12 withdrawn. 12 Phase 1, 5 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 12 | Withdrawn 12 |
| Phase 2 | 5 | Withdrawn 5 |
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 |
|---|---|---|---|---|---|---|
| Phase I/IIa Trial of scAAV1.tMCK.NTF3 for Treatment of CMT1A scAAV1.tMCK.NTF3 - Charcot-Marie-Tooth Neuropathy Type 1A | Phase 1 / Phase 2 | Withdrawn | Nationwide Children's Hospital | 2027-04-01 | 2026-07-29 | |
| A Study of ASP2016 in Adults Who Have Heart Disease Associated With Friedreich Ataxia ASP2016, Prednisolone - Friedreich Ataxia, Cardiomyopathy | Phase 1 | Withdrawn | Astellas Gene Therapies | 2024-11-08 | 2025-11-28 | |
| Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II Genetic HMI-203 - Mucopolysaccharidosis II | Phase 1 | Withdrawn | Homology Medicines, Inc | 2022-09-08 | 2023-08-28 | |
| Safety of Cultured Allogeneic Adult Umbilical Cord or Placental Derived Mesenchymal Stem Cells for Tourette Syndrome Cultured allogeneic adult umbilical cord or placental derived mesenchymal stem cells - Tourette Syndrome | Phase 1 | Withdrawn | The Foundation for Orthopaedics and Regenerative Medicine | 2022-01-01 | 2026-10-16 | |
| Safety and Tolerability Study With VY-HTT01, in Adults With Early Manifesting Huntington's Disease Intraparenchymal rAAV1 - (mi)RNA HTT - Huntington Disease | Phase 1 | Withdrawn | Voyager Therapeutics | 2021-07-30 | 2021-08-16 | |
| Novel Clinical Target in Fragile X Syndrome 18F-FTC-146 - Fragile X Syndrome (FXS) | Phase 1 | Withdrawn | Guido A. Davidzon, MD, SM | 2021-01-12 | 2022-11-15 | |
| Epacadostat in Combination With Pembrolizumab and Azacitidine in Subjects With Metastatic Colorectal Cancer Epacadostat (INCB024360) in Combination with Pembrolizumab (MK-3475) and Azacitidine (VIDAZA) - Metastatic Colorectal Cancer | Phase 1 / Phase 2 | Withdrawn | James J Lee | 2018-09-30 | 2018-08-28 | |
| Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy Sobetirome (NV1205) - X-Linked Adrenoleukodystrophy | Phase 1 / Phase 2 | Withdrawn | NeuroVia, Inc. | 2018-08-01 | 2019-03-07 | |
| Safety and Efficacy Study of Neratinib and Cetuximab to Treat Patients With Quadruple Wild-Type Metastatic Colorectal Cancer Cetuximab, Neratinib - Colorectal Cancer | Phase 1 / Phase 2 | Withdrawn | NSABP Foundation Inc | 2013-10-01 | 2021-10-27 | |
| Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD) Sobetirome - X-Linked Adrenoleukodystrophy, Adrenomyeloneuropathy | Phase 1 | Withdrawn | Thomas S. Scanlan | 2013-04-01 | 2014-02-14 | |
| Optimising Anterior Pallidal Deep Brain Stimulation for Tourette's Syndrome Deep brain stimulator ventral electrode up to 2 mA, Deep brain stimulator ventral electrode up to 3 mA, Deep brain stimulator dorsal electrode up to 2 mA - Tourette's Syndrome | Phase 1 / Phase 2 | Withdrawn | The University of Western Australia | 2013-03-01 | 2021-05-18 | |
| Safety and Efficacy Study of HuCNS-SC in Subjects With Neuronal Ceroid Lipofuscinosis HuCNS-SC - Neuronal Ceroid Lipofuscinosis | Phase 1 | Withdrawn | StemCells, Inc. | 2010-11-01 | 2015-01-15 |
Adrenoleukodystrophy (2) · Colorectal Neoplasms (2) · Tourette Syndrome (2) · Cardiomyopathies (1) · Charcot-Marie-Tooth Disease (1) · Fragile X Syndrome (1) · Friedreich Ataxia (1) · Hereditary Sensory and Autonomic Neuropathies (1) · Huntington Disease (1) · Mucopolysaccharidosis II (1) · Neuronal Ceroid-Lipofuscinoses (1) · Spinocerebellar Degenerations (1) · Sudden Infant Death (1) · Thyroid Diseases (1) · Tics (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (12) · Genetic Diseases, Inborn (12) · Nervous System Diseases (12) · Neurodegenerative Diseases (8) · Brain Diseases (7) · Central Nervous System Diseases (7) · Metabolic Diseases (5) · Neurologic Manifestations (5) · Nutritional and Metabolic Diseases (5) · Genetic Diseases, X-Linked (4) · Intellectual Disability (4) · Metabolism, Inborn Errors (4) · Neurobehavioral Manifestations (4) · X-Linked Intellectual Disability (4)
GC 1 compound (2) · 6-(3-fluoropropyl)-3-(2-(azepan-1-yl)ethyl)benzo(d)thiazol-2(3H)-one (1) · Azacitidine (1) · Cetuximab (1) · Disease Vectors (1) · Prednisolone (1) · epacadostat (1) · neratinib (1) · pembrolizumab (1)
Astellas Gene Therapies (1) · Guido A. Davidzon, MD, SM (1) · Homology Medicines, Inc (1) · James J Lee (1) · NSABP Foundation Inc (1) · Nationwide Children's Hospital (1) · NeuroVia, Inc. (1) · StemCells, Inc. (1) · The Foundation for Orthopaedics and Regenerative Medicine (1) · The University of Western Australia (1) · Thomas S. Scanlan (1) · Voyager Therapeutics (1)
United States (7) · Australia (2) · Argentina (1) · Canada (1) · Chile (1) · Colombia (1) · France (1) · Russia (1) · Ukraine (1) · United Kingdom (1)
Christopher Lind, FRACS (1) · David Koeller, MD (1) · Guido Davidzon, MD SM (1) · James J Lee, MD, PhD (1) · John Henderson, MD (1) · Nathan Selden, MD, PhD (1) · Norman Wolmark, MD (1) · Zarife Sahenk, MD., PhD. (1)