24 recruiting. 24 Phase 3, 5 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 24 | Recruiting 24 |
| Phase 2 | 5 | Recruiting 5 |
Follow Heredodegenerative Disorders, Nervous System: new trials by email Literature Review of Heredodegenerative Disorders, Nervous System trials Research Report on Heredodegenerative Disorders, Nervous System
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 |
|---|---|---|---|---|---|---|
| A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia Nomlabofusp, Placebo - Friedreich Ataxia, Friedreich's Ataxia | Phase 3 | Recruiting | Larimar Therapeutics, Inc. | 150 | 2026-10-26 | 2026-10-09 |
| 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 |
| Trientine Tetrahydrochloride Administered Once a Day for the First Line Treatment of Wilson's Disease Patients. TETA 4HCl formulation, D-Penicillamine - Wilson's Disease | Phase 3 | Recruiting | Orphalan | 38 | 2026-09-03 | 2026-10-15 |
| A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping AOC 1044, Placebo - Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy) | Phase 3 | Recruiting | Avidity Biosciences, Inc. | 70 | 2026-09-01 | 2026-10-13 |
| Everolimus bAsed caLcineurin inhibiTor frEe immunosuppRession oNe Year AfTer lIver transplantatiON (ALTERNATION) Everolimus, Tacrolimus or Cyclosporine - Liver Allograft, Liver Diseases | Phase 3 | Recruiting | Hannover Medical School | 150 | 2026-08-19 | 2026-10-05 |
| A Study to Learn About the Investigational Drug Rinzimetostat (ORIC-944) in Patients With mCRPC Who Were Previously Treated With Abiraterone Acetate (Himalayas-1) Rinzimetostat, Darolutamide, Enzalutamide - Metastatic Castration Resistant Prostate Cancer | Phase 3 | Recruiting | ORIC Pharmaceuticals | 600 | 2026-08-03 | 2026-11-03 |
| A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome fenfluramine hydrochloride, Placebo - Rett Syndrome | Phase 3 | Recruiting | UCB BIOSCIENCES, Inc. | 200 | 2026-07-14 | 2026-11-02 |
| [64Cu]FBP8 PET for Early Detection of Intracardiac Thrombus in Amyloid Cardiomyopathy [64Cu]FBP8 PET/MR - Cardiac Amyloidosis | Phase 3 | Recruiting | Brigham and Women's Hospital | 20 | 2026-07-07 | 2026-07-21 |
| Pridopidine Phase 3 Study in Huntington's Disease Pridopidine, Placebo - Huntington Disease | Phase 3 | Recruiting | Prilenia | 400 | 2026-07-01 | 2026-11-04 |
| Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO) Zeleciment Rostudirsen (DYNE-251), Placebo - Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne | Phase 3 | Recruiting | Dyne Therapeutics | 90 | 2026-06-01 | 2026-05-27 |
| Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1 zeleciment basivarsen (DYNE-101), Placebo - Myotonic Dystrophy Type 1 (DM1), DM1 | Phase 3 | Recruiting | Dyne Therapeutics | 150 | 2026-05-14 | 2026-10-13 |
| A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease Votoplam (blinded), Placebo - Huntington Disease | Phase 3 | Recruiting | Novartis Pharmaceuticals | 770 | 2026-03-24 | 2026-09-22 |
| Study to Evaluate the Pharmacodynamics, Safety and Efficacy of SKY-0515 in Participants With Huntington's Disease SKY-0515, SKY-0515 Placebo - Huntington Disease | Phase 2 / Phase 3 | Recruiting | Skyhawk Therapeutics, Inc. | 400 | 2026-01-06 | 2026-10-29 |
| Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION) sepofarsen, Placebo IVT - Leber Congenital Amaurosis 10, Blindness | Phase 3 | Recruiting | Laboratoires Thea | 32 | 2025-06-04 | 2026-10-21 |
| Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant Acoramidis, Placebo oral tablet - Amyloidosis, Amyloid Cardiomyopathy | Phase 3 | Recruiting | Eidos Therapeutics, a BridgeBio company | 587 | 2025-05-12 | 2026-08-04 |
| Clinical Trial of Mexiletine Hydrochloride for Spinal and Bulbar Muscular Atrophy Mexiletine hydrochloride, Placebo - Spinal and Bulbar Muscular Atrophy | Phase 2 / Phase 3 | Recruiting | Masahisa Katsuno | 68 | 2025-02-28 | 2025-04-16 |
| The Efficacy and Safety of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2 Mexiletine granules for prolonged-release oral suspension, Placebo - Myotonic Dystrophy | Phase 3 | Recruiting | Lupin Ltd. | 176 | 2025-02-13 | 2026-09-24 |
| Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease) Treatment taken - Steinert's Disease, Myotonic Dystrophy 1 | Phase 3 | Recruiting | Assistance Publique - Hôpitaux de Paris | 142 | 2024-11-29 | 2025-11-24 |
| Treatment With Full-spectrum Cannabis Extract of Refractory Epilepsy Associated With Tuberous Sclerosis Complex (TSC) YCJ-01 Full Spectrum cannbis Extract, Placebo - Tuberous Sclerosis Complex (TSC) | Phase 3 | Recruiting | Oils4Cure | 84 | 2024-06-01 | 2026-02-11 |
| Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With Tuberous Sclerosis Complex Rapamycin, Placebo - Tuberous Sclerosis Complex | Phase 3 | Recruiting | Katarzyna Kotulska | 200 | 2023-01-23 | 2026-10-22 |
Showing 20 of 24. Sign in to see all of them.
Myotonic Dystrophy (6) · Huntington Disease (3) · Tuberous Sclerosis (3) · Amyloid Neuropathies, Familial (2) · Amyloidosis (2) · Bulbo-Spinal Atrophy, X-Linked (2) · Epilepsy (2) · Genetic Diseases, Inborn (2) · Genetic Diseases, X-Linked (2) · Hereditary Sensory and Autonomic Neuropathies (2) · Muscular Dystrophies (2) · Muscular Dystrophy, Duchenne (2) · Neuromuscular Diseases (2) · Optic Atrophy, Hereditary, Leber (2) · Rett Syndrome (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (23) · Nervous System Diseases (23) · Genetic Diseases, Inborn (22) · Neurodegenerative Diseases (22) · Central Nervous System Diseases (10) · Neuromuscular Diseases (10) · Brain Diseases (8) · Metabolic Diseases (7) · Nutritional and Metabolic Diseases (7) · Congenital Abnormalities (6) · Muscular Diseases (5) · Muscular Disorders, Atrophic (5) · Musculoskeletal Diseases (5) · Myotonic Disorders (5)
Sirolimus (2) · CA-19-9 Antigen (1) · Cyclosporine (1) · Docetaxel (1) · Everolimus (1) · Fenfluramine (1) · Fluid Therapy (1) · Mexiletine (1) · Penicillamine (1) · Secretin (1) · Tacrolimus (1) · Vigabatrin (1) · attruby (1) · darolutamide (1) · enzalutamide (1)
Dyne Therapeutics (2) · Katarzyna Kotulska (2) · AMO Pharma Limited (1) · Assistance Publique - Hôpitaux de Paris (1) · Avidity Biosciences, Inc. (1) · Biomed Industries, Inc. (1) · Brigham and Women's Hospital (1) · Eidos Therapeutics, a BridgeBio company (1) · Hannover Medical School (1) · Johns Hopkins University (1) · Laboratoires Thea (1) · Larimar Therapeutics, Inc. (1) · Lupin Ltd. (1) · Masahisa Katsuno (1) · Novartis Pharmaceuticals (1)
United States (15) · Germany (10) · Spain (10) · United Kingdom (9) · Belgium (8) · France (8) · Australia (7) · Italy (7) · Canada (6) · Netherlands (6) · Japan (5) · Poland (5) · Denmark (4) · Argentina (3) · Brazil (3)
Bin Li, MD (1) · David Nguyen, PhD (1) · Harriet Gray-Stephens, BM BCh, MA (Oxon), MFPM (1) · Katarzyna Kotulska-Jozwiak (1) · Lloyd L Tran, PhD (1) · Masahisa Katsuno, PhD, MD (1) · Michael Goggins, MD (1) · Richard Taubert, Professor (1) · Zung V Tran, PhD (1)
PMID 31197699, PMID 31676359, PMID 33161160, PMID 34149034
2025 (5), 2023 (1), R01CA176828 (1), U01CA210170 (1)