348 completed. 348 Phase 2, 65 Phase 1, 34 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 348 | Completed 348 |
| Phase 1 | 65 | Completed 65 |
| Phase 3 | 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 |
|---|---|---|---|---|---|---|
| Patient Preference Study: Standard of Care Versus Once-daily Trientine Tetrahydrochloride New TETA 4HCl Formulation, Standard of Care - Wilson's Disease | Phase 2 | Completed | Orphalan | 10 | 2025-07-15 | 2026-04-06 |
| Tuvusertib Combined With Niraparib or Lartesertib in Participants With Epithelial Ovarian Cancer (DDRiver EOC 302) Tuvusertib (M1774), Niraparib, Lartesertib (M4076) - Ovarian Cancer | Phase 2 | Completed | EMD Serono Research & Development Institute, Inc. | 63 | 2024-10-30 | 2026-09-14 |
| Safety and Efficacy of NMD670 in Adult Patients With Type 1 and Type 2 Charcot-Marie-Tooth Disease NMD670, Placebo - Charcot-Marie-Tooth Disease | Phase 2 | Completed | NMD Pharma A/S | 81 | 2024-09-30 | 2025-11-12 |
| Study of SPG601 in Adult Men With Fragile X Syndrome SPG601, Placebo - Fragile X Syndrome | Phase 2 | Completed | Spinogenix | 10 | 2024-07-05 | 2026-05-26 |
| Trimetazidine Efficacy in Attenuating Paclitaxel-Induced Peripheral Neuropathy Trimetazidine, Placebo - Peripheral Neuropathy Due to Chemotherapy | Phase 1 / Phase 2 | Completed | Minia University | 60 | 2024-06-23 | 2025-03-30 |
| 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 |
| A Study of NIDO-361 in Patients With SBMA NIDO-361, Placebo - Spinal and Bulbar Muscular Atrophy, Kennedy's Disease | Phase 2 | Completed | Nido Biosciences, Inc. | 54 | 2024-03-01 | 2026-03-03 |
| Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of MBF-015 in Huntington's Disease Patients MBF-015 16 mg oral capsules - Huntington Disease | Phase 2 | Completed | Medibiofarma S.L. | 10 | 2024-02-01 | 2025-03-03 |
| Placebo-Controlled, Single-Dose Challenge Study of Gaboxadol in Adult Males With Fragile X Syndrome (FXS) Gaboxadol, Placebo - Fragile X Syndrome | Phase 2 | Completed | Craig Erickson | 10 | 2024-01-29 | 2026-09-30 |
| Assessing the Safety and Efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) in the Treatment of Rett Syndrome (RTT) NTI164 - Rett Syndrome | Phase 1 / Phase 2 | Completed | Fenix Innovation Group | 14 | 2023-09-20 | 2026-09-09 |
| Tuvusertib (M1774) in Combination With Cemiplimab in Participants With Non-Squamous NSCLC (DDRiver NSCLC 322) M1774, Cemiplimab - Non-Small Cell Lung Cancer | Phase 1 / Phase 2 | Completed | EMD Serono Research & Development Institute, Inc. | 61 | 2023-09-13 | 2026-09-14 |
| A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Of AJ201 In Patients AJ201, Placebo - Spinal and Bulbar Muscular Atrophy, Kennedy's Disease | Phase 1 / Phase 2 | Completed | AnnJi Pharmaceutical Co., Ltd. | 25 | 2023-02-28 | 2024-08-15 |
| Open-label, Long-term Safety Study of LP352 in Subjects With Developmental and Epileptic Encephalopathy LP352, bexicaserin - Developmental and Epileptic Encephalopathy, Dravet Syndrome | Phase 2 | Completed | Longboard Pharmaceuticals | 41 | 2022-11-08 | 2025-12-03 |
| A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia Vatiquinone - Friedreich Ataxia | Phase 2 | Completed | PTC Therapeutics | 5 | 2022-10-12 | 2025-08-22 |
| A Double-Blind, Placebo-Controlled, Dose Exploration Study of CTI-1601 in Adult Subjects With Friedreich's Ataxia CTI-1601, Placebo - Friedreich Ataxia | Phase 2 | Completed | Larimar Therapeutics, Inc. | 28 | 2022-09-21 | 2024-11-29 |
| Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients Placebo, AOC 1001 - DM1, Muscular Dystrophies | Phase 2 | Completed | Avidity Biosciences, Inc. | 37 | 2022-08-04 | 2026-09-11 |
| Efficacy and Safety on SOM3355 in Huntington's Disease Chorea Placebo capsules, SOM3355 200 mg capsules, SOM3355 300 mg capsules - Huntington Chorea | Phase 2 | Completed | SOM Innovation Biotech SA | 139 | 2022-08-02 | 2025-08-28 |
| A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD) PTC518, Placebo - Huntington Disease | Phase 2 | Completed | Novartis Pharmaceuticals | 159 | 2022-06-03 | 2026-06-16 |
| 28-Day Study of SAGE-718 on Functioning Capacity in Participants With Huntington's Disease SAGE-718, Placebo - Huntington Disease | Phase 2 | Completed | Supernus Pharmaceuticals, Inc. | 69 | 2022-05-26 | 2025-09-15 |
| Evaluation of the Effect of Artesunate in Friedreich Ataxia (FA) Artesunate Oral Product - Friedreich Ataxia | Phase 1 / Phase 2 | Completed | Institut National de la Santé Et de la Recherche Médicale, France | 20 | 2022-05-06 | 2026-05-20 |
Showing 20 of 348. Sign in to see all of them.
Huntington Disease (46) · Fragile X Syndrome (32) · Hereditary Sensory and Autonomic Neuropathies (31) · Friedreich Ataxia (29) · Tourette Syndrome (29) · Neurofibromatosis 1 (22) · Tuberous Sclerosis (20) · Mucopolysaccharidosis II (15) · Rett Syndrome (14) · Neurofibromatosis 2 (13) · Myotonic Dystrophy (11) · Adrenoleukodystrophy (10) · Autistic Disorder (10) · Bulbo-Spinal Atrophy, X-Linked (10) · Neurofibromatoses (9)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (348) · Nervous System Diseases (343) · Genetic Diseases, Inborn (341) · Neurodegenerative Diseases (273) · Central Nervous System Diseases (173) · Brain Diseases (156) · Neurologic Manifestations (112) · Neuromuscular Diseases (102) · Mental Disorders (96) · Congenital Abnormalities (94) · Metabolic Diseases (85) · Nutritional and Metabolic Diseases (85) · Genetic Diseases, X-Linked (81) · Basal Ganglia Diseases (79)
Everolimus (12) · Sirolimus (10) · Creatine (5) · alpha-tocotrienol quinone (5) · valbenazine (5) · 2-chloro-4-(1-(4-fluorophenyl)-2,5-dimethyl-1H-imidazol-4-ylethynyl)pyridine (4) · Acetylcysteine (4) · Bevacizumab (4) · ecopipam (4) · idursulfase (4) · Ascorbic Acid (3) · Cetuximab (3) · Injections (3) · Lovastatin (3) · Metformin (3)
Children's Hospital Medical Center, Cincinnati (8) · Massachusetts General Hospital (7) · NYU Langone Health (7) · Novartis Pharmaceuticals (7) · University of Alabama at Birmingham (7) · National Institute of Neurological Disorders and Stroke (NINDS) (6) · Neurocrine Biosciences (6) · Pfizer (6) · Shire (6) · University of Rochester (6) · Hoffmann-La Roche (5) · National Cancer Institute (NCI) (5) · Assistance Publique - Hôpitaux de Paris (4) · Institut National de la Santé Et de la Recherche Médicale, France (4) · National Center for Research Resources (NCRR) (4)
United States (235) · France (52) · Germany (50) · Spain (49) · United Kingdom (48) · Canada (47) · Italy (47) · Australia (31) · Netherlands (18) · Belgium (15) · Poland (14) · Denmark (13) · Brazil (12) · Japan (11) · Israel (10)
Elizabeth Berry-Kravis, MD, PhD (4) · Paul Orchard, MD (4) · David Lynch, MD, PhD (3) · Diana Rosas, MD, MS (3) · Matthias A Karajannis, MD, MS (3) · Theresa Zesiewicz, MD (3) · Alan Percy, MD (2) · Alessandro Filla, MD (2) · Allan L Reiss (2) · Arnold Munnich, M.D. (2)
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