35 active not recruiting. 35 Phase 3, 9 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 35 | Active not recruiting 35 |
| Phase 2 | 9 | Active not recruiting 9 |
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 |
|---|---|---|---|---|---|---|
| Pivotal Study of N-acetyl-L-leucine for CACNA1A N-Acetyl-L-Leucine, Placebo - CACNA1A, Spinocerebellar Ataxia Type 6 | Phase 3 | Active not recruiting | IntraBio Inc | 60 | 2026-08-17 | 2026-11-03 |
| Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome TSHA-102 - Rett Syndrome | Phase 3 | Active not recruiting | Taysha Gene Therapies, Inc. | 4 | 2026-05-08 | 2026-08-19 |
| RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome) RGX-121-3102 - MPS II, Hunter Syndrome (MPS II) | Phase 3 | Active not recruiting | REGENXBIO Inc. | 2 | 2025-11-25 | 2026-06-04 |
| Confirmatory Study 3 of KPS-0373 in Patients With Spinocerebellar Degeneration KPS-0373, Placebo - Spinocerebellar Degeneration | Phase 3 | Active not recruiting | Kissei Pharmaceutical Co., Ltd. | 142 | 2025-08-15 | 2026-09-04 |
| A Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia Omaveloxolone, Placebo - Friedreich Ataxia | Phase 3 | Active not recruiting | Biogen | 255 | 2025-06-09 | 2026-10-02 |
| Study of SKY-0515 for Safety, Efficacy, and Pharmacodynamics in Participants With Huntington's Disease SKY-0515, SKY-0515 Placebo - Huntington Disease, Huntingtons Disease | Phase 2 / Phase 3 | Active not recruiting | Skyhawk Therapeutics, Inc. | 120 | 2025-05-27 | 2026-05-01 |
| A Study to Compare the Efficacy and Safety of BMS-986365 Versus the Investigator's Choice of Therapy in Participants With Metastatic Castration-resistant Prostate Cancer BMS-986365, Enzalutamide, Abiraterone - Metastatic Castration-resistant Prostate Cancer | Phase 3 | Active not recruiting | Celgene | 960 | 2025-03-13 | 2026-09-30 |
| A Phase 3, Multicenter, Randomized, Double-Masked, Sham-Controlled Clinical Trial for Leber's Hereditary Optic Neuropathy (LHON) Associated With ND4 Mutation rAAV2-ND4, Sham (No Treatment) - Leber Hereditary Optic Neuropathy (LHON) | Phase 3 | Active not recruiting | Wuhan Neurophth Biotechnology Limited Company | 95 | 2024-09-19 | 2026-02-12 |
| Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment Idursulfase-IT, Elaprase - Hunter Syndrome | Phase 2 / Phase 3 | Active not recruiting | Takeda | 6 | 2024-03-05 | 2025-11-06 |
| A Study to Evaluate Long-term Safety of Ecopipam Tablets in Children, Adolescents and Adults With Tourette's Disorder Ecopipam - Tourette Syndrome | Phase 3 | Active not recruiting | Emalex Biosciences Inc. | 150 | 2023-08-16 | 2026-07-22 |
| FCN-159 in Adult Patients With Symptomatic, Inoperable Neurofibromatosis Type 1-Related Plexiform Neurofibromas Test group (Group A): FCN-159 8 mg, orally, once daily;, Control group (Group B): Placebo, orally, once daily; - Neurofibromatosis 1, Plexiform Neurofibroma | Phase 3 | Active not recruiting | Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. | 167 | 2023-06-20 | 2025-03-06 |
| A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome NGN-401 - Rett Syndrome | Phase 3 | Active not recruiting | Neurogene Inc. | 33 | 2023-06-13 | 2026-06-10 |
| Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab Apitegromab - Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3 | Phase 3 | Active not recruiting | Scholar Rock, Inc. | 238 | 2023-04-17 | 2026-09-01 |
| A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study) TSHA-102 - Rett Syndrome | Phase 3 | Active not recruiting | Taysha Gene Therapies, Inc. | 17 | 2023-03-06 | 2026-07-07 |
| Molecular Imaging of Myocardial Fibrosis in Cardiac Amyloidosis [68Ga]CBP8 PET/MRI - Cardiac Amyloidosis | Phase 3 | Active not recruiting | Brigham and Women's Hospital | 18 | 2023-01-03 | 2026-07-06 |
| Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials onasemnogene abeparvovec - Spinal Muscular Atrophy (SMA) | Phase 3 | Active not recruiting | Novartis Pharmaceuticals | 20 | 2022-12-19 | 2026-07-09 |
| A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia Vatiquinone - Friedreich Ataxia | Phase 3 | Active not recruiting | PTC Therapeutics | 130 | 2022-12-08 | 2025-10-30 |
| An Open-Label Extension Study of BPN14770 in Subjects With Fragile X Syndrome Zatolmilast/ BPN14770 - Fragile X Syndrome | Phase 3 | Active not recruiting | Tetra Discovery Partners | 314 | 2022-11-01 | 2025-10-03 |
| A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II) tividenofusp alfa, idursulfase - Mucopolysaccharidosis II | Phase 2 / Phase 3 | Active not recruiting | Denali Therapeutics Inc. | 65 | 2022-07-21 | 2026-09-09 |
| A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT) JR-141, Idursulfase, JR-141 or Idursulfase - Mucopolysaccharidosis II | Phase 3 | Active not recruiting | JCR Pharmaceuticals Co., Ltd. | 86 | 2022-02-14 | 2026-10-02 |
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Mucopolysaccharidosis II (7) · Nervous System Diseases (5) · Neurologic Manifestations (5) · Genetic Diseases, X-Linked (4) · Intellectual Disability (4) · Neurodevelopmental Disorders (4) · Sudden Infant Death (4) · Neurofibromatosis 1 (3) · Pathologic Processes (3) · Rett Syndrome (3) · Spinocerebellar Ataxias (3) · X-Linked Intellectual Disability (3) · Amyloid Neuropathies, Familial (2) · Bulbo-Spinal Atrophy, X-Linked (2) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (33) · Genetic Diseases, Inborn (33) · Nervous System Diseases (30) · Neurodegenerative Diseases (22) · Neurologic Manifestations (16) · Nutritional and Metabolic Diseases (15) · Central Nervous System Diseases (14) · Metabolic Diseases (14) · Pathological Conditions, Signs and Symptoms (14) · Genetic Diseases, X-Linked (11) · Metabolism, Inborn Errors (11) · Neurobehavioral Manifestations (11) · Brain Diseases (10) · Signs and Symptoms (10)
idursulfase (3) · Zolgensma (2) · salicylhydroxamic acid (2) · AZD 6244 (1) · BPN14770 (1) · Carboplatin (1) · Control Groups (1) · Dichloroacetic Acid (1) · Docetaxel (1) · Everolimus (1) · Genotype (1) · Magnetic Resonance Spectroscopy (1) · Prednisolone (1) · Vincristine (1) · abiraterone (1)
JCR Pharmaceuticals Co., Ltd. (2) · Novartis Pharmaceuticals (2) · REGENXBIO Inc. (2) · Taysha Gene Therapies, Inc. (2) · AO GENERIUM (1) · AstraZeneca (1) · Biogen (1) · Biohaven Pharmaceuticals, Inc. (1) · Brigham and Women's Hospital (1) · Celgene (1) · Denali Therapeutics Inc. (1) · Eidos Therapeutics, a BridgeBio company (1) · Emalex Biosciences Inc. (1) · IntraBio Inc (1) · Ionis Pharmaceuticals, Inc. (1)
United States (28) · Canada (15) · Italy (15) · United Kingdom (15) · Australia (12) · France (12) · Spain (11) · Brazil (10) · Germany (9) · Belgium (7) · Japan (7) · Netherlands (7) · China (6) · Poland (6) · Denmark (4)
Alexander Schramm (1) · Alice P. Chen, MD (1) · Amy E Brown, MD (1) · Elizabeth Berry-Kravis, MD (1) · Jason R Fangusaro (1) · Jose Alcantara Rodriguez, PharmD (1) · Julie Jordan, MD (1) · Masoud Mokhtarani, MD (1) · Oksana A. Markova, MD, MSc (1) · Richard Neibeger, MD (1)
PMID 38723617, PMID 33395032, PMID 33395098, PMID 34115419, PMID 34383289, PMID 38254921, PMID 38814532, PMID 39556242, PMID 39806095, PMID 40173263, PMID 40455815, PMID 40473450, PMID 41205147, PMID 41254489, PMID 41911489, PMID 42389794
2023 (2), 2025 (2), 2018 (1), 2019 (1), U10CA180886 (1)