6 enrolling by invitation. 6 Phase 3, 2 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 6 | Enrolling by invitation 6 |
| Phase 2 | 2 | Enrolling by invitation 2 |
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 |
|---|---|---|---|---|---|---|
| An Extension of SKY-0515 in Participants With Huntington's Disease SKY-0515 - Huntington's Disease (HD) | Phase 2 / Phase 3 | Enrolling by invitation | Skyhawk Therapeutics, Inc. | 500 | 2026-04-10 | 2026-07-24 |
| An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2 Who Have Completed MEX-DM-302 Study. Mexiletine granules for prolonged-release oral suspension - Myotonic Dystrophy | Phase 3 | Enrolling by invitation | Lupin Ltd. | 176 | 2025-09-12 | 2026-01-26 |
| Global Open-Label Extension Study of Del-desiran for the Treatment of DM1 Del-desiran (AOC 1001) - Myotonic Dystrophy Type 1, DM1 | Phase 3 | Enrolling by invitation | Avidity Biosciences, Inc. | 199 | 2025-06-26 | 2026-08-19 |
| Long-term Study to Evaluate Safety and Tolerability of Valbenazine in Participants With Chorea Associated With Huntington Disease in Canada Valbenazine - Chorea, Huntington | Phase 3 | Enrolling by invitation | Neurocrine Biosciences | 7 | 2024-04-24 | 2026-06-18 |
| An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007 tividenofusp alfa - Mucopolysaccharidosis II | Phase 2 / Phase 3 | Enrolling by invitation | Denali Therapeutics Inc. | 99 | 2023-09-20 | 2026-10-27 |
| An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subjects JR-141 - Mucopolysaccharidosis II | Phase 3 | Enrolling by invitation | JCR Pharmaceuticals Co., Ltd. | 80 | 2023-02-22 | 2026-01-21 |
Huntington Disease (2) · Mucopolysaccharidosis II (2) · Myotonic Dystrophy (2) · Myotonia (1) · Myotonic Disorders (1) · Sudden Infant Death (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (6) · Genetic Diseases, Inborn (6) · Nervous System Diseases (6) · Neurodegenerative Diseases (4) · Neurologic Manifestations (3) · Basal Ganglia Diseases (2) · Brain Diseases (2) · Carbohydrate Metabolism, Inborn Errors (2) · Central Nervous System Diseases (2) · Chorea (2) · Cognition Disorders (2) · Connective Tissue Diseases (2) · Dementia (2) · Dyskinesias (2)
valbenazine (1)
Avidity Biosciences, Inc. (1) · Denali Therapeutics Inc. (1) · JCR Pharmaceuticals Co., Ltd. (1) · Lupin Ltd. (1) · Neurocrine Biosciences (1) · Skyhawk Therapeutics, Inc. (1)
Germany (4) · Italy (4) · Spain (4) · Canada (3) · France (3) · United Kingdom (3) · United States (3) · Argentina (2) · Belgium (2) · Brazil (2) · Denmark (2) · Netherlands (2) · Australia (1) · Czechia (1) · Japan (1)
2023 (1)