72 terminated. 72 Phase 2, 16 Phase 1, 10 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 72 | Terminated 72 |
| Phase 1 | 16 | Terminated 16 |
| Phase 3 | 10 | Terminated 10 |
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 |
|---|---|---|---|---|---|---|
| A Study Investigating the Efficacy and Safety of BGB-45035 Versus Placebo in Adults With Moderate to Severe Active Rheumatoid Arthritis BGB-45035, Placebo - Rheumatoid Arthritis | Phase 2 | Terminated | BeiGene | 49 | 2025-08-21 | 2026-05-28 |
| Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND1 Mutations NFS-02 Injection - Leber Hereditary Optic Neuropathy (LHON) | Phase 1 / Phase 2 | Terminated | Neurophth Therapeutics Inc | 11 | 2023-08-15 | 2024-09-20 |
| A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN) SBT101 Treatment - AMN, AMN Gene Mutation | Phase 1 / Phase 2 | Terminated | SwanBio Therapeutics, Inc. | 9 | 2022-11-17 | 2026-08-26 |
| Efficacy and Safety of REC-2282 in Patients With Progressive Neurofibromatosis Type 2 (NF2) Mutated Meningiomas REC-2282, Placebo - Neurofibromatosis Type 2 | Phase 2 / Phase 3 | Terminated | Recursion Pharmaceuticals Inc. | 25 | 2022-06-20 | 2026-08-17 |
| STRIDES - a Clinical Research Study of an Investigational New Drug to Treat Spinocerebellar Ataxia SLS-005, Placebo - Spinocerebellar Ataxia Type 3 | Phase 2 / Phase 3 | Terminated | Seelos Therapeutics, Inc. | 23 | 2022-06-03 | 2024-04-24 |
| Atomoxetine in Patients With Tourette's Syndrome Atomoxetine - Tourette Syndrome | Phase 2 | Terminated | Poitiers University Hospital | 5 | 2022-05-31 | 2024-01-23 |
| Study of Safety and Efficacy of Dapagliflozin + Metformin XR Versus Metformin XR in Participants With HR+, HER2-, Advanced Breast Cancer While on Treatment With Alpelisib and Fulvestrant Alpelisib, Fulvestrant, Metformin XR - Breast Cancer | Phase 2 | Terminated | Novartis Pharmaceuticals | 2 | 2022-04-06 | 2024-10-09 |
| Apalutamide Plus Cetrelimab in Patients With Treatment-Emergent Small Cell Neuroendocrine Prostate Cancer Apalutamide, Cetrelimab - Small Cell Neuroendocrine Carcinoma, Prostate Cancer | Phase 2 | Terminated | Rahul Aggarwal | 2 | 2022-03-16 | 2025-05-15 |
| A Dose Range Finding Study With Open-Label Extension to Evaluate the Safety of Oral LMI070/Branaplam in Early Manifest Huntington's Disease Branaplam, Placebo - Early Manifest Huntington Disease | Phase 2 | Terminated | Novartis Pharmaceuticals | 26 | 2021-12-08 | 2025-05-16 |
| An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome Trofinetide - Rett Syndrome | Phase 2 / Phase 3 | Terminated | ACADIA Pharmaceuticals Inc. | 15 | 2021-09-22 | 2024-09-24 |
| A Clinical Trial to Evaluate the Safety and Efficacy of AL2846 Capsules in Chinese Patients With Type I Neurofibromatosis AL2846 capsules - Neurofibromatosis and Malignant Peripheral Nerve Sheath Tumors | Phase 1 / Phase 2 | Terminated | Chia Tai Tianqing Pharmaceutical Group Co., Ltd. | 22 | 2021-09-07 | 2024-03-21 |
| Exploratory Study of Danicamtiv in Patients With Primary Dilated Cardiomyopathy (DCM) Due to Genetic Variants or Other Causalities danicamtiv - Primary Familial Dilated Cardiomyopathy | Phase 2 | Terminated | Bristol-Myers Squibb | 41 | 2020-08-04 | 2025-05-21 |
| Phase 2 Study to Determine if RTB101 Reduces the Severity of COVID-19 in Older Adults Residing in Nursing Homes RTB101, Placebo - COVID19 | Phase 2 | Terminated | Restorbio Inc. | 36 | 2020-07-11 | 2021-02-10 |
| Open-label Extension Study to Evaluate the Safety and Tolerability of WVE-120101 in Patients With Huntington's Disease WVE-120101 - Huntington Disease | Phase 1 / Phase 2 | Terminated | Wave Life Sciences USA, Inc. | 27 | 2020-04-13 | 2022-02-09 |
| Open-label Extension Study to Evaluate the Safety and Tolerability of WVE-120102 in Patients With Huntington's Disease WVE-120102 - Huntington Disease | Phase 1 / Phase 2 | Terminated | Wave Life Sciences USA, Inc. | 36 | 2019-09-24 | 2022-02-09 |
| Within Subject Crossover Study of Cognitive Effects of Neflamapimod in Early-Stage Huntington Disease neflamapimod, Placebo - Huntington Disease | Phase 2 | Terminated | EIP Pharma Inc | 15 | 2019-07-08 | 2022-04-06 |
| Extension Study to Study PQ-110-001 (NCT03140969) QR-110 - Leber Congenital Amaurosis 10, Blindness | Phase 1 / Phase 2 | Terminated | Laboratoires Thea | 9 | 2019-05-13 | 2024-10-15 |
| Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX) ACE-083 - Facioscapulohumeral Muscular Dystrophy, Charcot-Marie-Tooth Disease | Phase 2 | Terminated | Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA | 62 | 2019-05-10 | 2022-09-26 |
| Synaptic Plasticity and Cognitive Function in RASopathies Lovastatin, Lamotrigine - Impaired Synaptic Plasticity, Impaired Cognition | Phase 2 | Terminated | Technical University of Munich | 16 | 2019-03-22 | 2023-11-30 |
| Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3 Amifampridine Phosphate 10 MG Oral Tablet - Spinal Muscular Atrophy Type 3 | Phase 2 | Terminated | Catalyst Pharmaceuticals, Inc. | 13 | 2019-03-07 | 2023-11-30 |
Showing 20 of 72. Sign in to see all of them.
Hereditary Sensory and Autonomic Neuropathies (12) · Huntington Disease (11) · Neurofibromatosis 1 (6) · Bulbo-Spinal Atrophy, X-Linked (4) · Neurofibromatoses (4) · Neurofibromatosis 2 (4) · Optic Atrophy, Hereditary, Leber (4) · Tourette Syndrome (4) · Adrenoleukodystrophy (3) · Breast Neoplasms (3) · Charcot-Marie-Tooth Disease (3) · Fragile X Syndrome (3) · Mucopolysaccharidosis II (3) · Neurofibroma, Plexiform (3) · Prostatic Neoplasms (3)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (72) · Nervous System Diseases (71) · Genetic Diseases, Inborn (70) · Neurodegenerative Diseases (61) · Neuromuscular Diseases (34) · Central Nervous System Diseases (30) · Peripheral Nervous System Diseases (27) · Neoplasms (25) · Brain Diseases (23) · Congenital Abnormalities (23) · Neoplasms by Histologic Type (19) · Nervous System Malformations (18) · Neurologic Manifestations (18) · Mental Disorders (17)
Alpelisib (3) · mavoglurant (3) · 4-phenylbutyric acid (2) · Everolimus (2) · Fulvestrant (2) · Lamotrigine (2) · valbenazine (2) · AZD 6244 (1) · Amifampridine (1) · Aminolevulinic Acid (1) · Atomoxetine Hydrochloride (1) · Cetuximab (1) · Cholecalciferol (1) · Cysteamine (1) · Dasatinib (1)
Novartis Pharmaceuticals (9) · Wave Life Sciences USA, Inc. (4) · Pfizer (3) · Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA (2) · Hugo W. Moser Research Institute at Kennedy Krieger, Inc. (2) · Neurocrine Biosciences (2) · Westat (2) · ACADIA Pharmaceuticals Inc. (1) · Amgen (1) · ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA) (1) · Aucta Pharmaceuticals, Inc (1) · BeiGene (1) · Bristol-Myers Squibb (1) · Catalyst Pharmaceuticals, Inc. (1) · Chia Tai Tianqing Pharmaceutical Group Co., Ltd. (1)
United States (52) · United Kingdom (17) · Germany (16) · France (14) · Australia (12) · Canada (12) · Spain (10) · Italy (8) · Denmark (7) · Netherlands (7) · Poland (6) · Belgium (5) · China (5) · Switzerland (3) · Austria (2)
Albert Assad, MD (1) · Chie-Schin Shih, MD (1) · Daniel George, MD (1) · David H. Viskochil, MD, PhD (1) · Donald G Basel, MD (1) · Dusica Babovic-Vuksanovic (1) · Ezra Cohen, MD (1)
PMID 29480210, PMID 32250312, PMID 25689146, PMID 26855682, PMID 30572891, PMID 33639990, PMID 33862302, PMID 33974419, PMID 35110415, PMID 35545446, PMID 36040329, PMID 36299240, PMID 36394867, PMID 36463457, PMID 37280688, PMID 37460385, PMID 37875771, PMID 38290135, PMID 38372904, PMID 38489195, PMID 39340758, PMID 40043705, PMID 40121838, PMID 40216220, PMID 40713644, PMID 41217321, PMID 41491108, PMID 42389794, PMID 42481172
2016 (2), 2017 (2), 2018 (2), 2019 (2), N01NS42361 (2), 2009 (1), 2014 (1), 2015 (1), N01CM17104 (1), N01CM62205 (1), P30CA015083 (1)