5 enrolling by invitation. 5 Phase 1, 2 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 5 | Enrolling by invitation 5 |
| Phase 2 | 2 | Enrolling by invitation 2 |
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 |
|---|---|---|---|---|---|---|
| PET Imaging With [18F]Fluselenamyl in People With Cardiac Amyloidosis [18F]Fluselenamyl (FSA) - Cardiac Amyloidosis | Phase 1 | Enrolling by invitation | Washington University School of Medicine | 50 | 2026-01-14 | 2026-08-18 |
| Assessing the Efficacy of Repeat, Monthly Treatments of Deoxycholate for NF1 Associated Cutaneous Neurofibromas (cNFs) Kybella - Neurofibromas, Cutaneous, Neurofibromatosis 1 | Phase 1 | Enrolling by invitation | Massachusetts General Hospital | 15 | 2025-04-08 | 2026-04-22 |
| Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy SRP-1003 IV Infusion, Placebo IV Infusion, SRP-1003 SC Injection - Myotonic Dystrophy 1 | Phase 1 / Phase 2 | Enrolling by invitation | Sarepta Therapeutics, Inc. | 78 | 2024-03-04 | 2026-10-05 |
| Sensitivity of the NIH Toolbox to Stimulant Treatment in Intellectual Disabilities Methylphenidate Oral Solution - Intellectual Disability, Fragile X Syndrome | Phase 1 | Enrolling by invitation | University of California, Davis | 68 | 2023-02-01 | 2025-12-15 |
| rAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan Disease rAAV-Olig001-ASPA, Levetiracetam, Prednisone - Canavan Disease | Phase 1 / Phase 2 | Enrolling by invitation | Myrtelle Inc. | 24 | 2021-04-01 | 2025-11-14 |
Amyloid Neuropathies, Familial (1) · Attention Deficit Disorder with Hyperactivity (1) · Canavan Disease (1) · Down Syndrome (1) · Fragile X Syndrome (1) · Immunoglobulin Light-chain Amyloidosis (1) · Intellectual Disability (1) · Myotonic Dystrophy (1) · Neurofibroma (1) · Neurofibromatosis 1 (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (5) · Genetic Diseases, Inborn (5) · Nervous System Diseases (5) · Neurodegenerative Diseases (4) · Neuromuscular Diseases (3) · Metabolic Diseases (2) · Metabolism, Inborn Errors (2) · Neoplasms (2) · Neoplasms by Histologic Type (2) · Nutritional and Metabolic Diseases (2) · Peripheral Nervous System Diseases (2) · Abnormalities, Multiple (1) · Amyloid Neuropathies (1) · Amyloidosis (1)
Deoxycholic Acid (1) · Levetiracetam (1) · Methylphenidate (1) · Prednisone (1)
Massachusetts General Hospital (1) · Myrtelle Inc. (1) · Sarepta Therapeutics, Inc. (1) · University of California, Davis (1) · Washington University School of Medicine (1)
United States (4) · Australia (1) · Belgium (1) · Canada (1) · France (1) · Germany (1) · Italy (1) · New Zealand (1) · Spain (1) · Taiwan (1) · Thailand (1) · United Kingdom (1)
Richard R Anderson, MD (1) · Robert Lober, MD, PhD (1)
R01HD076189 (1)