[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"gene-therapy\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:gene-therapy":40},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,9,0,[8,59,90,124,148,174,207,227,271],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":41,"overallStatus":46,"whyStopped":4,"lastUpdateSubmitDate":47,"lastUpdatePostDateStruct":48,"startDateStruct":51,"completionDateStruct":53,"leadSponsor":55,"locationsCount":58},"100598722","phase-1-a-study-to-evaluate-a-novel-gene-therapy-in-patients-with-relapsed-and-refractory-multiple-myeloma-100598722",false,"NCT07075185","A Study to Evaluate a Novel Gene Therapy in Patients With Relapsed and Refractory Multiple Myeloma","A Phase 1 Study to Evaluate the Safety of KLN-1010, a Novel, In Vivo Gene Therapy to Generate Anti-B Cell Maturation Antigen (Anti-BCMA) Chimeric Antigen Receptor-T Cells (CAR-T) in Patients With Relapsed and Refractory Multiple Myeloma","inMMyCAR","Inclusion Criteria:\n\n* Participants must have relapsed and refractory multiple myeloma (RRMM) with measurable disease\n* Participants must have received at least 3 prior lines of therapy including a proteasome inhibitor (PI), an immunomodulatory drug (IMiD), and CD38-directed monoclonal antibody\n* Participants must have an Eastern Cooperative Group (ECOG) performance status of 0-1\n* Participants must have acceptable laboratory values as defined by the protocol\n\nExclusion Criteria:\n\n* Participants must not have known central nervous system (CNS) involvement with myeloma\n* Participants cannot have plasma cell leukemia, Waldenstrom's macroglobulinemia, POEMS (polyneuropathy, organomegaly, endocrinopathy, and skin changes) syndrome, or primary light chain amyloidosis\n* Participants cannot have ongoing acute systemic infection requiring antimicrobial therapy\n* Participants cannot require systemic steroids for any condition","ALL","18 Years",{"count":20,"type":21},70,"ESTIMATED","INTERVENTIONAL",[24],"PHASE1","The goal of this clinical trial is to evaluate the safety, tolerability, and recommended Phase 2 Dose (RP2D) of KLN-1010 in patients with relapsed or refractory multiple myeloma.",[27,28,29,30,31,32,33,34,35,36,37,38,39,40],"Multiple Myeloma in Relapse","Myeloma Multiple","Multiple Myeloma Progression","Neoplasms by Histologic Type","Neoplasm","Hemostatic Disorders","Vascular Disorder","Paraproteinemias","Blood Protein Disorders","Hematologic Disease and Disorders","Lymphoproliferative Disorders","Immunoproliferative Disorders","Immune System Disease","Gene Therapy",[42,43,44,45],"in vivo CAR-T","multiple myeloma","gene therapy","BMCA","RECRUITING","2026-04-22",{"date":49,"type":50},"2026-04-24","ACTUAL",{"date":52,"type":50},"2025-07-16",{"date":54,"type":21},"2042-05",{"name":56,"class":57},"Kelonia Therapeutics, Inc.","INDUSTRY",7,{"id":60,"slug":61,"hasResults":11,"nctId":62,"briefTitle":63,"officialTitle":63,"acronym":4,"eligibilityCriteria":64,"healthyVolunteers":11,"sex":65,"minAge":66,"maxAge":67,"enrollmentInfo":68,"targetDuration":4,"studyType":22,"phases":70,"briefSummary":72,"conditions":73,"keywords":75,"overallStatus":46,"whyStopped":4,"lastUpdateSubmitDate":79,"lastUpdatePostDateStruct":80,"startDateStruct":82,"completionDateStruct":84,"leadSponsor":86,"locationsCount":89},"100309680","phase-1-phase-iii-trial-of-lentiviral-gene-transfer-for-scid-x1-with-low-dose-targeted-busulfan-conditioning-100309680","NCT03311503","Phase I\u002FII Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning","Inclusion Criteria:\n\n\\- 1. Diagnosis of SCID-X1 based on immunophenotype and lack of T cell function (proliferation to PHA \\\u003C10% of the lower limit of normal for the laboratory) AND confirmed by a mutation in IL2RG 2. Lack of an HLA identical (A, B, C, DR, DQ) related donor 3. Age 5 years old or younger 4. Signed informed consent 5. Documentation of willingness to follow up for 15 years post-infusion as currently required by the FDA 6. If the patient has previously undergone allogeneic transplant, lack of donor T cell engraftment must be documented.\n\n7\\. Age at least 8 weeks by the time of busulfan administration\n\nExclusion Criteria:\n\n1. Patients with an active, therapy-resistant infection. Infections that are known to be highly morbid in SCID patients will be considered active and therapy-resistant if the infectious agent is repeatedly isolated despite a minimum of 2 weeks of appropriate therapy and is associated with significant organ dysfunction (including but not limited to abnormalities listed below).\n\n   1. Mechanical ventilation including continuous positive airway pressure\n   2. Abnormal liver function defined by AST and ALT \\>10 times the upper range of normal OR Bilirubin \\>2 mg\u002FdL\n   3. Shortening fraction on echocardiogram \\\u003C25% or ejection fraction \\\u003C50%\n   4. Renal failure defined as glomerular filtration rate \\\u003C30 ml\u002Fmin\u002F1.73 m2 or dialysis dependence\n2. Uncontrolled seizure disorder\n3. Encephalopathy\n4. Documented coexistence of any disorder known to affect DNA repair\n5. Diagnosis of active malignant disease other than EBV-associated lymphoproliferative disease\n6. Patients with evidence of infection with HIV-1\n7. Major (life-threatening) congenital anomalies. Examples of \"major (life-threatening) congenital anomalies\" include, but are not limited to: unrepaired cyanotic heart disease, hypoplastic lungs, anencephaly or other major central nervous system malformations, other severe non-repairable malformations of the gastrointestinal or genitourinary tracts that significantly impair organ function.\n8. Other conditions which in the opinion of the P.I. or co-investigators, contra-indicate collection and\u002For infusion of transduced cells or indicate patient's inability to follow the protocol. These may include for example clinical ineligibility to receive anesthesia, severe deterioriation of clinical condition of the patient after collection of bone marrow but before infusion of transduced cells, or documented refusal or inability of the family to return for scheduled visits. There may be other unforeseen rare circumstances that would result in exclusion of the patient, such as sudden loss of legal guardianship\n\n   \\-","MALE","0 Years","5 Years",{"count":69,"type":21},12,[24,71],"PHASE2","This is a phase I\u002FII open label multi-center study in which patients will receive low dose targeted busulfan followed by infusion of autologous CD34+ selected bone marrow or mobilized peripheral blood cells transduced with the G2SCID vector. Subjects will be enrolled over 3 years and be followed for 2 years post-infusion on this protocol, then followed long-term on a separate long-term follow-up protocol.\n\nEnrollment of subjects will be agreed upon by representatives of both sites. Data will be collected uniformly from both sites through an electronic capture system and key laboratory studies will be centralized.\n\nHarvest, cellular manufacturing and infusion will occur at each site using the same SOPs. Key aspects of cellular product characterization will be centralized",[74,40],"Severe Combined Immunodeficiency, X Linked",[76,77,78],"lentiviral","Gene therapy","busulfan","2025-12-04",{"date":81,"type":50},"2025-12-11",{"date":83,"type":50},"2018-02-26",{"date":85,"type":21},"2028-01-01",{"name":87,"class":88},"David Williams","OTHER",4,{"id":91,"slug":92,"hasResults":11,"nctId":93,"briefTitle":94,"officialTitle":95,"acronym":4,"eligibilityCriteria":96,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":97,"targetDuration":4,"studyType":99,"phases":4,"briefSummary":100,"conditions":101,"keywords":108,"overallStatus":46,"whyStopped":4,"lastUpdateSubmitDate":113,"lastUpdatePostDateStruct":114,"startDateStruct":116,"completionDateStruct":118,"leadSponsor":120,"locationsCount":123},"100145560","cibmtr-research-database-100145560","NCT01166009","CIBMTR Research Database","Protocol For A Research Database For Hematopoietic Stem Cell Transplantation, Other Cellular Therapies and Marrow Toxic Injuries","Eligibility to Participate in the Research Database\n\nRecipient Eligibility Criteria:\n\n* Any recipient of an unrelated or related donor or autologous HSC transplant in a CIBMTR center is eligible to participate in the Research Database. This includes adults with and without decision making capacity, and children.\n\nIndividual with Marrow Toxic Injury Eligibility Criteria:\n\n* Any individual who is treated for a marrow toxic injury at a center participating in the NMDP's Radiation Injury Transplant Network (RITN) is eligible to participate in the Research Database. This includes adults with and without decision making capacity, and children. Eligible individuals may have received supportive care only, growth factor support, HSC transplant or other appropriate medical treatment for marrow toxic injury. Treatments applied are at the discretion of the care facility, and are not determined by the NMDP or CIBMTR.\n\nUnrelated Donor Eligibility Criteria:\n\n* All donors registered on the NMDP Registry who have been requested to donate a product for a recipient are eligible to participate in the Research Database.\n* All maternal cord blood donors are enrolled in the NMDP Cord Blood Bank Investigational New Drug (IND) protocol, and sign an informed consent document specific to that protocol. Data collected as part of the Cord Blood Bank protocol are included in the Research Database.",{"count":98,"type":21},99999999,"OBSERVATIONAL","The primary purpose of the Research Database is to have a comprehensive source of observational data that can be used to study HSC transplantation and cellular therapies.\n\nA secondary purpose of the Research Database is to have a comprehensive source of data to study marrow toxic injuries.\n\nObjectives:\n\nTo learn more about what makes stem cell transplants and cellular therapies work well such as:\n\n* Determine how well recipients recover from their transplants or cellular therapy;\n* Determine how recovery after a transplant or cellular therapy can be improved;\n* Determine how a donor's or recipient's genetics impact recipient recovery after a transplant or cellular therapy;\n* Determine how access to transplant or cellular therapy for different groups of patients can be improved;\n* Determine how well donors recover from the collection procedures.",[102,103,104,105,106,40,107],"Autologous Stem Cell Transplantation","Allogeneic Stem Cell Transplantation","Solid Tumors","Blood Cancers","CAR-T","Non-malignant Disease",[109,110,111,112,40,106],"Data Collection","CIBMTR","NMDP","Stem Cell Transplantation","2025-08-07",{"date":115,"type":50},"2025-08-12",{"date":117,"type":4},"2002-07",{"date":119,"type":21},"2050-07",{"name":121,"class":122},"Center for International Blood and Marrow Transplant Research","NETWORK",2,{"id":125,"slug":126,"hasResults":11,"nctId":127,"briefTitle":128,"officialTitle":129,"acronym":130,"eligibilityCriteria":131,"healthyVolunteers":11,"sex":65,"minAge":18,"maxAge":4,"enrollmentInfo":132,"targetDuration":4,"studyType":22,"phases":133,"briefSummary":135,"conditions":136,"keywords":4,"overallStatus":46,"whyStopped":4,"lastUpdateSubmitDate":138,"lastUpdatePostDateStruct":139,"startDateStruct":141,"completionDateStruct":143,"leadSponsor":145,"locationsCount":147},"100418448","gene-therapy-for-chinese-hemophilia-a-100418448","NCT04728841","Gene Therapy for Chinese Hemophilia A","Clinical Exploration of Clinical Exploration Adeno-associated Virus Vector Expression of Human Coagulation Factor VIII Gene Therapy for Hemophilia A","HA","Inclusion Criteria:\n\n1. Be able to understand the purpose and risks of the study and provide informed consent according to national and local privacy laws;\n2. Male subjects and ≥ 18 years of age;\n3. Have hemophilia A with ≤1 IU\u002FdL (≤1%) endogenous FVIII activity levels at the time of screening. If the screening result is \\>1% due to previous treatment with FVIII product, then it may be confirmed by documented historical evidence from a certified clinical laboratory demonstrating ≤1% FVIII activity levels ;\n4. No history of hypersensitivity or anaphylaxis associated with FVIII product administration;\n5. Have no measurable FVII inhibitor as assessed by laboratory two times that were at least one week apart; or documented no prior history of FVIII inhibitor after 150 EDs and no clinical signs or symptoms of decreased response to FVIII infusion ;\n6. Have acceptable laboratory values sampled at screening and repeated prior to Day 0; A. Hemoglobin ≥ 11 g\u002FdL; B. Platelets ≥ 100 x 10\\^9\u002FL; C. AST, ALT, alkaline phosphatase ≤ 1.25 upper limit of normal (ULN); D. Bilirubin ≤ 1.25 ULN; E. Creatinine ≤ 2 mg\u002FdL.\n7. Agree to use reliable barrier contraception until the end of the 52 weeks observation period, and three consecutive semen samples are negative for vector sequences after GS001 infusion.\n\nExclusion Criteria:\n\n1. Have Hepatitis B, hepatitis C or HBsAg, HCVAb, HBV-DNA, HCV-RNA are positive and have clinical significance. Both natural clearers and those who have cleared HCV on antiviral therapy are deemed eligible;\n2. Currently Receiving antiviral therapy for hepatitis B and C;\n3. Have history of chronic infections or other chronic diseases that may pose a risk to the study participation;\n4. Have participated in a previous gene therapy research trial within the last 52 weeks or in a clinical study with an investigational drug within the past 30 days;\n5. The subject has any concurrent diseases that cannot tolerate treatments of prednisone or prednisolone as judged by the investigator;\n6. History of arterial or venous thromboembolic events (e.g., deep vein thrombosis, non-hemorrhagic stroke, pulmonary embolism, myocardial infarction, arterial embolism);\n7. Known inherited or acquired thrombophilia, including conditions associated with increased risk of thromboembolism, such as atrial fibrillation;\n8. Major surgery planned in 1 year period following the infusion with GS001;\n9. Hypersensitivity to the study vector;\n10. Have clinically major diseases or any other unspecified conditions that, in the opinion of the Investigator, makes the subject unsuitable for participating in the study;\n11. Patients who are unable or unwilling to comply with the schedule of visits and study assessments described in the clinical protocol;\n12. Evidence of other bleeding disorders not associated with hemophilia A.",{"count":69,"type":21},[134],"NA","IHBDH-GTHA-2020 is an open- label, non- randomized study to evaluate the safety, tolerability and kinetics of a single intravenous infusion of GS001 in hemophilia A subjects with \\\u003C1 IU\u002Fdl residual FVIII levels.",[137,40],"Hemophilia A","2025-07-31",{"date":140,"type":50},"2025-08-01",{"date":142,"type":50},"2021-03-04",{"date":144,"type":21},"2028-07-31",{"name":146,"class":88},"Institute of Hematology & Blood Diseases Hospital, China",1,{"id":149,"slug":150,"hasResults":11,"nctId":151,"briefTitle":152,"officialTitle":152,"acronym":4,"eligibilityCriteria":153,"healthyVolunteers":11,"sex":17,"minAge":154,"maxAge":155,"enrollmentInfo":156,"targetDuration":4,"studyType":22,"phases":158,"briefSummary":160,"conditions":161,"keywords":4,"overallStatus":165,"whyStopped":4,"lastUpdateSubmitDate":166,"lastUpdatePostDateStruct":167,"startDateStruct":169,"completionDateStruct":170,"leadSponsor":172,"locationsCount":147},"100599492","early-phase-1-a-clinical-study-of-the-safety-and-efficacy-of-chemogenetics-therapy-in-the-treatment-of-parkinsons-disease-100599492","NCT07085195","A Clinical Study of the Safety and Efficacy of Chemogenetics Therapy in the Treatment of Parkinson's Disease","Inclusion criteria\n\nParticipants who meet all the following criteria can be included in this clinical study:\n\n1. Clinically diagnosed with idiopathic Parkinson's Disease \\[in accordance with the Diagnostic Criteria for Parkinson's Disease in China published in 2016, or in accordance with the Diagnostic Criteria for Primary PD of the International Parkinson's and Movement Disorders Society (MDS) in 2015\\];\n2. Subjects aged 40-65 years old (including the boundary value), regardless of gender;\n3. Medical history of disease ≥5 years;\n4. Hoehn-Yahr staging scale for \"off\" period is 2.5-4;\n5. Regular use of dopaminergic drugs before the screening period, including taking levodopa for at least 4 weeks;\n6. MDS-UPDRS III score \\>35 in the \"off\" period, and the improvement rate of acute levodopa stress test ≥30%;\n7. AAV neutralizing antibody titer ≤1:2000;\n8. The subject agrees to postpone other neurosurgery during the main study phase (including deep brain stimulation, except for sudden life-threatening conditions requiring neurosurgery during the trial);\n9. No acute adverse reactions to the scheduled therapeutic dose of clozapine (1-month observation period);\n10. The subject agrees not to participate in other therapeutic intervention studies during the trial;\n11. The subject agrees not to be vaccinated during the study phase;\n12. Use reliable contraceptive methods from the screening period to at least 8 weeks after drug infusion;\n13. The subject has good compliance and can follow up regularly. During the follow-up period, the PD patient diary can be accurately completed. Family members, guardians, or caregivers can help the subject fill in the patient diary;\n14. The subject fully understands the nature, purpose, methods, and possible adverse reactions of this clinical trial, voluntarily participates as a subject, and signs the informed consent form; if the subject is unable to read, the legal agent or impartial witness can read the informed consent form and other written materials and witness the informed consent.\n\nExclusion criteria\n\nIf any of the following criteria are met, the patient shall be excluded from this study:\n\n1. Atypical or secondary Parkinson's syndrome (such as Parkinson's plus syndrome, hereditary Parkinson's syndrome, drug-induced Parkinson's syndrome);\n2. Contraindications to surgery or previous deep brain stimulation surgery, pallidotomy, extrapyramidal surgery, other brain surgeries, or other neurosurgery that the researcher determines will affect their participation in this trial;\n3. Previous cranial imaging suggests abnormal brain structure, cerebrovascular malformations, intracranial tumors, intracranial hemorrhage risk, brain trauma, and other abnormalities;\n4. Mini-Mental State Examination (MMSE) score \\\u003C24 points;\n5. Montreal Cognitive Assessment (MoCA) score \\\u003C26 points;\n6. Mini-Mental State Questionnaire (PHQ-9) ≥16;\n7. Abnormal liver and kidney function: Aspartate aminotransferase (AST) or Alanine aminotransferase (ALT) \\> 1.5 times the upper limit of normal value, serum creatinine (Cr) \\>1.5 times the upper limit of normal value;\n8. Abnormal coagulation function or using anticoagulants;\n9. Infectious disease screening: Hepatitis B surface antigen (HBsAg) or hepatitis B virus DNA (HBV-DNA) positive, hepatitis C virus RNA (HCV-RNA) positive, human immunodeficiency virus (HIV) positive, or syphilis serum positive;\n10. Undergoing antiviral treatment for hepatitis B and hepatitis C;\n11. Suffering from unstable or severe other systemic diseases: including active tuberculosis, cardiovascular system, respiratory system, Diseases of the digestive system, urinary system, mental and nervous system (such as epilepsy), blood system, immune system, etc., or abnormal laboratory test values, the investigator determines that the patient is not suitable for participating in this trial;\n12. Suffering from malignant tumors or having a history of malignant tumors;\n13. Having had severe allergic reactions, allergic to contrast agents, or being unable to accept surgical anesthesia;\n14. Participating in other clinical trials or having participated in other clinical trials within 3 months before the screening period;\n15. Having received gene therapy before being enrolled into this trial;\n16. Having received stem cell therapy within 6 months before the screening period;\n17. Having used other investigational drugs within 4 weeks before screening or within 5 half-lives of the investigational drug (whichever is longer), or drugs that the investigator determines affect this trial;\n18. Having received live vaccines within 2 months before the screening period or having a history of vaccination within 30 days before the screening period;\n19. Have a history of alcohol dependence or drug addiction and cannot stop drinking as advised by the doctor during the trial;\n20. Female subjects who are pregnant or breastfeeding;\n21. Severe motor complications after anti-PD drug treatment and no suitable alternative treatment options;\n22. Subjects who are assessed by the researchers as unsuitable for inclusion.","40 Years","65 Years",{"count":157,"type":21},6,[159],"EARLY_PHASE1","The investigators propose a gene therapy strategy using chemical genetic inhibition to intervene in the abnormal activity of the subthalamic nucleus in Parkinson's disease. The investigators design and construct a highly efficient therapeutic injection STP-001 (first drug), through the efficient adeno-associated virus capsid (AAV), neuronal promoter (hSyn), and chemical genetic effector element (hM4Di), and accurately inject the drug into the bilateral subthalamic nucleus, the core pathological nucleus of Parkinson's disease, through stereotactic technology. Combined with a very low dose of clozapine (the second drug), the abnormal activity of the subthalamic nucleus is precisely intervened to improve the core motor symptoms of Parkinson's disease.",[162,40,163,164],"Parkinson Disease (PD)","Safety and Efficacy","Clozapine","NOT_YET_RECRUITING","2025-07-24",{"date":168,"type":50},"2025-07-25",{"date":140,"type":21},{"date":171,"type":21},"2025-12-30",{"name":173,"class":88},"Ruijin Hospital",{"id":175,"slug":176,"hasResults":11,"nctId":177,"briefTitle":178,"officialTitle":179,"acronym":4,"eligibilityCriteria":180,"healthyVolunteers":181,"sex":17,"minAge":182,"maxAge":183,"enrollmentInfo":184,"targetDuration":4,"studyType":22,"phases":186,"briefSummary":187,"conditions":188,"keywords":193,"overallStatus":46,"whyStopped":4,"lastUpdateSubmitDate":166,"lastUpdatePostDateStruct":199,"startDateStruct":201,"completionDateStruct":203,"leadSponsor":205,"locationsCount":147},"100594828","perceptual-evaluation-and-rehabilitation-system-development-for-congenital-hearing-loss-100594828","NCT07024524","Perceptual Evaluation and Rehabilitation System Development for Congenital Hearing Loss","Development and Application of A Multimodal Perceptual Evaluation and Precision Rehabilitation System in Congenital Deafness","Inclusion Criteria:\n\nFor Congenital Deafness Group: Patients with congenital hearing loss with hearing thresholds ≥65 dB who have received hearing aids, cochlear implantation, or gene therapy; Age between 1 and 35 years old, regardless of gender.\n\nFor Healthy Controls Group: Participants with normal hearing thresholds (≤20 dB) whose age and gender matched to the patient group.\n\nMandarin Chinese as the native language. Participants and\u002For their guardians must provide informed consent before the trial, voluntarily sign a written consent form, and commit to receive evaluation at specified time points.\n\nCapable of effective communication with researchers under the guardian's assistance and willing to cooperate and comply with the researchers' requirements.\n\nParticipants and\u002For their guardians should have a correct understanding of the trial and appropriate expectations regarding potential benefits.\n\nExclusion Criteria:\n\nPresence of other otological disorders that may interfere with the surgical outcome or interpretation of study endpoints, such as otitis media, Meniere's disease, etc.\n\nPresence of other severe congenital diseases, such as congenital heart disease. Presence of severe systemic diseases or in the acute onset of diseases, such as pulmonary tuberculosis, active hepatitis B or C infection, active herpes zoster infection, pancreatitis, renal insufficiency, etc.\n\nIndividuals with low immunity, a history of immune deficiency or organ transplantation.\n\nIndividuals with a history of neurological, mental disorders, or moderate-to-severe cognitive dysfunction, such as epilepsy, dementia, autism spectrum disorders, etc.\n\nAny other conditions for which the investigators consider the subject unsuitable for participation in this clinical study.",true,"1 Year","35 Years",{"count":185,"type":21},210,[134],"This study aims to establish a novel multimodal evaluation and precision rehabilitation system for patients with congenital hearing loss who have received hearing aids, cochlear implantation, or emerging gene therapy. The proposed system will integrate age-stratified difficulty levels, combined with a gamified interaction platform or software designed to assess and train three core perceptual domains: auditory speech perception, music perception, and spatial hearing capabilities.",[189,40,190,191,192],"Congenital Hearing Loss","Speech Perception","Rehabilitation","Reliability and Validity",[194,77,195,196,197,198,191],"Congenital hearing loss","Multimodal assessment","Auditory speech perception","Music perception","Spatial hearing",{"date":200,"type":50},"2025-07-28",{"date":202,"type":50},"2025-07-04",{"date":204,"type":21},"2028-05-05",{"name":206,"class":88},"Eye & ENT Hospital of Fudan University",{"id":208,"slug":209,"hasResults":11,"nctId":210,"briefTitle":211,"officialTitle":211,"acronym":4,"eligibilityCriteria":212,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":213,"targetDuration":4,"studyType":22,"phases":214,"briefSummary":215,"conditions":216,"keywords":4,"overallStatus":165,"whyStopped":4,"lastUpdateSubmitDate":218,"lastUpdatePostDateStruct":219,"startDateStruct":221,"completionDateStruct":223,"leadSponsor":225,"locationsCount":4},"100590099","early-phase-1-a-prospective-single-center-open-label-clinical-study-to-evaluate-the-safety-and-efficacy-of-inhaled-bmd003cftr-mrna-in-chinese-adult-patients-with-cystic-fibrosis-100590099","NCT06963008","A Prospective, Single-center, Open-label Clinical Study to Evaluate the Safety and Efficacy of Inhaled BMD003(CFTR mRNA) in Chinese Adult Patients With Cystic Fibrosis","Inclusion Criteria:\n\nPatients must meet all of the following inclusion criteria to be included in this study:\n\n1. Gender not limited, 18 years old and above (including threshold values);\n2. The subjects themselves agree to participate in the study and sign an informed consent form\n3. Diagnosed with cystic fibrosis (CF);\n4. According to the investigators' assessment, the subject's cystic fibrosis disease is clinically stable;\n5. During screening, the subject's body mass index (BMI) is ≥ 17.5 kg\u002Fm ² and their weight is ≥ 40 kg;\n6. Forced Expiratory Volume in 1 second (FEV1) is expected to be ≥ 40% of the normal value;\n7. Resting oxygen saturation (SpO2) ≥ 92%;\n8. Quit smoking for at least 2 years;\n9. The subjects are willing and able to comply with the scheduled visits, treatments, laboratory tests, contraception requirements, and other study procedures.\n\nExclusion Criteria:\n\nIndividuals who meet any of the following criteria are not eligible for inclusion in this study:\n\n1. Acute respiratory infection, acute exacerbation of lung disease, clinically significant hemoptysis (\\>30 mL, or as determined by the investigator), or changes in respiratory medications (including antibiotics, oral steroids, etc.) used to treat CF occurred within 4 weeks prior to the first administration;\n2. Infected with highly virulent bacteria associated with accelerated decline in lung function and\u002For shortened survival time (e.g. Burkholderia cepacia, Mycobacterium abscesses). For subjects with a positive culture history, they can be considered uninfected according to the following guidelines:\n\n   All culture results obtained in the past 12 months have been negative. Subject should have undergone at least 2 cultures within the past 12 months, with a minimum interval of 3 months between each culture, and at least one culture should have been conducted within 6 months prior to screening in this study.\n3. During screening, the 12 lead electrocardiogram showed prolonged QTcF interval (\\>450 ms for males and\\>460 ms for females) and clinically significant abnormalities in other indicators of the 12 lead electrocardiogram.\n4. During screening, total bilirubin exceeded the upper limit of normal, ALT and AST exceeded three times the upper limit of normal, and blood creatinine (CRE) was ≥ 1.5 times the upper limit of normal.\n5. History of physical organ or blood transplantation or registered waiting for transplantation;\n6. During screening, positive for human immunodeficiency virus antibodies (HIV Ab) and positive for Treponema pallidum specific antibodies (TPPA);\n7. Participated in inhalation drug or device studies within 30 days prior to the initial screening visit (a 30 day window period applies to inhalation drugs with elimination half-life\\\u003C6 days. If the elimination half-life of inhalation drugs is ≥ 6 days, the window period should be extended to at least 5 half lives after the last administration);\n8. Pregnant (positive blood pregnancy test result) or lactating women;\n9. Having a history of drug allergies or other allergies, which investigators consider to be contraindications for participation in the study;\n10. History of drug allergies or other allergies, and investigators believe it contraindications for participation in the study;\n11. Investigators believe that any medical indicators (including other laboratory test indicators with clinical significance) or other conditions that may affect clinical trials.",{"count":69,"type":21},[159],"The study adopts a prospective, single-center, open-label clinical study to evaluate the safety and efficacy of inhaled BMD003(CFTR mRNA) in Chinese adult patients with cystic fibrosis This study is a single dose escalation study of BMD003 nebulized inhalation in CF patients.\n\nThe study is divided into four cohorts, they are 2mg, 4mg, 8mg, and 16mg dose groups. The first dose group (2mg) is planned to enroll one patient. During the study, if there are ≥ 2 grade treatment-related adverse events (TRAEs) related to the investigational product that occur during the study period, 2 patients will be added at the current dose level. Each of the remaining groups will include 2-3 subjects, regardless of gender. Based on the safety data review of subjects in the previous dose group 4 weeks after administration, subjects in the next dose group will be allowed to receive the next higher dose, while patients in the previous dose group will complete the study.\n\nThe study includes three stages: screening period (2 weeks), administration period (1 day), and follow-up period (approximately 13 months). After passing the screening of the subjects, D1 was admitted to the hospital, D1 received a single nebulized inhalation investigational product (BMD003), and D2 completed safety observation before being discharged.\n\nDuring the study, PK sample collection and safety assessment will be conducted at the corresponding visit points. After the treatment is completed, each patient will be followed up according to the schedule specified in the protocol.This study will conduct multiple dosing studies after the end of a single dose escalation. Multiple dosing doses, dosing intervals, etc. will be revised based on preclinical data and single dose data, and will be implemented after ethical approval.",[217,40],"Cystic Fibrosis (CF)","2025-04-30",{"date":220,"type":50},"2025-05-08",{"date":222,"type":21},"2025-05-10",{"date":224,"type":21},"2027-05-10",{"name":226,"class":88},"Peking Union Medical College Hospital",{"id":228,"slug":229,"hasResults":11,"nctId":230,"briefTitle":231,"officialTitle":232,"acronym":4,"eligibilityCriteria":233,"healthyVolunteers":11,"sex":17,"minAge":234,"maxAge":235,"enrollmentInfo":236,"targetDuration":4,"studyType":22,"phases":238,"briefSummary":239,"conditions":240,"keywords":253,"overallStatus":165,"whyStopped":4,"lastUpdateSubmitDate":262,"lastUpdatePostDateStruct":263,"startDateStruct":265,"completionDateStruct":267,"leadSponsor":269,"locationsCount":147},"100588946","phase-1-safety-and-efficacy-of-aav9ap4b1-bfb-101-for-patients-with-ap4b1-related-hereditary-spastic-paraplegia-type-47-spg47-100588946","NCT06948019","Safety and Efficacy of AAV9\u002FAP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)","Safety and Efficacy of AAV9\u002FAP4B1 For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47): A Phase 1\u002F2 Single-Center, Open-Label Study of Stereotactic Intra-cisterna Magna Administration","Inclusion Criteria:\n\n1. Male and females between the ages of 12 months - 5 years at the time of treatment\n2. A molecularly confirmed diagnosis of SPG47 (confirmed by a CLIA certified, CE-marked, or equivalent lab): Genomic DNA mutation analysis demonstrating bi-allelic pathogenic variants in the AP4B1 gene.\n3. Proband must have features of neurologic dysfunction by clinical history and physical examination.\n4. Stable doses of concomitant medications such as anti-spasticity medications, anti-epileptic medications, behavioral management medications, sleep medications, and special diets, supplements or nutritional support for at least 3 months prior to Screening. If recent changes (\\\u003C 3 months) in medications, the participant may be allowed per Investigator judgement.\n5. Proband must be fully vaccinated per Centers for Disease Control recommendations for childhood vaccinations.\n6. Two competent custodial parents\u002Fguardians with legal capacity (legally acceptable representatives) to execute an Institutional Review Board\u002FIndependent Ethics Committee (IRB\u002FIEC) approved consent for medical research must be able to participate in the consent process. If only one parent has sole custody to consent for medical research, then that parent must be able to actively participate in the consent process.\n7. Legally acceptable representatives must be able to attend all scheduled study visits and provide feedback regarding the participant's symptoms and performance as described in the protocol.\n8. Legally acceptable representatives agree not to post any of the participant's personal medical data or information related to the study on any website or social media site (e.g., Facebook, Instagram, Twitter, YouTube, etc.) until notified that the study is completed.\n9. Proband and the proband's family must demonstrate ability to travel to the study center. For the first 30 days post treatment probands will need to stay within a 100-mile radius from the treatment center.\n\nExclusion Criteria:\n\n1. Inability to participate in the clinical evaluation as determined by the principal investigator.\n2. Clinically significant abnormal laboratory values (hemoglobin \\\u003C 8 or \\> 20 g\u002FdL; white blood cell \\> 20,000 per cmm, platelets count \\\u003C 100,000 per cmm; international normalized ratio \\[INR\\] \\> upper limit of normal \\[ULN\\]; gamma-glutamyl transferase \\[GGT\\], alanine aminotransferase \\[ALT\\], and aspartate aminotransferase \\[AST\\] or total bilirubin \\> 1.5 × ULN, creatinine\n\n   ≥ 1.5 mg\u002FdL) prior to gene replacement therapy.\n3. Presence of a concomitant medical condition that precludes a cisterna magna or lumbar puncture or use of anesthetics for sedated procedures.\n4. Bleeding disorder or any other medical condition or circumstance in which a cisterna magna or lumbar puncture is contraindicated according to local institutional policy.\n5. Documented cardiomyopathy or significant congenital heart abnormalities.\n6. Inability to be safely sedated in the opinion of the clinical anesthesiologist.\n7. History of severe\u002Flife-threatening allergic reaction to sirolimus, tacrolimus, corticosteroids, or gadolinium.\n8. Any known history and\u002For family history of hemophagocytic lymphohistiocytosis (HLH) or multisystem inflammatory syndrome (MIS)\n9. Concomitant illness or requirement for chronic drug treatment that in the opinion of the investigator creates unnecessary risks for gene transfer.\n10. Concomitant chronic drug treatment that would cause clinically significant interactions with immunosuppressive agents used in the study.\n11. Any item which would exclude the participant from being able to undergo magnetic resonance imaging (MRI) according to local institutional policy.\n12. Any other situation that would exclude the participant from undergoing any other procedure required in this study.\n13. Visual or hearing impairment sufficient to preclude cooperation with neurodevelopmental testing.\n14. The presence of significant non-SPG47 related central nervous system (CNS) impairment or behavioral disturbances that would confound the scientific rigor or interpretation of results of the study.\n15. Recent or planned elective surgical procedures that would confound the scientific rigor or interpretation of results of the study, as determined by the Investigator\u002Fstudy team.\n16. Failure to obtain appropriate informed consent.\n17. Reason to believe that the participant or parents\u002Fguardians of the participant will not comply with the study procedures outlined in the study protocol.\n18. Receiving a live vaccine within 30 days prior to gene transfer.\n19. Receiving an investigational drug within 30 days prior to screening or plan to receive an investigational drug (other than gene therapy) during the study.\n20. Enrollment and participation in another interventional clinical trial.","12 Months","60 Months",{"count":237,"type":21},5,[24,71],"Safety and Efficacy of AAV9\u002FAP4B1 For Patients with AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47): A Phase 1\u002F2 Single-Center, Open-Label Study of Stereotactic Intra-cisterna Magna Administration.\n\nThe goal of this clinical trial is to evaluate whether a gene therapy can safely treat children with SPG47, a rare genetic condition that causes progressive spasticity and developmental delays. The main questions it aims to answer are:\n\n* Is the gene therapy safe and well tolerated?\n* Does the gene therapy improve motor function and developmental outcomes?\n\nParticipants will:\n\n* Undergo screening assessments to confirm eligibility\n* Receive a single dose of the gene therapy vector\n* Attend follow-up visits for safety monitoring and developmental assessments over the course of five years",[241,242,243,244,245,246,247,248,249,250,251,252,40],"HSP","Hereditary Spastic Paraplegia","Hereditary Spastic Paraparesis","Hereditary Spastic Paraplegia Type 50","Hereditary Spastic Paraplegia Type 47","Hereditary Spastic Paraplegia Type 51","Hereditary Spastic Paraplegia Type 52","SPG47","AP4B1","Neurogenetic Disorders","Neurodevelopmental Conditions","Movement Disorders",[241,242,244,245,246,247,243,254,40,255,248,249,256,257,258,259,260,261],"Spasticity","AAV9","AP4M1","AP4E1","AP4S1","movement disorders","neurogenetic conditions","neurodevelopmental conditions","2025-04-22",{"date":264,"type":50},"2025-04-28",{"date":266,"type":21},"2025-08",{"date":268,"type":21},"2032-08",{"name":270,"class":57},"BlackfinBio Ltd",{"id":272,"slug":273,"hasResults":11,"nctId":274,"briefTitle":275,"officialTitle":276,"acronym":4,"eligibilityCriteria":277,"healthyVolunteers":181,"sex":17,"minAge":278,"maxAge":279,"enrollmentInfo":280,"targetDuration":4,"studyType":99,"phases":4,"briefSummary":282,"conditions":283,"keywords":285,"overallStatus":46,"whyStopped":4,"lastUpdateSubmitDate":288,"lastUpdatePostDateStruct":289,"startDateStruct":291,"completionDateStruct":293,"leadSponsor":295,"locationsCount":147},"100515425","growth-and-development-related-outcomes-in-children-with-transfusion-dependent-beta-thalassemia-after-gene-therapy-100515425","NCT05991336","Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy","Growth and Development, Health-related Quality of Life of Children With Transfusion-dependent Beta-thalassemia After Gene Therapy","Inclusion Criteria:\n\nGene therapy group-inclusion\n\n* Male or female age of 3-14 years\n* TDT Children who have received gene therapy.\n* Subjects who are willing and able to provide written informed consent.\n\nSupportive therapy group-inclusion\n\n* Gender same as the matched case\n* Age similar to the matched case\n* Children with β-TDT\n* No history of gene therapy or allogeneic hematopoietic stem cell transplantation\n* Subjects who are willing and able to provide written informed consent.\n\nHealthy children group-inclusion\n\n* Gender same as the matched case\n* Age similar to the matched case\n* Subjects who are willing and able to provide written informed consent.\n\nExclusion Criteria:\n\n* Diagnosis of compound α-thalassemia\n* Uncontrolled systemic fungal, bacterial, or viral infection\n* History of malignant solid tumors, myeloproliferative or immunodeficiency diseases\n* Diagnosed with mental illness\n* Patients considered to be ineligible for the study by the investigator for reasons other than the above\n\nDiscontinuation of Study :\n\n* Subjects who are unwilling or unable to continue participating in the study (withdrawal of informed consent) may withdraw from the study\n* Subjects who received gene therapy or allogeneic hematopoietic stem cell transplantation during the study\n* The subject is seriously non-compliant with the study requirements, such as missing 2 consecutive visits\n* Subject lost to follow-up","3 Years","14 Years",{"count":281,"type":21},100,"The investigate will conduct a cohort study to compare the growth and development, metabolism, lifestyle behavior, and health-related quality of life among three groups: children with transfusion-dependent β-thalassemia (TDT) who have received gene therapy, TDT children with lifelong supportive therapy and healthy children.",[284,40],"Transfusion-dependent Beta-Thalassemia",[284,44,286,287],"cohort study","case-control","2023-08-06",{"date":290,"type":50},"2023-08-14",{"date":292,"type":50},"2023-06-05",{"date":294,"type":21},"2029-12-31",{"name":146,"class":88}]