[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"neurodegenerative-disease-hereditary\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:neurodegenerative-disease-hereditary":30},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,77,106],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":47,"overallStatus":64,"whyStopped":4,"lastUpdateSubmitDate":65,"lastUpdatePostDateStruct":66,"startDateStruct":69,"completionDateStruct":71,"leadSponsor":73,"locationsCount":76},"100567749","phase-3-a-phase-3-study-of-ntla-2001-in-attrv-pn-100567749",false,"NCT06672237","A Phase 3 Study of NTLA-2001 in ATTRv-PN","MAGNITUDE-2: A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2001 in Participants With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN)","Inclusion Criteria:\n\n* Diagnosis of ATTRv-PN\n* Karnofsky Performance Status (KPS) ≥ 60\n\nExclusion Criteria:\n\n* Other causes of amyloidosis (amyloidosis caused by non-TTR protein)\n* Other known causes of sensorimotor or autonomic neuropathy\n* Diabetes mellitus\n* New York Heart Association Class III or IV heart failure\n* Liver failure\n* Hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection\n* Prior receipt of a TTR silencer (Small interfering RNA (siRNA) or Antisense oligonucleotides (ASOs))\n* Estimated Glomerular Filtration Rate \\\u003C 30 mL\u002Fmin\u002F1.73 m2\n* Unable or unwilling to take vitamin A supplementation for the duration of the study\n* History of liver disease","ALL","18 Years","85 Years",{"count":20,"type":21},60,"ESTIMATED","INTERVENTIONAL",[24],"PHASE3","This study will be conducted to evaluate the efficacy and safety of a single dose of nexiguran ziclumeran (NTLA-2001) compared to placebo in participants with ATTRv-PN.",[27,28,29,30,31,32,33,34,35,36,37,38,39,40,41,42,43,44,45,46],"Neuromuscular Disease","Neuromuscular Diseases (NMD)","Neurodegenerative Disease","Neurodegenerative Disease, Hereditary","Neurodegenerative Diseases","Neuromuscular Diseases","Nerve Disorders","Nervous System Disease","Nervous System Diseases","Genetic Disease, Inborn","Amyloidosis, Familial","Amyloidosis, Hereditary","Amyloidosis","Polyneuropathies","Amyloid Neuropathies","Amyloid Neuropathies, Familial","Peripheral Nervous System Disease","Peripheral Nervous System Diseases","Metabolism, Inborn Errors","Metabolic Diseases",[48,39,49,50,51,52,53,54,55,56,57,58,59,60,61,62,63],"TTR","Polyneuropathy","NTLA-2001","ATTR","ATTR-PN","ATTRv-PN","Transthyretin","TTR-mediated amyloidosis","Amyloidosis, hereditary","Amyloidosis, hereditary, transthyretin-related amyloidosis","Transthretin amyloid polyneuropathy","TTR PN","TTR polyneuropathy","nexiguran ziclumeran","nex-z","CRISPR","RECRUITING","2026-04-13",{"date":67,"type":68},"2026-04-16","ACTUAL",{"date":70,"type":68},"2024-11-22",{"date":72,"type":21},"2028-08",{"name":74,"class":75},"Intellia Therapeutics","INDUSTRY",14,{"id":78,"slug":79,"hasResults":11,"nctId":80,"briefTitle":81,"officialTitle":82,"acronym":83,"eligibilityCriteria":84,"healthyVolunteers":11,"sex":16,"minAge":85,"maxAge":86,"enrollmentInfo":87,"targetDuration":4,"studyType":89,"phases":4,"briefSummary":90,"conditions":91,"keywords":4,"overallStatus":64,"whyStopped":4,"lastUpdateSubmitDate":95,"lastUpdatePostDateStruct":96,"startDateStruct":98,"completionDateStruct":100,"leadSponsor":102,"locationsCount":105},"100479102","molecular-and-functional-imaging-in-monogenic-pd-100479102","NCT05518617","Molecular and Functional Imaging in Monogenic PD.","Molecular and Functional Imaging of Parkinson's Pathology in SNCA, Parkin and PINK1 Mutation Carriers","FOX_1","Inclusion Criteria:\n\n* All subjects must be judged by the investigator able to understand the nature, design, and procedures of the study and must be able to provide a signed and dated informed consent in accordance with Good Clinical Practice (GCP), International Conference on Harmonization (ICH), and local regulations.\n* All subjects must be willing and able to comply with scheduled visits, required study procedures and laboratory tests.\n* All subjects must be able to travel to the research sites for the study procedures.\n* For female subjects: They must be either of non-childbearing potential (either surgically sterile or post- menopausal - defined as 12 months of spontaneous amenorrhea), or, if of childbearing potential, subjects must demonstrate to be non-pregnant (as demonstrated by negative urine β-HCG test at screening), non-breastfeeding.\n* All subjects must comply with highly effective contraceptive measures. A highly effective contraceptive measure is defined as a measure that can achieve a failure rate of less than 1% per year when used consistently and correctly. These methods are listed in more detail below:\n\nOral, intravaginal, or transdermal combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation;\n\nOral, injectable, or implantable progestogen-only hormonal contraception associated with inhibition of ovulation:\n\nIntrauterine device (IUD)\n\nIntrauterine hormone-releasing system (IUS)\n\nBilateral tubal occlusion\n\nVasectomised partner\n\nSexual abstinence\n\n* For sexually active male subjects, they must agree to use condoms to protect their partners from becoming pregnant for the duration of the study and for 3 months after the last administration of PET or SPECT ligands. They must also agree to ensure that they and their partners are routinely using a medically approved contraceptive method. It is important that male subjects not impregnate others for the duration of the study and for 3 months after the last administration of PET or SPECT ligands.\n\n  \\*\\*All subjects must have adequate visual and auditory acuity according to investigator's judgement to complete the psychological testing.\n* All subjects must have no use of medications with known interaction with serotonergic transmission (e.g. selective serotonin reuptake inhibitors, tricyclic antidepressant, triptans, etc).\n* For subjects taking any drugs that might interfere with dopamine transporter SPECT imaging (neuroleptics, metoclopramide, alpha methyldopa, methylphenidate, reserpine, or amphetamine derivative) must be willing and able from a medical standpoint to hold the medication for at least 5 half-lives prior to screening DaTSCANä imaging.\n\nExclusion Criteria:\n\n* Subjects lacking capacity according to investigator judgement.\n* Subjects with a clinical diagnosis of dementia as determined by the investigator.\n* Current treatment with anticoagulants (e.g. warfarin, heparin) that might preclude safe completion of the lumbar puncture.\n* Condition that precludes the safe performance of routine lumbar puncture, such as prohibitive lumbar spinal disease, bleeding diathesis, or clinically significant coagulopathy or thrombocytopenia.\n* Use of any of the following drugs that might interfere with dopamine transporter SPECT imaging: neuroleptics, metoclopramide, alpha methyldopa, methylphenidate, reserpine, or amphetamine derivative, within 5 months of Screening.\n* Use of investigational drugs or devices within 60 days prior to Baseline (dietary supplements taken outside of a clinical trial are not exclusionary, e.g., coenzyme Q10).\n* History of cancer within the last 5 years, with the exception of non-metastatic basal cell carcinoma of the skin.\n* Subjects with current or recent history of drug or alcohol abuse\u002Fdependence.\n* Contraindication to MRI, such as presence of metal devises or implants (e.g. pacemaker, vascular- or heart- valves, stents, clips), metal deposited in the body (e.g. bullets or shells), or metal grains in the eyes;\n* Claustrophobia or history of back pain that makes prolonged laying on the PET or MRI scanner intolerable.\n* Previously obtained MRI scan with evidence of clinically significant neurological disorder (in the opinion of the Investigator).\n* Any other medical or psychiatric condition or lab abnormality, which in the opinion of the investigator might preclude participation.","25 Years","80 Years",{"count":88,"type":21},45,"OBSERVATIONAL","In this study, the investigators aim to find a biomarker of Parkinson's disease. This is done using imaging scans called Positron Emission tomography (PET), Single Photon Emission Computed Tomography (SPECT), and Magnetic Resonance Imaging (MRI). The findings will provide a deeper understanding of the brain changes in Parkinson's disease. More importantly, this study will help with the discovery and development of new medications aiming to delay progression of PD symptoms.",[92,93,31,30,94],"Parkinson Disease","Nervous System Disorder","Parkinson's","2025-10-01",{"date":97,"type":68},"2025-10-07",{"date":99,"type":68},"2022-07-01",{"date":101,"type":21},"2026-06-30",{"name":103,"class":104},"University of Exeter","OTHER",1,{"id":107,"slug":108,"hasResults":11,"nctId":109,"briefTitle":110,"officialTitle":111,"acronym":4,"eligibilityCriteria":112,"healthyVolunteers":113,"sex":16,"minAge":114,"maxAge":115,"enrollmentInfo":116,"targetDuration":4,"studyType":22,"phases":117,"briefSummary":119,"conditions":120,"keywords":125,"overallStatus":133,"whyStopped":4,"lastUpdateSubmitDate":134,"lastUpdatePostDateStruct":135,"startDateStruct":137,"completionDateStruct":139,"leadSponsor":141,"locationsCount":4},"100529688","phase-2-mclena-2-a-phase-ii-clinical-trial-for-the-assessment-of-lenalidomide-in-patients-with-mild-cognitive-impairment-100529688","NCT06177028","MCLENA-2: A Phase II Clinical Trial for the Assessment of Lenalidomide in Patients With Mild Cognitive Impairment","MCLENA-2: A Phase II Clinical Trial for the Assessment of Biomarker Trajectory in Patients With Mild Cognitive Impairment Due to Alzheimer's Disease Treated With Lenalidomide (Amendment to IND # 142121)","In order to be eligible for this study, subjects must meet the following inclusion criteria:\n\nInclusion Criteria:\n\n1. Male or female outpatients.\n2. At least 50 years of age, but less than 90 (89 at time of screening)\n3. Females must be surgically sterile (bilateral tubal ligation, oophorectomy, or hysterectomy) or postmenopausal for 2 years (no women at risk of pregnancy will be accepted in this study).\n4. Must have been diagnosed with amnestic MCI based on the most recent NIA-AA criteria (Albert et al., 2011), i.e. at both the screening and baseline visits (visits 1 and 2) have a documented Mini Mental State Exam (MMSE) score between 22-28.\n5. CT or MRI scan of the brain obtained during the course of the dementia must be consistent with the diagnosis and show no evidence of significant focal lesions or of pathology which could contribute to dementia. If neither a CT nor an MRI scan is available from the past 12 months, a CT scan fulfilling the requirements must be obtained before randomization.\n6. Vision and hearing must be sufficient to comply with study procedures.\n7. Be able to take oral medications.\n8. Hachinski ischemic score must be ≤ 4.\n9. Geriatric depression scale must be ≤ 10.\n10. Can be on stable doses of a cholinesterase inhibitor and\u002For memantine as long as it is stable for at least 90 days before screening and is expected to remain on a stable dose for the remainder of the study period; or have demonstrated intolerance to or lack of efficacy from these medications.\n11. Must have a collateral informant\u002Fstudy partner who has significant direct contact with the patient at least 10 hours per week and who is willing to accompany the patient to specified clinic visits, supervise administration of all study medication, and be available for telephone visits\u002Finterviews.\n12. If the patient has a legally authorized representative (LAR), the LAR must review and sign the informed consent form. If the patient does not have an LAR, the patient must appear able to provide informed consent and must review and sign the informed consent form. In addition, the patient's informant\u002Fstudy partner (as defined above) must sign an informed consent form. If the LAR and the patient's informant \u002Fstudy partner is the same individual, he\u002Fshe should sign under both designations.\n13. Must reside in the community.\n14. Patients with stable prostate cancer may be included at the discretion of the Medical Monitor.\n15. Positivity for amyloid brain scan: Amyloid PET positive at SUVr of 1.05\n\nExclusion Criteria:\n\nSubjects will be excluded if they have any of the condition listed below:\n\n1. Current evidence or history within the last 3 years of a neurological or psychiatric illness that could contribute to dementia, including (but not limited to) epilepsy, focal brain lesion, Parkinson's disease, seizure disorder, head injury with loss of consciousness\n2. DSM IV criteria for any major psychiatric disorder including psychosis, major depression and bipolar disorder.\n3. Unwilling or unable to undergo a Lumbar Puncture.\n4. Known history or self-reported alcohol or substance abuse.\n5. Living alone.\n6. Poorly controlled hypertension. 7 .History of myocardial infarction or signs or symptoms of unstable coronary artery disease within the last year (including revascularization procedure\u002Fangioplasty).\n\n8\\. Severe pulmonary disease (including chronic obstructive pulmonary disease) requiring more than 2 hospitalizations within the past year.\n\n9\\. Untreated sleep apnea. 10. Any thyroid disease (unless euthyroid on treatment for at least 6 months prior to screening).\n\n11\\. Active neoplastic disease (except for skin tumors other than melanoma) within five years.\n\n12\\. History of multiple myeloma. 13. Absolute neutropenia of \\\u003C750\u002Fmm3, or a history of neutropenia. 14. History of or current thromboembolism (including deep venous thrombosis). 15. Any clinically significant hepatic or renal disease (including presence of Hepatitis B or C antigen\u002Fantibody or an elevated transaminase levels of greater than two times the upper limit of normal (ULN) or creatinine greater than 1.5 x ULN).\n\n16\\. Clinically significant hematologic or coagulation disorder including any unexplained anemia or a platelet count less than 100,000\u002FμL at screening.\n\n17\\. Use of any investigational drug within 30 days or within five half-lives of the investigational agent, whichever is longer.\n\n18\\. Use any investigational medical device within two weeks before screening or after end of the present study.\n\n19\\. Females who are at risk of pregnancy or are of child bearing age. 20. Unwilling or unable to undergo MRI and PET imaging. 21. Cardiac pacemaker or defibrillator or other implanted device. 22. In the opinion of the investigator, participation would not be in the best interest of the subject",true,"50 Years","90 Years",{"count":88,"type":21},[118],"PHASE2","This is a randomized, double-blind, placebo-controlled, parallel group study. The use of placebo is appropriate to minimize bias related to treatment expectations of the subject, study partner, and site investigator, as well as to changes in the relationship between the subject and study partner that might occur with the initiation of treatment and expectation of improvement in motor symptoms or cognition. Changes in subject\u002Fstudy partner interactions can impact subject mood and might introduce biases that cannot be quantified. The double-blind use of placebo will also prevent bias in the clinical and scientific assessments.",[121,122,123,30,124],"Cognitive Impairment, Mild","Cognitive Dysfunction","Amyloid Plaque","Inflammation, Brain",[126,127,128,129,130,131,132],"Alzheimer's disease","CSF","Biomarkers","Brain Imaging","Brain Amyloid","Memory Loss","Immunomodulation","NOT_YET_RECRUITING","2025-05-14",{"date":136,"type":68},"2025-05-18",{"date":138,"type":21},"2025-06-01",{"date":140,"type":21},"2027-01-02",{"name":142,"class":104},"St. Joseph's Hospital and Medical Center, Phoenix"]