.\n6. A history of unexplained syncope, family history of syncope, or a history of convulsions or epilepsy (excluding the history of febrile seizures in childhood), or unstable medical condition, serious heart issues (e.g., corrected QTcF interval: males \\>450ms, females \\>470ms, torsades de pointes, NYHA class 3 or higher heart failure, myocardial infarction or unstable angina within 6 months prior to screening), lung, liver, kidney diseases, or tumors, or other clinically significant diseases or medical history (excluding ALS), participation in this study could threaten the safety of the participants.\n7. Current clinically significant urinary retention, or current use of medications for urinary retention, or clinically significant abnormalities in residual urinary bladder ultrasound at screening.\n8. Clinically significant ophthalmological abnormalities found in visual acuity examination (best corrected visual acuity), fundus photography, OCT examination, etc. during screening period, or clinically significant fundus lesions or retinopathy known or recorded in medical history.\n9. Hepatitis B surface antigen (HBsAg) positive, or hepatitis C antibody (HCVAb) positive and hepatitis C virus ribonucleic acid (HCV-RNA) test higher than the lower limit of detection, or human immunodeficiency virus antibody (HIVAb) positive, or Treponema pallidum (TP) antibody positive (also judged as active infection by the investigator) at screening.\n10. Severe infections (e.g., infectious pneumonia, sepsis) within 4 weeks prior to screening, or infections requiring hospitalization or intravenous administration of antibiotics, antiviral drugs, or antifungal medications, or chronic active bacterial infections (e.g., tuberculosis) deemed by the investigator to be unsuitable for participation in this trial.\n11. Received Tofersen treatment before screening.\n12. Significant risk of suicidality based on the Investigator's opinion or with an answer of \"yes\" on either item 4 or item 5 of the Suicidal Ideation Section of the Columbia Suicide Severity Rating Scale (C-SSRS) or any answer of \"yes\" within the Suicidal Behavior Section of the C SSRS within the 6 months before Screening.\n13. Exposure to any other investigational medicinal product or product within 4 weeks or 5 half-lives of the investigational product (whichever is longer) prior to Screening; or exposure to monoclonal antibody drug within 6 months prior to Screening (or if the washout period at the time of screening has not reached more than 3 months), or had received cell therapy or gene therapy at any time in the past.\n14. Treatment with CYP3A4 strong inducers or strong inhibitors prior to screening, and washout time of more than 5 half-lives of the drug was not reached before enrollment。\n15. Pregnant, currently breastfeeding women or women with a positive pregnancy test at screening.\n16. Known history of allergy to any component of the investigational medicinal product.\n17. History of drug abuse within 12 months of Screening.\n18. Anything else that, in the opinion of the Investigator, would place the participant at increased risk or preclude the participant's full compliance with or completion of the trial.",{"count":661,"type":22},240,[89,513],"The goal of this clinical trial is to learn if drug CB03-154 works to treat ALS in adults. It will also learn about the safety of drug CB03-154.\n\nThe main questions it aims to answer are:\n\n* Does drug CB03-154 have an effect on delaying disease progression, improving function, and prolonging survival in adult ALS patients?\n* What medical problems do patients have when taking drug CB03-154? Researchers will compare drug CB03-154 to a placebo (a look-alike substance that contains no drug) to see if drug CB03-154 works to treat ALS.\n\nParticipants (adult ALS patients) will:\n\n* Take drug CB03-154 or a placebo every day for 39 weeks (an additional 39 weeks would be required if entering the open-label extension phase).\n* Visit the clinic approximately every 2-3 months for checkups and tests, and there is also telephone follow-up in between.\n* Keep a diary of daily medication (CB03-154 or other concomitant medications), and if there are any unplanned medications, the reason (disease or symptoms) also need be recorded.",[29],"2025-07-23",{"date":667,"type":36},"2025-07-24",{"date":598,"type":22},{"date":670,"type":22},"2027-10",{"name":672,"class":126},"Shanghai Zhimeng Biopharma, Inc.",{"id":674,"slug":675,"hasResults":12,"nctId":676,"briefTitle":677,"officialTitle":677,"acronym":4,"eligibilityCriteria":678,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":358,"enrollmentInfo":679,"targetDuration":4,"studyType":23,"phases":680,"briefSummary":682,"conditions":683,"keywords":684,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":687,"lastUpdatePostDateStruct":688,"startDateStruct":690,"completionDateStruct":692,"leadSponsor":694,"locationsCount":4},"100598913","early-phase-1-extended-study-of-rag-17-in-the-treatment-of-amyotrophic-lateral-sclerosis-patients-with-sod1-gene-mutation-100598913","NCT07077668","Extended Study of RAG-17 in the Treatment of Amyotrophic Lateral Sclerosis Patients With SOD1 Gene Mutation","Inclusion Criteria:\n\n* 18 ≤ Age ≤ 75 years old, regardless of gender.\n* ALS patients with confirmed SOD1 gene mutations (known SOD1 mutation sites with reported relevant disease progression).\n* The diagnosis meets the criteria for definite or probable familial or sporadic ALS in the El Escorial diagnostic criteria for amyotrophic lateral sclerosis revised by the World Federation of Neurology.\n* The patient himself\u002Fherself or their legal representative clearly understands, voluntarily participates in this study, and signs the informed consent form.\n\nExclusion Criteria:\n\n* Patients with SOD1 mutation sites occurring at nucleotides 44 - 66 (counting from the start of SOD1 protein translation) and patients with P.F21C mutation.\n* Patients diagnosed with other mental illnesses according to the DSM - V diagnostic criteria, or those with obvious suicidal intent.\n* Patients with severe hepatic insufficiency, severe renal insufficiency, or severe cardiac insufficiency. (Severe hepatic insufficiency refers to an ALT value ≥ 2.0 times the upper limit of normal or an AST value ≥ 2.0 times the upper limit of normal; severe renal insufficiency refers to a CRE ≥ 1.5 times the upper limit of normal or an eGFR \\\u003C 40 mL\u002Fmin\u002F1.73m²; severe cardiac insufficiency refers to a NYHA score of 3 - 4.)\n* Patients with a history of alcohol or drug abuse.\n* Pregnant, lactating patients, those with a possibility of pregnancy, or patients planning to become pregnant.\n* Patients who have received any vaccination within 28 days.\n* Patients who are unable to cooperate with the follow - up for other reasons.",{"count":136,"type":22},[681],"EARLY_PHASE1","This study primarily evaluates the safety, tolerability, and efficacy of RAG - 17 in adult ALS patients with SOD1 - mutated genes in the real - world setting.",[29],[333,685,686],"RAG-17","SOD1","2025-07-13",{"date":689,"type":36},"2025-07-22",{"date":691,"type":22},"2025-07",{"date":693,"type":22},"2026-12-31",{"name":695,"class":43},"Beijing Tiantan Hospital"]