142 completed. 142 Phase 3, 34 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 142 | Completed 142 |
| Phase 2 | 34 | Completed 34 |
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 |
|---|---|---|---|---|---|---|
| REVEAL - Research With I-124 EVuzamitide to Elucidate Cardiac AmyLoidosis I-124 evuzamitide - Cardiac Amyloidosis | Phase 3 | Completed | Sharmila Dorbala | 204 | 2025-01-14 | 2026-04-30 |
| Global Study of Del-desiran for the Treatment of DM1 AOC 1001 (del-desiran), Placebo - DM1, Myotonic Dystrophy | Phase 3 | Completed | Avidity Biosciences, Inc. | 159 | 2024-05-30 | 2026-09-01 |
| To Study the Efficacy & Safety of Oral NA-921 (Bionetide) in Girls and Women with Rett Syndrome Drug: Bionetide, Placebo - Rett Syndrome | Phase 2 / Phase 3 | Completed | Biomed Industries, Inc. | 187 | 2024-03-15 | 2025-02-27 |
| Ecopipam Tablets to Study Tourette's Disorder in Children, Adolescents and Adults Ecopipam Hydrochloride - Tourette Disorder | Phase 3 | Completed | Emalex Biosciences Inc. | 216 | 2023-01-31 | 2026-06-12 |
| Efficacy of [18F]Florbetaben PET for Diagnosis of Cardiac AL Amyloidosis [18F]florbetaben - Cardiac Amyloidosis, AL Amyloidosis | Phase 3 | Completed | Lantheus Germany GmbH | 244 | 2023-01-13 | 2026-10-26 |
| Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam OAV101 - Spinal Muscular Atrophy | Phase 3 | Completed | Novartis Pharmaceuticals | 27 | 2023-01-12 | 2026-01-13 |
| A Study of BPN14770 in Male Adults (Aged 18 to 45) With Fragile X Syndrome BPN14770/ zatolmilast, Placebo - Fragile X Syndrome | Phase 3 | Completed | Tetra Discovery Partners | 171 | 2022-11-01 | 2026-07-10 |
| Clinical Study of NPC-12Y Gel in Patients With Skin Lesions Associated With TSC NPC-12Y gel, NPC-12Y placebo gel - Tuberous Sclerosis Complex | Phase 3 | Completed | Nobelpharma | 43 | 2022-06-01 | 2024-11-22 |
| Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam Apitegromab, Placebo - Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3 | Phase 3 | Completed | Scholar Rock, Inc. | 188 | 2022-04-14 | 2026-01-22 |
| TEsting METformin Against Cognitive Decline in HD Metformin, Placebo - Huntington Disease | Phase 3 | Completed | Instituto de Investigacion Sanitaria La Fe | 60 | 2022-04-11 | 2025-09-25 |
| Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy Ganaxolone, Placebo - Tuberous Sclerosis Complex | Phase 3 | Completed | Marinus Pharmaceuticals | 129 | 2022-04-01 | 2025-07-11 |
| A Randomized Study of BPN14770 in Male Adolescents (Aged 9 to < 18 Years) With Fragile X Syndrome zatolmilast, Placebo - Fragile X Syndrome | Phase 2 / Phase 3 | Completed | Tetra Discovery Partners | 163 | 2022-03-29 | 2026-08-31 |
| Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA) OAV101, Sham control - Type 2 Spinal Muscular Atrophy | Phase 3 | Completed | Novartis Pharmaceuticals | 126 | 2022-02-01 | 2026-01-13 |
| Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults With Fragile X Syndrome ZYN002, Placebo - Fragile X Syndrome, Fragile X Syndrome (FXS) | Phase 3 | Completed | Harmony Biosciences Management, Inc. | 257 | 2021-09-13 | 2026-09-01 |
| Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) OAV101 - Spinal Muscular Atrophy | Phase 3 | Completed | Novartis Pharmaceuticals | 24 | 2021-09-08 | 2024-10-09 |
| Open Label Study in Adolescents and Children With Myotonic Disorders Mexiletine - Myotonic Dystrophy | Phase 3 | Completed | Lupin Ltd. | 12 | 2021-09-03 | 2024-11-25 |
| Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy Tideglusib, Placebo - Congenital Myotonic Dystrophy | Phase 2 / Phase 3 | Completed | AMO Pharma Limited | 56 | 2021-03-03 | 2025-10-08 |
| A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia Vatiquinone, Placebo - Friedreich Ataxia | Phase 2 / Phase 3 | Completed | PTC Therapeutics | 146 | 2020-12-17 | 2026-04-13 |
| PRidopidine's Outcome On Function in Huntington Disease, PROOF- HD Pridopidine, Placebo - Huntington Disease | Phase 3 | Completed | Prilenia | 499 | 2020-10-16 | 2025-03-12 |
| Open-Label Rollover Study for Continuing Valbenazine Administration for the Treatment of Chorea Associated With Huntington Disease Valbenazine - Chorea, Huntington | Phase 3 | Completed | Neurocrine Biosciences | 154 | 2020-09-18 | 2026-06-17 |
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Tourette Syndrome (18) · Huntington Disease (15) · Amyloid Neuropathies, Familial (14) · Amyloidosis (11) · Friedreich Ataxia (11) · Tuberous Sclerosis (11) · Tic Disorders (9) · Fragile X Syndrome (8) · Mucopolysaccharidosis II (8) · Spinocerebellar Ataxias (8) · Adrenoleukodystrophy (7) · Alzheimer Disease (6) · Myotonic Dystrophy (6) · Rett Syndrome (6) · Muscular Atrophy, Spinal (5)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (142) · Genetic Diseases, Inborn (139) · Nervous System Diseases (138) · Neurodegenerative Diseases (110) · Central Nervous System Diseases (79) · Brain Diseases (72) · Neurologic Manifestations (59) · Nutritional and Metabolic Diseases (51) · Metabolic Diseases (50) · Mental Disorders (42) · Neuromuscular Diseases (37) · Basal Ganglia Diseases (36) · Movement Disorders (36) · Pathological Conditions, Signs and Symptoms (35)
Aripiprazole (7) · deutetrabenazine (4) · idebenone (4) · tafamidis (4) · Riluzole (3) · Zolgensma (3) · idursulfase (3) · interferon gamma-1b (3) · Ascorbic Acid (2) · BPN14770 (2) · Cannabidiol (2) · Deferiprone (2) · Everolimus (2) · Inotersen (2) · Isoflurophate (2)
Novartis Pharmaceuticals (6) · Pfizer (6) · Assistance Publique - Hôpitaux de Paris (5) · Kissei Pharmaceutical Co., Ltd. (5) · Otsuka Pharmaceutical Development & Commercialization, Inc. (5) · GenSight Biologics (4) · Nobelpharma (4) · Santhera Pharmaceuticals (4) · Shire (4) · Teva Branded Pharmaceutical Products R&D, Inc. (4) · Alnylam Pharmaceuticals (3) · Amgen (3) · ACADIA Pharmaceuticals Inc. (2) · Anavex Life Sciences Corp. (2) · ApoPharma (2)
United States (89) · United Kingdom (46) · France (45) · Germany (42) · Canada (39) · Italy (37) · Spain (36) · Australia (25) · Netherlands (21) · Japan (19) · Brazil (17) · Belgium (14) · South Korea (14) · Sweden (12) · Taiwan (12)
Eva Kohegyi, MD (5) · David Lynch, MD (2) · Elliott Vichinsky, MD (2) · Jeff Packman (2) · Joseph Jankovic, MD (2) · Nancy Newman, MD (2) · Paul Orchard, MD (2) · Paul Wang, M.D. (2)
PMID 36449262, PMID 34108929, PMID 21779958, PMID 26666500, PMID 27494299, PMID 28265459, PMID 29023494, PMID 30053159, PMID 30558645, PMID 30936738, PMID 31353964, PMID 31937586, PMID 32107748, PMID 32524297, PMID 33070419, PMID 33395098, PMID 33461041, PMID 34355354, PMID 34923848, PMID 34957550, PMID 35799473, PMID 35961250, PMID 36715498, PMID 37434378, PMID 37460385, PMID 37460782, PMID 37915038, PMID 37943223, PMID 38548524, PMID 38557959, PMID 38932583, PMID 39383458, PMID 39825184, PMID 40488446, PMID 42658466, PMID 15492353, PMID 15965209, PMID 18678823, PMID 19064745, PMID 20021666, PMID 20483969, PMID 20697044, PMID 21078496, PMID 21150784, PMID 21392622, PMID 21555779, PMID 21586746, PMID 22071279, PMID 22578232, PMID 22929184
2015 (5), 2016 (4), 2017 (4), 2018 (4), 2014 (2), 2004 (1), 2008 (1), 2023 (1), R01MH070802 (1), R01NS051306 (1)