1195 completed, 426 recruiting, 278 unknown, 184 terminated. 657 Phase 2, 521 Not applicable, 375 Phase 1, 281 Phase 3, 61 Phase 4.
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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
| 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scAAV1.tMCK.NTF3 for Treatment of CMT1A scAAV1.tMCK.NTF3 - Charcot-Marie-Tooth Neuropathy Type 1A | Phase 1 / Phase 2 | Withdrawn | Nationwide Children's Hospital | 2027-04-01 | 2026-07-29 | |
| Multi-Modal Digital Monitoring of Disease Symptoms Huntington's Disease Huntington Disease | Not yet recruiting | BioSensics | 75 | 2027-01-31 | 2026-10-13 | |
| Assessing the Efficacy of Repeat, Monthly Treatments of Alexandrite Laser for NF1-associated Cutaneous Neurofibromas (cNFs) GentleMax Pro (with skin cooling), GentleMax Pro (without skin cooling) - Neurofibromatosis 1 (NF1), Neurofibromatosis Type I | Not applicable | Suspended | Massachusetts General Hospital | 10 | 2027-01-01 | 2026-06-04 |
| Efficacy of Mirdametinib Alone or Combination With Radiotherapy for Germline and Sporadic NF1-Altered High-Grade Glioma mirdametinib with radiation, mirdametinib - Neurofibromatosis 1 (NF1) | Phase 2 | Not yet recruiting | University of Alabama at Birmingham | 55 | 2027-01-01 | 2026-10-05 |
| SPX-01 Gene Therapy in Pediatric Patients With Spinal Muscular Atrophy Type 1 (RISE) SPX-01 - Spinal Muscular Atrophy (SMA) | Early Phase 1 | Not yet recruiting | Exir Gene Pharmed | 1 | 2027-01-01 | 2026-10-29 |
| Combination of Avutometinib and Defactinib for Treatment of Relapsed, Refractory, Metastatic, or Unresectable Malignant Peripheral Nerve Sheath Tumor avutometinib capsule + defactinib tablet - MPNST (Malignant Peripheral Nerve Sheath Tumor) | Phase 2 | Not yet recruiting | Girish Dhall, MD | 25 | 2027-01-01 | 2026-10-28 |
| Umbilical Cord Mesenchymal Stem Cells Therapy (19#iSCLife®-SA) for Patients With Spinocerebellar Ataxia Intravenous infusion, Intrathecal injection, umbilical cord mesenchymal stem cell - Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2 | Phase 2 | Not yet recruiting | Sclnow Biotechnology Co., Ltd. | 45 | 2026-12-31 | 2026-04-23 |
| Aspirin and PAClitaxel in Head & Neck Squamous Cell Carcinoma Aspirin, Paclitaxel - Head and Neck (HNSCC), Head and Neck Squamous Cell Carcinoma (HNSCC) - Recurrent/Metastatic (R/M) | Phase 2 | Not yet recruiting | Washington University School of Medicine | 40 | 2026-12-31 | 2026-10-28 |
| Multi-Modal Digital Monitoring of Disease Symptoms in Spinocerebellar Ataxia Spinocerebellar Ataxia (SCA) | Not yet recruiting | BioSensics | 25 | 2026-12-15 | 2026-10-01 | |
| 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 |
| Brain Iron in Beta-Propeller Protein Associated Neurodegeneration (BPAN) Standardized brain MRI including specific sequences usable for Quantitative Susceptibility Mapping (QSM) - Neurodegeneration With Brain Iron Accumulation (NBIA), Iron Metabolism Disorders | Not applicable | Not yet recruiting | University Hospital, Bordeaux | 3 | 2026-12-01 | 2026-10-09 |
| 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 |
| Muscle MRI Fat Fraction Before Surgery to Predict Strength Recovery After Foot-Drop Surgery in Charcot-Marie-Tooth Disease Tibialis posterior tendon transfer, Individualized adjunct procedures for cavovarus deformity correction, Ankle-foot orthosis (AFO) - Foot Drop, Charcot-Marie-Tooth Disease Type 1A | Not yet recruiting | Peking University Third Hospital | 100 | 2026-12-01 | 2026-10-26 | |
| Selumetinib for NF2-Related Schwannomatosis Selumetinib - Neurofibromatosis Type 2, NF2 | Phase 2 | Not yet recruiting | Beijing Tiantan Hospital | 20 | 2026-12-01 | 2026-10-28 |
| Safety, Tolerability, and Efficacy of PLX-200 in Patients With CLN3 PLX-200, Placebo - Juvenile Neuronal Ceroid Lipofuscinosis | Phase 3 | Withdrawn | Polaryx Therapeutics, Inc. | 2026-11-01 | 2026-08-19 | |
| Clinical, Electrophysiological and Genetic Characteristics of Hereditary and Inflammatory Polyneuropathies in France Genetic and Inflammatory Polyneuropathies | Not yet recruiting | Assistance Publique - Hôpitaux de Paris | 1000 | 2026-11-01 | 2026-10-01 | |
| To Evaluate the Long-term Safety and Tolerability of Acoramidis in Participants With Newly Diagnosed ATTR-CM (ACT-EARLY OLE) Acoramidis (AG10) - Amyloidosis in Transthyretin (TTR), Amyloidosis, Familial | Phase 3 | Not yet recruiting | Eidos Therapeutics, a BridgeBio company | 250 | 2026-11-01 | 2026-08-11 |
| GV101 for Friedreich's Ataxia (FA) Treatment GV101, Placebo - Friedreich's Ataxia | Phase 2 | Not yet recruiting | Graviton Bioscience Corporation | 48 | 2026-11-01 | 2026-10-15 |
Showing 20 of 2483. Sign in to see all of them.
Huntington Disease (292) · Tourette Syndrome (233) · Amyloid Neuropathies, Familial (186) · Hereditary Sensory and Autonomic Neuropathies (175) · Neurofibromatosis 1 (174) · Myotonic Dystrophy (124) · Fragile X Syndrome (114) · Tuberous Sclerosis (114) · Charcot-Marie-Tooth Disease (112) · Friedreich Ataxia (110) · Rett Syndrome (95) · Spinocerebellar Ataxias (92) · Hepatolenticular Degeneration (79) · Mucopolysaccharidosis II (77) · Tic Disorders (75)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (2479) · Nervous System Diseases (2440) · Genetic Diseases, Inborn (2430) · Neurodegenerative Diseases (2104) · Central Nervous System Diseases (1214) · Brain Diseases (1075) · Neuromuscular Diseases (951) · Nutritional and Metabolic Diseases (748) · Metabolic Diseases (726) · Neurologic Manifestations (712) · Peripheral Nervous System Diseases (710) · Mental Disorders (638) · Basal Ganglia Diseases (624) · Congenital Abnormalities (621)
Everolimus (28) · Sirolimus (24) · AZD 6244 (22) · Blood Specimen Collection (20) · Exercise (14) · idursulfase (14) · idebenone (13) · Transcranial Magnetic Stimulation (12) · Aripiprazole (11) · Cannabidiol (11) · Deep Brain Stimulation (11) · Fulvestrant (11) · alpha-tocotrienol quinone (11) · latrepirdine (11) · pembrolizumab (11)
Assistance Publique - Hôpitaux de Paris (49) · Massachusetts General Hospital (41) · Novartis Pharmaceuticals (41) · National Cancer Institute (NCI) (37) · National Institute of Neurological Disorders and Stroke (NINDS) (31) · Pfizer (29) · NYU Langone Health (26) · AstraZeneca (24) · Children's Hospital Medical Center, Cincinnati (23) · Johns Hopkins University (21) · Institut National de la Santé Et de la Recherche Médicale, France (20) · Shire (17) · University of Florida (17) · University of Rochester (17) · University of Alabama at Birmingham (15)
United States (1270) · France (371) · United Kingdom (275) · Germany (255) · Italy (248) · Canada (229) · Spain (210) · China (144) · Australia (141) · Netherlands (126) · Belgium (101) · Denmark (80) · Poland (74) · Brazil (73) · Japan (64)
Brigitte C Widemann, M.D. (11) · Staci M Peron, Ph.D. (7) · Ana-Maria Vranceanu, PhD (5) · Elizabeth Berry-Kravis, MD, PhD (5) · Eva Kohegyi, MD (5) · Horacio Kaufmann, MD (5)
PMID 36463457, PMID 37460385, PMID 36449262, PMID 38489195, PMID 29480210, PMID 30936738, PMID 32250312, PMID 34108929, PMID 34383289, PMID 40739200, PMID 25803389, PMID 26554876, PMID 28029918, PMID 29023494, PMID 31381524, PMID 32187457, PMID 33395098, PMID 36811812, PMID 37171121, PMID 37460782, PMID 38363467, PMID 39383458, PMID 42389794, PMID 15683544, PMID 17105749, PMID 17562928, PMID 18473686, PMID 19772971, PMID 20483969, PMID 21586746, PMID 21732836, PMID 21779958, PMID 21996156, PMID 22353803, PMID 22868933, PMID 23035069, PMID 24001701, PMID 24144615, PMID 24327518, PMID 24399845, PMID 24780882, PMID 25071871, PMID 25283752, PMID 25519680, PMID 25568297, PMID 25801175, PMID 25988365, PMID 26606867, PMID 26666500, PMID 26714284
2025 (21), 2023 (20), 2018 (19), 2017 (16), 2015 (12), 2019 (11), 2016 (10), 2014 (9), 2009 (5), 2008 (4), U54NS065712 (4), U10CA180886 (3), U54NS048843 (3), U54NS065768 (3), K23HD109375 (2)