15 not yet recruiting. 15 Phase 1, 9 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 15 | Not yet recruiting 15 |
| Phase 2 | 9 | Not yet recruiting 9 |
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 |
|---|---|---|---|---|---|---|
| Calcium Channel Blocker in Myotonic Dystrophy Type 1 Amlodipine - Myotonic Dystrophy 1 | Phase 1 | Not yet recruiting | University of Rochester | 20 | 2026-12-07 | 2026-11-09 |
| A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of ATI-105 in Healthy Adults ATI-105, Placebo - Friedreich Ataxia, Cardiomyopathies | Phase 1 | Not yet recruiting | Adjuvia Therapeutics, Inc. | 60 | 2026-10-26 | 2026-10-09 |
| A Trial of ALN-6457 in Adult Participants With Symptomatic Prion Disease (PrD) ALN-6457 - Prion Disease | Phase 1 / Phase 2 | Not yet recruiting | Regeneron Pharmaceuticals | 60 | 2026-10-23 | 2026-09-22 |
| Safety and Efficacy Study of GEB-101 Injection in Subjects With TGFBI-related Corneal Dystrophy GEB-101 Injection - Hereditary Corneal Dystrophy, Corneal Abnormality | Phase 1 / Phase 2 | Not yet recruiting | GenEditBio Limited | 30 | 2026-10-01 | 2026-09-10 |
| Transcranial Ultrasound Stimulation for Tourette Syndrome Focused ultrasound stimulation, Sham stimulation - Tourette Syndrome | Phase 1 | Not yet recruiting | University of Calgary | 20 | 2026-08-01 | 2026-07-13 |
| Gene Therapy Trial for CLN6 Batten Disease scAAV9.CB.CLN6 (dose: 1.5E14 vector genomes) - CLN6, Batten Disease | Phase 1 / Phase 2 | Not yet recruiting | The Charlotte and Gwenyth Gray Foundation | 12 | 2026-08-01 | 2026-05-14 |
| Luvometinib in Combination With Serplulimab for NF2-Related Tumors Luvometinib, Serplulimab - NF2-related Schwannomatosis, NF2 | Phase 1 / Phase 2 | Not yet recruiting | Beijing Tiantan Hospital | 30 | 2026-07-31 | 2026-07-16 |
| Study to Determine Optimal Dose, Evaluate the Efficacy and Safety of PRG-N-01 in Patients With Neurofibromatosis Type II Trineumin - Neurofibromatosis Type II, NF2 | Phase 1 / Phase 2 | Not yet recruiting | PRG Science & Technology Co., Ltd. | 25 | 2026-07-01 | 2026-01-23 |
| Phase I/II Clinical Study to Evaluate the Safety, Tolerability and Efficacy of LY-M003 Injection in Adult Patients With Wilson's Disease LY-M003 Injection - Wilson's Disease | Phase 1 / Phase 2 | Not yet recruiting | Lingyi Biotech Co., Ltd. | 18 | 2026-06-15 | 2026-07-02 |
| Mass Balance Study of [14C] LPM3770164 in Healthy Participants [14C] LPM3770164 - Huntington Disease | Phase 1 | Not yet recruiting | Luye Pharma Group Ltd. | 8 | 2026-04-30 | 2026-04-08 |
| Pilot Study of the Efficacy of Nicotinamide (Vitamin B3) in Leber's Hereditary Optic Neuropathy Nicotinamide treatment - Leber Hereditary Optic Neuropathy (LHON), Leber's Hereditary Optic Neuropathy (LHON) | Phase 1 | Not yet recruiting | University Hospital, Angers | 13 | 2026-04-01 | 2025-12-18 |
| Phase I/II Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of GC310 Injection in Patients With Wilson's Disease (WD) GC310 - Wilson Disease | Phase 1 / Phase 2 | Not yet recruiting | GeneCradle Inc | 15 | 2025-10-01 | 2025-09-15 |
| Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47) BFB-101 (AAV9-CBh-AP4B1) - HSP, Hereditary Spastic Paraplegia | Phase 1 / Phase 2 | Not yet recruiting | BlackfinBio Ltd | 5 | 2025-08-01 | 2025-04-28 |
| Follow-up Study to Evaluate the Safety and Efficacy of FCN-159 in Pediatric Participants With Neurofibromatosis Type 1 Luvometinib Tablets - Neurofibromatosis 1, Plexiform Neurofibroma | Phase 1 / Phase 2 | Not yet recruiting | Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. | 65 | 2025-06-12 | 2025-06-17 |
| A Single Ascending Dose Study of DT-216P2 in Normal Healthy Participants DT-216P2, Saline - Friedreich Ataxia | Phase 1 | Not yet recruiting | Design Therapeutics, Inc. | 36 | 2025-02-01 | 2025-02-21 |
Central Nervous System Diseases (2) · Friedreich Ataxia (2) · Hepatolenticular Degeneration (2) · Mitochondrial Diseases (2) · Neurofibromatosis 2 (2) · Amyloid Neuropathies, Familial (1) · Cardiomyopathies (1) · Central Nervous System Infections (1) · Corneal Dystrophies, Hereditary (1) · Corneal dystrophy, Thiel-Behnke type (1) · Creutzfeldt-Jakob Syndrome (1) · Dementia (1) · Genetic Diseases, Inborn (1) · Gerstmann-Straussler-Scheinker Disease (1) · Huntington Disease (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (15) · Nervous System Diseases (15) · Genetic Diseases, Inborn (14) · Neurodegenerative Diseases (14) · Metabolic Diseases (8) · Nutritional and Metabolic Diseases (8) · Brain Diseases (7) · Central Nervous System Diseases (6) · Basal Ganglia Diseases (4) · Metabolism, Inborn Errors (4) · Movement Disorders (4) · Neuromuscular Diseases (4) · Cranial Nerve Diseases (3) · Mental Disorders (3)
Amlodipine (1) · Sodium Chloride (1)
Adjuvia Therapeutics, Inc. (1) · Beijing Tiantan Hospital (1) · BlackfinBio Ltd (1) · Design Therapeutics, Inc. (1) · GenEditBio Limited (1) · GeneCradle Inc (1) · Lingyi Biotech Co., Ltd. (1) · Luye Pharma Group Ltd. (1) · PRG Science & Technology Co., Ltd. (1) · Regeneron Pharmaceuticals (1) · Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. (1) · The Charlotte and Gwenyth Gray Foundation (1) · University Hospital, Angers (1) · University of Calgary (1) · University of Rochester (1)
China (6) · United States (4) · Australia (1) · France (1)
Chaohui Yu, PhD (1) · Nester Quezada, MD (1) · Ofer Gronen, MD (1)
2025 (1)