[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Genethon\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":122},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,49,75,98],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":14,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":29,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":37,"lastUpdatePostDateStruct":38,"startDateStruct":41,"completionDateStruct":43,"leadSponsor":45,"locationsCount":48},"100643744","multicenter-epidemiological-study-for-the-evaluation-of-the-seroprevalence-of-total-and-neutralizing-antibodies-against-adenoassociated-virus-serotypes-used-in-gene-therapy-in-patients-with-neuromuscular-diseases-of-genetic-origin-100643744",false,"NCT07633951","Multicenter Epidemiological Study for the Evaluation of the Seroprevalence of Total and Neutralizing Antibodies Against Adenoassociated Virus Serotypes Used in Gene Therapy in Patients With Neuromuscular Diseases of Genetic Origin.","SeroDysMyo","Inclusion Criteria:\n\n1. Pediatric patient 6 years of age or older or adult under 60 years of age with genetic neuromuscular disease\n2. Informed patient who signed informed consent\n3. No opposition from the holders of parental authority or guardian, for minor patients.\n4. Affiliated\u002Fbeneficiary of a national health insurance scheme\n\nExclusion Criteria:\n\n* Gene or cell therapy treatment prior to blood collection","ALL","6 Years","60 Years",{"count":20,"type":21},450,"ESTIMATED","INTERVENTIONAL",[24],"NA","This study aims to assess the overall seroprevalence of neutralizing antibodies against different AAVs potentially used in gene therapy in patients with genetic neuromuscular diseases.",[27,28],"Neuromuscular Deficits","Genetic Disorder",[30,31,32,33,34,35],"AAV","Seroprevalence","Nab","Tab","neuromuscular","Genetic disorder","NOT_YET_RECRUITING","2026-06-03",{"date":39,"type":40},"2026-06-08","ACTUAL",{"date":42,"type":21},"2026-07-31",{"date":44,"type":21},"2027-12-31",{"name":46,"class":47},"Genethon","OTHER",1,{"id":50,"slug":51,"hasResults":11,"nctId":52,"briefTitle":53,"officialTitle":54,"acronym":4,"eligibilityCriteria":55,"healthyVolunteers":11,"sex":56,"minAge":57,"maxAge":58,"enrollmentInfo":59,"targetDuration":4,"studyType":61,"phases":4,"briefSummary":62,"conditions":63,"keywords":4,"overallStatus":65,"whyStopped":4,"lastUpdateSubmitDate":66,"lastUpdatePostDateStruct":67,"startDateStruct":69,"completionDateStruct":71,"leadSponsor":73,"locationsCount":74},"100353510","natural-history-of-duchenne-muscular-dystrophy-100353510","NCT03882827","Natural History of Duchenne Muscular Dystrophy","A Prospective, Interventional, Baseline Study In Young Male Subjects Aged From 4 to 9 Years","Inclusion Criteria:\n\n1. Male\n2. 4 to 9 years old inclusive\n3. Body-weight ≤ 95th percentile or the BMI scale ≤ 95th percentile (according to validated scale in force in country site).\n\n   Related to the DMD disease:\n4. Diagnosis of DMD based upon documented gene testing with detailed genotyping\n5. Able to achieve at inclusion and screening visits:\n\n   1. NSAA (North Star Ambulatory Assessment) scale \\> 18 or ≥ 16 if participant is between 4 and \\\u003C 5 years old at screening and:\n   2. Gowers test \\\u003C or = 7 sec and\u002For\n   3. 6-Minute Walk Test (6MWT): a distance ≥ 350 meters at inclusion visit (M0)\n6. Ongoing corticosteroid therapy or initiation of corticosteroid therapy according to standard of care prior to Screening visit\n\n   Related to the study protocol and ICH\u002FGCP (Good Clinical Practice) requirements:\n7. Signed informed consent by at least one parent or both parents or legal guardian representative(s), when applicable and according to the country regulation\n8. Affiliated to or a beneficiary of a Health Care scheme (according to country regulation)\n\n   Exclusion Criteria:\n\n   Subject will be excluded from enrolment into the study for any of the following reasons:\n\n   Related to the DMD disease severity:\n9. Cardiomyopathy based on physical\u002Fcardiological examination and echocardiography with Left Ventricular Simpson biplane Ejection Fraction (LVEF) below 55%\n10. Respiratory Assistance: need for either a diurnal and\u002For a nocturnal ventilation\n11. Any co-morbidity (ies) and or previous or planned surgical event(s) which may interfere with DMD natural evolution and or evaluation of outcomes designed to assess DMD Natural History\n\n    Related to specific assessments:\n12. Muscle testing: inability to cooperate with\n13. MRI: metal implants in regions of interest for the study\n\n    Related to the study protocol and ICH\u002FGCP requirements:\n14. Unwilling and\u002For unable to comply with all the study protocol requirements and\u002For procedures\n15. Previous inclusion to another clinical trial with an Investigational Medicinal Product (IMP), within the 3 months or IMP washout period (whichever is longer) prior to the screening visit of the study\n16. Previously treated with a gene therapy drug for DMD, such as:\n\n    * any AAV mediated gene transfer products or any gene editing products in a clinical trial or in a clinical setting,\n    * if exons skipping drug was used, the last dose of exon skipping drug within 5 half-lives prior to the screening visit\n17. Concomitant participation to any other interventional clinical trial","MALE","4 Years","9 Years",{"count":60,"type":21},220,"OBSERVATIONAL","Baseline Study on Duchenne Muscular Dystrophy (DMD) in view to collect data on the natural disease course in a cohort in young male subjects aged from 4 to 9 Years over a period of 6 to 36 months using disease appropriate evaluations.",[64],"Duchenne Muscular Dystrophy","RECRUITING","2026-02-10",{"date":68,"type":40},"2026-02-12",{"date":70,"type":40},"2019-12-19",{"date":72,"type":21},"2029-09-30",{"name":46,"class":47},15,{"id":76,"slug":77,"hasResults":11,"nctId":78,"briefTitle":79,"officialTitle":80,"acronym":4,"eligibilityCriteria":81,"healthyVolunteers":11,"sex":16,"minAge":82,"maxAge":4,"enrollmentInfo":83,"targetDuration":4,"studyType":22,"phases":85,"briefSummary":87,"conditions":88,"keywords":4,"overallStatus":65,"whyStopped":4,"lastUpdateSubmitDate":90,"lastUpdatePostDateStruct":91,"startDateStruct":93,"completionDateStruct":95,"leadSponsor":97,"locationsCount":48},"100555893","phase-2-efficacy-and-safety-of-gnt0003-following-imlifidase-pre-treatment-in-severe-crigler-najjar-syndrome-100555893","NCT06518005","Efficacy and Safety of GNT0003 Following Imlifidase Pre-treatment in Severe Crigler-Najjar Syndrome","An Open-label, Phase 2 Trial to Evaluate the Efficacy and Safety of a Single Intravenous Administration of GNT0003 (an Adeno-associated Viral (AAV) Vector Expressing the UGT1A1 Transgene) Following Imlifidase Pre-treatment in Adult Participants With Severe Crigler-Najjar Syndrome (CNS) Requiring Daily Phototherapy and Presenting Pre-existing Anti-AAV8 Antibodies","Main Inclusion Criteria:\n\n1. Severe Crigler-Najjar syndrome requiring ≥ 6 hours\u002F day of phototherapy\n2. Molecular confirmation of mutation in the UGT1A1gene by DNA sequencing\n3. Detectable serum neutralizing antibodies against AAV8\n4. Laboratory parameters value not clinically significant\n5. Highly effective method of contraception\n6. Affiliated to or a beneficiary of a health care system\n\nExclusion Criteria:\n\n1. Participation in another interventional trial within 6 months prior to start of clinical trial intervention and during the whole clinical trial\n2. Fibrosis score ≥ 3 (METAVIR) or 10 kPa (FibroScan®)\n3. Liver transplantation\n4. Significant underlying liver disease, chronic hepatitis B, C and\u002For infected with Human immunodeficiency virus\n5. Any other clinically significant illness\n6. Uncontrolled hyperlipidemia.\n7. History of major thrombotic events, active peripheral vascular disease, proven hypercoagulable conditions,\n8. History or presence of thrombotic thrombocytopenic purpura (TTP) or known familial history of TTP\n9. Prior or current treatment with Gene therapy, cell based therapy, CRISPR\u002FCas9 or any other form of gene editing, imlifidase","18 Years",{"count":84,"type":21},3,[86],"PHASE2","Clinical trial rationale:\n\nCNS is an ultra-rare (\\\u003C1\u002F1 million newborns), autosomal recessive disorder of bilirubin conjugation caused by mutation in the gene coding for uridine 5'-diphosphate glucuronosyltransferase (UGT1A1), that causes the accumulation of neurotoxic unconjugated bilirubin (UCB).\n\nReduction of UCB is managed with phenobarbital in mild CNS, and daily phototherapy in severe CNS.\n\nThere is no authorized curative medical treatment for CNS. Liver transplantation is currently the only curative treatment for severe CNS.\n\nGNT0003 is a genetically modified recombinant (r) viral vector composed of the AAV8 viral capsid carrying the UGT1A1 transgene which aims to correct the dysfunction of the mutated gene by achieving durable expression of a functional copy of the affected gene.\n\nImlifidase (IgG-degrading enzyme) has demonstrated its efficacy in highly sensitized adult kidney transplant patients.\n\nTo give participants with pre-existing anti-AAV8 antibodies access to gene therapy treatments, this trial aims to demonstrate the safety and efficacy of GNT0003 following imlifidase pre-treatment in adult participants with severe CNS requiring daily phototherapy and presenting with pre-existing anti-AAV8 antibodies.\n\nPrimary objective: to assess efficacy of a single intravenous administration of GNT0003 following imlifidase pre-treatment in participants with severe CNS requiring phototherapy and pre-existing AAV8 antibodies\n\nSecondary objective: to collect data on safety and tolerability of GNT0003 and imlifidase, efficacy of imlifidase, pharmacokinetic and pharmacodynamic profile of GNT0003, and Quality of Life.\n\nThe trial will include 3 parts:\n\n* A baseline period for at least 3 months\n* A treatment period\n* A follow-up period:\n\n  * Initial post-treatment follow-up over 48 weeks\n  * Long-term follow-up for 4 additional years\n\nThis trial will be conducted in accordance with the International Conference on Harmonization Guideline for Good Clinical Practice and the Declaration of Helsinki. Participants must be consented using the approved Informed Consent Form before any procedures specified in the protocol are performed.",[89],"Crigler-Najjar Syndrome","2024-11-28",{"date":92,"type":40},"2024-12-03",{"date":94,"type":40},"2024-11-08",{"date":96,"type":21},"2030-09-30",{"name":46,"class":47},{"id":99,"slug":100,"hasResults":11,"nctId":101,"briefTitle":102,"officialTitle":103,"acronym":104,"eligibilityCriteria":105,"healthyVolunteers":11,"sex":16,"minAge":58,"maxAge":4,"enrollmentInfo":106,"targetDuration":4,"studyType":22,"phases":108,"briefSummary":109,"conditions":110,"keywords":111,"overallStatus":65,"whyStopped":4,"lastUpdateSubmitDate":114,"lastUpdatePostDateStruct":115,"startDateStruct":117,"completionDateStruct":119,"leadSponsor":121,"locationsCount":5},"100321561","gene-therapy-for-severe-crigler-najjar-syndrome-100321561","NCT03466463","Gene Therapy for Severe Crigler Najjar Syndrome","A Phase I\u002FII, Open Label, Study to Evaluate Safety and Efficacy of an Intravenous Injection of GNT0003 (AAV Vector Expressing the UGT1A1 Transgene) in Patients With Severe Crigler-Najjar Syndrome Requiring Phototherapy","CareCN","Inclusion Criteria:\n\n* Patients with severe Crigler-Najjar syndrome resulting from a molecular confirmation of mutations in the UGT1A1 gene and requiring phototherapy\n* Male or female at least 9 years at the date of signature of informed consent\n* Patient able to give informed assent and\u002For consent in writing\n\nExclusion Criteria:\n\n* Patients who underwent liver transplantation\n* Patients with chronic hepatitis B or C\n* Patients infected with Human immunodeficiency virus (HIV)\n* Patients with significant underlying liver disease\n* Patients with significant encephalopathy\n* Participation in any other investigational trial during this trial\n* Patients unable or unwilling to comply with the protocol requirements",{"count":107,"type":21},17,[24],"This is a Phase 1\u002F2, multinational, open-label, study to evaluate the safety and efficacy of an intravenous infusion of GNT0003 in patients with Crigler-Najjar aged ≥10 years and requiring phototherapy. Patients will received a single administration of GNT0003 and will be followed for safety and efficacy of approximately 60 months (5 years):\n\n* a follow-up of approximately 12 months (48 weeks)\n* a long term follow-up of approximately 48 months (4 years), in order to be in line with the latest EMEA Guideline on follow-up of patients administered with gene therapy medicinal products, released on 22 Oct.2009 by the Committee for medicinal products for human use.",[89],[112,113],"Crigler-Najjar","Adeno Associated Virus","2023-03-23",{"date":116,"type":40},"2023-03-28",{"date":118,"type":40},"2018-03-19",{"date":120,"type":21},"2030-03-30",{"name":46,"class":47},""]