39 terminated. 39 Phase 3, 10 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 39 | Terminated 39 |
| Phase 2 | 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 to Evaluate the Safety and Tolerability of SAGE-718 in Participants With Huntington's Disease SAGE-718 - Huntington's Disease | Phase 3 | Terminated | Supernus Pharmaceuticals, Inc. | 153 | 2022-12-14 | 2025-12-17 |
| 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 |
| Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy Ganaxolone - Tuberous Sclerosis Complex | Phase 3 | Terminated | Marinus Pharmaceuticals | 117 | 2022-05-16 | 2025-10-01 |
| A Study of Nivolumab-relatlimab Fixed-dose Combination Versus Regorafenib or TAS-102 in Participants With Later-lines of Metastatic Colorectal Cancer Nivolumab-relatlimab FDC, Regorafenib, TAS-102 - Colorectal Neoplasms | Phase 3 | Terminated | Bristol-Myers Squibb | 769 | 2022-04-28 | 2026-08-26 |
| Efficacy Evaluation of Enobosarm Monotherapy in Treatment of AR+/ER+/HER2- Metastatic Breast Cancer Enobosarm, Exemestane - Metastatic Breast Cancer | Phase 3 | Terminated | Veru Inc. | 52 | 2021-10-12 | 2024-01-26 |
| 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 |
| Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease ALXN1840, Standard of Care - Wilson Disease | Phase 3 | Terminated | Alexion Pharmaceuticals, Inc. | 40 | 2021-09-13 | 2024-10-23 |
| Assessment of Adjunctive Cannabidiol Oral Solution (GWP42003-P) in Children With Tuberous Sclerosis Complex (TSC), Dravet Syndrome (DS), or Lennox-Gastaut Syndrome (LGS) Who Experience Inadequately-controlled Seizures GWP42003-P - Seizure in Participants With Tuberous Sclerosis Complex, Seizure in Participants With Dravet Syndrome | Phase 3 | Terminated | Jazz Pharmaceuticals | 3 | 2021-05-19 | 2025-10-31 |
| Open-Label Extension Study of Trofinetide for Rett Syndrome trofinetide - Rett Syndrome | Phase 3 | Terminated | ACADIA Pharmaceuticals Inc. | 77 | 2020-11-08 | 2024-09-27 |
| A Long-term Study of NPC-12G Gel in Neurofibromatosis Type I NPC-12G gel - Neurofibromatosis | Phase 3 | Terminated | Nobelpharma | 100 | 2020-07-08 | 2022-05-24 |
| A Long-term Safety Study of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome GWP42003-P - Rett Syndrome, RTT | Phase 3 | Terminated | Jazz Pharmaceuticals | 21 | 2020-02-28 | 2022-08-08 |
| Efficacy and Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome GWP42003-P, Placebo - Rett Syndrome, RTT | Phase 3 | Terminated | Jazz Pharmaceuticals | 29 | 2019-07-29 | 2022-09-02 |
| Study of Safety and Efficacy of Brolucizumab 6 mg Dosed Every 4 Weeks Compared to Aflibercept 2 mg Dosed Every 4 Weeks in Patients With Retinal Fluid Despite Frequent Anti-VEGF Injections Brolucizumab, Aflibercept - Age-Related Macular Degeneration | Phase 3 | Terminated | Novartis Pharmaceuticals | 535 | 2018-10-30 | 2023-01-30 |
| Evaluation of Safety and Tolerability of Long-term TEV-50717 (Deutetrabenazine) for Treatment of Tourette Syndrome in Children and Adolescents TEV-50717, Placebo - Tourette Syndrome | Phase 3 | Terminated | Teva Branded Pharmaceutical Products R&D, Inc. | 228 | 2018-05-25 | 2021-11-09 |
| Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Disease ALXN1840, SoC Therapy - Wilson Disease | Phase 3 | Terminated | Alexion Pharmaceuticals, Inc. | 214 | 2018-02-22 | 2024-06-17 |
| Study of the Molecular Features of Postmenopausal Women With HR+ HER2-negative aBC on First-line Treatment With Ribociclib and Letrozole and, in Patients With a PIK3CA Mutation, on Second-line Treatment With Alpelisib Plus Fulvestrant Ribociclib, Letrozole, Alpelisib - Breast Cancer | Phase 3 | Terminated | Novartis Pharmaceuticals | 287 | 2018-02-02 | 2026-07-10 |
| Open-Label Rollover Study of PBI 4050 in Subjects With Alström Syndrome PBI-4050 - Alström Syndrome | Phase 2 / Phase 3 | Terminated | Liminal BioSciences Ltd. | 10 | 2017-10-09 | 2020-11-03 |
| Efficacy and Safety Study of Fosmetpantotenate (RE-024) in PKAN Participants Fosmetpantotenate, Placebo - Pantothenate Kinase-Associated Neurodegeneration | Phase 3 | Terminated | Travere Therapeutics, Inc. | 84 | 2017-07-17 | 2021-01-26 |
| Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms Sarizotan low dose, Sarizotan high dose, Placebo - Rett Syndrome | Phase 2 / Phase 3 | Terminated | Newron Pharmaceuticals SPA | 129 | 2016-10-26 | 2021-12-21 |
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Hereditary Sensory and Autonomic Neuropathies (6) · Rett Syndrome (5) · Huntington Disease (4) · Breast Neoplasms (3) · Adrenoleukodystrophy (2) · Arthritis (2) · Fragile X Syndrome (2) · Hepatolenticular Degeneration (2) · Mucopolysaccharidosis I (2) · Mucopolysaccharidosis II (2) · Mucopolysaccharidosis VI (2) · Neurofibromatoses (2) · Neurofibromatosis 2 (2) · Osteoarthritis (2) · Sudden Infant Death (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (39) · Nervous System Diseases (39) · Genetic Diseases, Inborn (38) · Neurodegenerative Diseases (29) · Central Nervous System Diseases (18) · Brain Diseases (16) · Neurologic Manifestations (14) · Genetic Diseases, X-Linked (13) · Neoplasms (13) · Congenital Abnormalities (11) · Intellectual Disability (11) · Neurobehavioral Manifestations (11) · X-Linked Intellectual Disability (11) · Neuromuscular Diseases (10)
Cannabidiol (3) · Fulvestrant (2) · tanezumab (2) · trofinetide (2) · Alpelisib (1) · Anti-Inflammatory Agents, Non-Steroidal (1) · Aspirin (1) · BID protein, human (1) · Creatine (1) · Glucose (1) · Growth Hormone (1) · HDAC-42 (1) · Human Growth Hormone (1) · Imatinib Mesylate (1) · Lamotrigine (1)
Novartis Pharmaceuticals (5) · Jazz Pharmaceuticals (3) · ACADIA Pharmaceuticals Inc. (2) · Alexion Pharmaceuticals, Inc. (2) · Massachusetts General Hospital (2) · Pfizer (2) · Aldagen (1) · Assistance Publique - Hôpitaux de Paris (1) · Boehringer Ingelheim (1) · Bristol-Myers Squibb (1) · Emalex Biosciences Inc. (1) · Erasmus Medical Center (1) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (1) · Liminal BioSciences Ltd. (1) · Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center (1)
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PMID 30936738, PMID 35747889, PMID 37460385, PMID 23707270, PMID 24625625, PMID 26554876, PMID 26855682, PMID 27913695, PMID 28701493, PMID 29223745, PMID 30103342, PMID 30850442, PMID 31055958, PMID 33862302, PMID 37460782, PMID 39340758, PMID 40043705, PMID 40121838, PMID 42481172
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