191 completed, 52 recruiting, 41 active not recruiting, 37 terminated. 375 Phase 1, 167 Phase 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 |
|---|---|---|---|---|---|---|
| Phase I/IIa Trial of scAAV1.tMCK.NTF3 for Treatment of CMT1A scAAV1.tMCK.NTF3 - Charcot-Marie-Tooth Neuropathy Type 1A | Phase 1 / Phase 2 | Withdrawn | Nationwide Children's Hospital | 2027-04-01 | 2026-07-29 | |
| Calcium Channel Blocker in Myotonic Dystrophy Type 1 Amlodipine - Myotonic Dystrophy 1 | Phase 1 | Not yet recruiting | University of Rochester | 20 | 2026-12-07 | 2025-12-09 |
| 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 |
| A Study of the Safety and Efficacy of Prime Editing (PM577) in Participants With Wilson Disease (WD) PM577a - Wilson Disease, Wilson's Disease | Phase 1 / Phase 2 | Recruiting | Prime Medicine, Inc. | 42 | 2026-09-01 | 2026-10-01 |
| NORTHERA (DROXIDOPA) for Dysautonomia in Pediatric Survivors of Menkes Disease Droxidopa Oral Product, Placebo Control - Menkes Disease | Phase 1 / Phase 2 | Recruiting | Stephen G. Kaler | 6 | 2026-08-10 | 2026-10-05 |
| 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 |
| A Study to Evaluate ARV-6723 Alone and With Pembrolizumab in Participants With Advanced Solid Tumors ARV-6723, Pembrolizumab, SOC - Advanced Solid Tumor | Phase 1 / Phase 2 | Recruiting | Arvinas Inc. | 499 | 2026-07-29 | 2026-10-13 |
| A Phase I/II Clinical Study to Evaluate SKG0201 Injection in Subjects With Spinal Muscular Atrophy Type I SKG0201 Injection - Spinal Muscular Atrophy 1 | Phase 1 / Phase 2 | Recruiting | Lanyue Biotech (Hangzhou) Co., Ltd. | 11 | 2026-07-15 | 2026-10-14 |
| Safety, Tolerability, and Pharmacokinetics of SVG103 (Paxalisib) in Focal Cortical Dysplasia Type II (FCD-II), Tuberous Sclerosis Complex (TSC) or Hemimegalencephaly (HME) SVG103 - Focal Cortical Dysplasia, Tuberous Sclerosis Complex (TSC) | Phase 1 / Phase 2 | Recruiting | Sovargen | 12 | 2026-07-14 | 2026-08-10 |
| 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 |
| Study of Cabozantinib With Selumetinib for Plexiform Neurofibromas Cabozantinib Oral Tablet, Selumetinib Oral Capsule - Neurofibromatosis 1, Plexiform Neurofibroma | Phase 1 | Recruiting | Girish Dhall, MD | 30 | 2026-06-09 | 2026-10-02 |
| Early Phase Study Evaluating MEK and MDM2 Inhibition in Patients With NF1 and MPNST APG-115, Selumetinib - Malignant Peripheral Nerve Sheath Tumor (MPNST), Neurofibromatosis 1 (NF1) | Phase 1 / Phase 2 | Recruiting | AeRang Kim | 45 | 2026-06-01 | 2026-07-27 |
| PrP-targeting siRNA Safety & Mechanism Study PrP-siRNA - Prion Disease | Phase 1 | Recruiting | Broad Institute of MIT and Harvard | 45 | 2026-05-15 | 2026-10-16 |
| A First-in-human Study of the Effects of SRP-1005 in Participants With Huntington's Disease SRP-1005, Placebo - Huntington's Disease | Phase 1 | Recruiting | Sarepta Therapeutics, Inc. | 32 | 2026-05-12 | 2026-10-19 |
| 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 |
Showing 20 of 375. Sign in to see all of them.
Huntington Disease (57) · Hereditary Sensory and Autonomic Neuropathies (55) · Neurofibromatosis 1 (30) · Friedreich Ataxia (22) · Mucopolysaccharidosis II (20) · Neuronal Ceroid-Lipofuscinoses (17) · Fragile X Syndrome (16) · Tuberous Sclerosis (16) · Bulbo-Spinal Atrophy, X-Linked (15) · Tourette Syndrome (15) · Myotonic Dystrophy (13) · Colorectal Neoplasms (12) · Alzheimer Disease (11) · Breast Neoplasms (11) · Hepatolenticular Degeneration (11)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (375) · Nervous System Diseases (369) · Genetic Diseases, Inborn (365) · Neurodegenerative Diseases (314) · Central Nervous System Diseases (168) · Neuromuscular Diseases (145) · Brain Diseases (143) · Nutritional and Metabolic Diseases (114) · Peripheral Nervous System Diseases (110) · Metabolic Diseases (109) · Congenital Abnormalities (98) · Neoplasms (98) · Basal Ganglia Diseases (82) · Mental Disorders (81)
AZD 6244 (9) · Everolimus (8) · latrepirdine (8) · Fulvestrant (6) · pembrolizumab (6) · Cetuximab (5) · Sirolimus (5) · Deoxycholic Acid (4) · 6-chloro-2,3,4,9-tetrahydro-1H-carbazole-1-carboxamide (3) · Injections (3) · PX-866 (3) · Prednisone (3) · cerliponase alfa (3) · deutetrabenazine (3) · idebenone (3)
AstraZeneca (11) · Pfizer (10) · National Cancer Institute (NCI) (9) · Children's Hospital Medical Center, Cincinnati (7) · Massachusetts General Hospital (6) · National Institute of Neurological Disorders and Stroke (NINDS) (5) · Novartis Pharmaceuticals (5) · Wave Life Sciences USA, Inc. (5) · Alexion Pharmaceuticals, Inc. (4) · Design Therapeutics, Inc. (4) · Luye Pharma Group Ltd. (4) · Stanford University (4) · University of Rochester (4) · Weill Medical College of Cornell University (4) · Cascadian Therapeutics Inc. (3)
United States (249) · United Kingdom (53) · Canada (38) · Australia (35) · China (32) · Germany (32) · France (30) · Spain (29) · Italy (17) · Netherlands (15) · Japan (12) · South Korea (11) · Denmark (9) · Poland (9) · Belgium (8)
Brigitte C Widemann, M.D. (4) · Richard R Anderson, MD (3) · David Russell, MD (2) · Harry T Whelan, MD (2) · Jason R Fangusaro (2) · Joanne Kurtzberg, MD (2) · Ken Nedd, M.D. (2)
PMID 29480210, PMID 32250312, PMID 36463457, PMID 37171121, PMID 38489195, PMID 40739200, PMID 17562928, PMID 29688815, PMID 33980287, PMID 38101904, PMID 38507752, PMID 38776275, PMID 42307907, PMID 11231629, PMID 11399225, PMID 12473512, PMID 15923132, PMID 16278898, PMID 17482283, PMID 18256395, PMID 18473686, PMID 18625864, PMID 18752978, PMID 19194885, PMID 19892975, PMID 20497190, PMID 20652413, PMID 20837825, PMID 21047224, PMID 21482669, PMID 21764821, PMID 22134099, PMID 22262746, PMID 22934754, PMID 23151076, PMID 23325902, PMID 23497636, PMID 23581634, PMID 23798472, PMID 23845948, PMID 23966770, PMID 24296492, PMID 24327173, PMID 24398093, PMID 24457989, PMID 24878825, PMID 25223836, PMID 25281031, PMID 25342621, PMID 26336641
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