863 completed, 231 recruiting, 176 unknown, 171 terminated. 458 Phase 2, 325 Phase 1, 232 Phase 3, 215 Not applicable, 55 Phase 4.
Follow Genetic Diseases, X-Linked: new trials by email Literature Review of Genetic Diseases, X-Linked trials Research Report on Genetic Diseases, X-Linked
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Lentiviral Gene Therapy for CGD Infusion of lentiviral TYF-CGD-modified autologous stem cells - Chronic Granulomatous Disease | Not applicable | Not yet recruiting | Shenzhen Geno-Immune Medical Institute | 10 | 2027-07-01 | 2026-08-26 |
| Efficacy and Safety of NTI164 in Children and Young Adults With Rett Syndrome NTI164, Placebo - RETT Syndrome With Proven MECP2 Mutation, Rett Syndrome | Phase 2 / Phase 3 | Not yet recruiting | Fenix Innovation Group | 40 | 2027-07-01 | 2026-09-09 |
| SCID-X1 Gene Therapy Via Intravenous Lentiviral (Ivlv-X1) Injection Direct intravenous injection of ivlv-X1 lentiviral vector - SCID, X-Linked | Phase 1 / Phase 2 | Not yet recruiting | Shenzhen Geno-Immune Medical Institute | 10 | 2027-06-30 | 2026-08-27 |
| Lentiviral FIX Gene Therapy YUVA-GT-F901 - Hemophilia B | Phase 1 | Not yet recruiting | Shenzhen Geno-Immune Medical Institute | 10 | 2027-06-01 | 2026-08-26 |
| IT and IV Lentiviral Gene Therapy for X-ALD Intrathecal and intravenous LV gene therapy - X-linked Adrenoleukodystrophy | Not applicable | Not yet recruiting | Shenzhen Geno-Immune Medical Institute | 30 | 2027-06-01 | 2026-09-01 |
| Multicomponent Pain Self-Management in Adults With Haemophilic Arthropathy Multicomponent pain self-management programme - Hemophilic Arthropathy, Chronic Pain | Not applicable | Not yet recruiting | Investigación en Hemofilia y Fisioterapia | 70 | 2027-03-01 | 2026-10-06 |
| Ultrasound Assessment of Joint Health in Patients With Mild Hemophilia (Factor Levels 5-40%) Joint Ultrasound (HEAD-US), Blood Sample for Coagulation Factor Measurement - Mild Hemophilia | Not applicable | Not yet recruiting | Assistance Publique - Hôpitaux de Paris | 150 | 2027-02-01 | 2026-08-17 |
| Outside the Cage (OTC) Robotic Esophagectomy OTC MIE Esophagectomy - Esophageal Cancer | Phase 1 | Not yet recruiting | Centre hospitalier de l'Université de Montréal (CHUM) | 31 | 2027-01-01 | 2026-03-18 |
| CD19-B Cell Depletion in PAIS-ME/CFS Patients Inebilizumab, Placebo - Post-acute Infectious Syndrome (PAIS), Post-COVID 19 Condition (PCC or Long COVID) | Phase 2 | Not yet recruiting | Charite University, Berlin, Germany | 38 | 2026-12-01 | 2026-07-24 |
| A Study to Investigate the Effects and Safety of Codabakalner for the Treatment of FXS in Male Participants and Adult Participants With ADS Codabakalner, Placebo - Fragile X Syndrome (FXS), Autism Spectrum Disorder (ASD) | Phase 2 / Phase 3 | Not yet recruiting | Spinogenix | 120 | 2026-12-01 | 2026-10-09 |
| Therapeutic Implications of CMR Low Native T1 in Non-treated Non-hypertrophic Females With Fabry Disease (FD) Decision tree for initiating treatment - Fabry Disease | Not applicable | Not yet recruiting | University Hospital, Bordeaux | 60 | 2026-11-01 | 2026-10-14 |
| Urotherapy Education for Families of Children With Duchenne Muscular Dystrophy Urotherapy Education - Duchenne Muscular Dystrophy (DMD), Lower Urinary Tract Symptoms (LUTS) | Not applicable | Not yet recruiting | Lokman Hekim University | 50 | 2026-11-01 | 2026-09-15 |
| Liver Biopsy Following Gene Therapy For Hemophilia Liver Biopsy - Hemophilia A, Hemophilia B | Not yet recruiting | St. Jude Children's Research Hospital | 8 | 2026-11-01 | 2026-10-22 | |
| Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting ELEVIDYS, Sirolimus, Glucocorticoids - Duchenne Muscular Dystrophy | Phase 4 | Recruiting | Sarepta Therapeutics, Inc. | 20 | 2026-10-30 | 2026-10-06 |
| A Study to Learn About the Study Medicine (Called Marstacimab) in People With Hemophilia A or B Marstacimab - Hemophilia | Not yet recruiting | Pfizer | 100 | 2026-10-05 | 2026-10-05 | |
| Post-acute Infectious Syndrome - Aripiprazole Symptom Evaluation (PAIS-AriSE) Aripiprazole (low-dose, 1mg), Placebo - Post-acute Infectious Syndrome (PAIS), Post-COVID-19 Condition (PCC or Long COVID) | Phase 2 | Not yet recruiting | Christiana Franke | 138 | 2026-10-01 | 2026-07-20 |
| A Phase III Clinical Trial Evaluating SR604 Injection for Prophylactic Treatment in Patients With Hemophilia SR604 - Hemophilia A, Hemophilia A and B | Phase 3 | Not yet recruiting | Shanghai RAAS Blood Products Co., Ltd. | 90 | 2026-09-30 | 2026-10-15 |
| To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome Bionetide, Placebo - Rett Syndrome | Phase 3 | Recruiting | Biomed Industries, Inc. | 210 | 2026-09-30 | 2026-03-27 |
| Phase 1/2 Study of KRRO-121 in Healthy Volunteers and Patients With UCD KRRO-121, Placebo - Urea Cycle Disorders, Inborn, Urea Cycle Disorders | Phase 1 / Phase 2 | Recruiting | Korro Bio, Inc. | 30 | 2026-09-28 | 2026-10-08 |
| Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy KER-065 - Duchenne Muscular Dystrophy | Phase 2 | Recruiting | Keros Therapeutics, Inc. | 36 | 2026-09-25 | 2026-09-30 |
Showing 20 of 1710. Sign in to see all of them.
Muscular Dystrophy, Duchenne (478) · Hemophilia B (260) · Fabry Disease (243) · Hemophilia A (140) · Fragile X Syndrome (113) · Rett Syndrome (95) · Muscular Dystrophies (90) · Granulomatous Disease, Chronic (78) · Mucopolysaccharidosis II (77) · Adrenoleukodystrophy (62) · Ornithine Carbamoyltransferase Deficiency Disease (59) · Bulbo-Spinal Atrophy, X-Linked (57) · Genetic Diseases, Inborn (50) · Wiskott-Aldrich Syndrome (41)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (1707) · Genetic Diseases, Inborn (1660) · Nervous System Diseases (1207) · Nutritional and Metabolic Diseases (563) · Metabolic Diseases (534) · Neuromuscular Diseases (528) · Musculoskeletal Diseases (522) · Central Nervous System Diseases (510) · Metabolism, Inborn Errors (484) · Muscular Disorders, Atrophic (479) · Muscular Diseases (475) · Heredodegenerative Disorders, Nervous System (444) · Brain Diseases (434) · Hemic and Lymphatic Diseases (416)
Factor IX (31) · agalsidase beta (28) · migalastat (28) · agalsidase alfa (24) · larazotide acetate (19) · Busulfan (18) · Alemtuzumab (16) · Cyclophosphamide (15) · Blood Specimen Collection (14) · ataluren (14) · fludarabine (14) · idursulfase (14) · Whole-Body Irradiation (12) · eteplirsen (12) · Prednisone (11)
National Institute of Allergy and Infectious Diseases (NIAID) (39) · Novo Nordisk A/S (36) · Pfizer (29) · Sarepta Therapeutics, Inc. (27) · Shire (27) · Amicus Therapeutics (24) · Assistance Publique - Hôpitaux de Paris (22) · Genzyme, a Sanofi Company (20) · Hacettepe University (20) · PTC Therapeutics (19) · Baxalta now part of Shire (17) · CSL Behring (17) · National Institute of Neurological Disorders and Stroke (NINDS) (17) · Cooperative International Neuromuscular Research Group (15) · Novartis Pharmaceuticals (15)
United States (877) · United Kingdom (292) · France (245) · Italy (228) · Canada (196) · Germany (186) · Spain (179) · Australia (139) · Turkey (Türkiye) (114) · Belgium (112) · Netherlands (107) · China (88) · Japan (87) · Brazil (70) · Poland (69)
Elizabeth M Kang, M.D. (9) · Dorothy K Grange, MD (6) · Suk S De Ravin, M.D. (6) · Craig McDonald, MD (5)
PMID 40750723, PMID 27657681, PMID 37460385, PMID 25075835, PMID 26196195, PMID 40386832, PMID 20004776, PMID 23818196, PMID 24139498, PMID 24290292, PMID 24331379, PMID 27262745, PMID 36408876, PMID 25295500, PMID 30194931, PMID 30261899, PMID 32956407, PMID 33395098, PMID 35075289, PMID 36848635, PMID 37539981, PMID 38363467, PMID 39383458, PMID 39847314, PMID 41671094, PMID 42613652, PMID 17546030, PMID 19772971, PMID 21586746, PMID 23035069, PMID 23176611, PMID 24399845, PMID 25071871, PMID 25283752, PMID 25795794, PMID 25801175, PMID 26573217, PMID 26764156, PMID 26855682, PMID 27509102, PMID 30193840, PMID 31889231, PMID 32434278, PMID 32522498, PMID 32851872, PMID 32994552, PMID 34693725, PMID 34779542, PMID 34922648, PMID 35568815
2023 (19), 2009 (14), 2018 (12), 2019 (11), 2015 (10), 2025 (10), 2016 (9), 2017 (9), 2014 (7), 2008 (5), U54NS065768 (5), U54DK083908 (4), K23HD109375 (2), P30CA015704 (2), R44NS095423 (2)