17 completed. 17 Early Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Early Phase 1 | 17 | Completed 17 |
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 |
|---|---|---|---|---|---|---|
| The Safety and Tolerability Study With ER2001 Intravenous Injection Repetitive Treatment in Adults With Early Manifest Huntington's Disease. ER2001 injection - Huntington Disease | Early Phase 1 | Completed | ExoRNA Bioscience | 8 | 2024-06-25 | 2026-05-22 |
| Esketamine for the Treatment of Rett Syndrome Esketamine hydrochloride - Rett Syndrome | Early Phase 1 | Completed | Children's Hospital of Fudan University | 3 | 2023-11-15 | 2026-05-13 |
| The Safety and Tolerability Study With ER2001 Intravenous Injection in Adults With Early Manifest Huntington's Disease ER2001 injection - Huntington Disease | Early Phase 1 | Completed | ExoRNA Bioscience | 10 | 2023-04-04 | 2025-12-19 |
| Spectrum of Peripheral and Autonomic Neuropathies in Patients With aTTRwt Amyloidosis and Response to Patisiran Therapy patisiran - Polyneuropathies, Wild Type ATTR Amyloidosis | Early Phase 1 | Completed | Austin Neuromuscular Center | 10 | 2022-08-03 | 2026-05-22 |
| Clinical Trial to Evaluate the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy Type I EXG001-307 injection - Type I Spinal Muscular Atrophy | Early Phase 1 | Completed | Guangzhou Jiayin Biotech Ltd | 2 | 2022-01-21 | 2024-08-28 |
| Pimavanserin Treatment in TS Pimavanserin - Tourette Syndrome, Tardive Dyskinesia | Early Phase 1 | Completed | Joseph Jankovic | 10 | 2018-11-12 | 2021-03-12 |
| PET/MRI Evaluation of Cardiac Amyloid 18F-Florbetaben (Neuraceq®) PET/MRI - Cardiac Amyloidosis | Early Phase 1 | Completed | Stanford University | 9 | 2016-05-26 | 2019-07-17 |
| Rosuvastatin (Crestor) in Friedreich Ataxia Rosuvastatin - Friedreich Ataxia | Early Phase 1 | Completed | Children's Hospital of Philadelphia | 12 | 2016-05-01 | 2021-03-25 |
| Neurophysiological and Acute Pharmacological Studies in FXS Patients Acamprosate, Lovastatin, Minocycline - Fragile X Syndrome | Early Phase 1 | Completed | Children's Hospital Medical Center, Cincinnati | 29 | 2016-01-01 | 2021-11-26 |
| Follow-up Measurement of Brain PDE10A Enzyme Levels in Huntington´s Disease Gene Expansion Carriers Radioligand [18F]MNI-659 - Huntington's Disease | Early Phase 1 | Completed | CHDI Foundation, Inc. | 45 | 2015-09-06 | 2019-10-10 |
| Methylprednisolone Treatment of Friedreich Ataxia Methylprednisolone - Friedreich Ataxia | Early Phase 1 | Completed | Children's Hospital of Philadelphia | 11 | 2015-06-01 | 2021-03-25 |
| Exploring the Activity of RAD001 in Vestibular Schwannomas and Meningiomas RAD001 - Neurofibromatosis Type 2, Vestibular Schwannomas | Early Phase 1 | Completed | NYU Langone Health | 5 | 2013-06-01 | 2020-05-13 |
| [PETDE10] Imaging of PDE10A Enzyme Levels in Huntington's Disease Gene Expansion Carriers and Healthy Controls With PET. PET Imaging with [18F]MNI-659 - Huntington's Disease | Early Phase 1 | Completed | CHDI Foundation, Inc. | 90 | 2013-01-01 | 2016-06-01 |
| Acceptance and Commitment Therapy for Adolescents and Young Adults With Neurofibromatosis and Chronic Pain ACT Workshop - Neurofibromatosis | Early Phase 1 | Completed | National Cancer Institute (NCI) | 28 | 2012-06-15 | 2019-09-30 |
| Study of Tasigna®/Nilotinib (AMN107) in Neurofibromatosis (NF1) Patients With Plexiform Neurofibromas Tasigna - Neurofibromatosis, NF1 | Early Phase 1 | Completed | Indiana University | 6 | 2011-01-01 | 2017-04-12 |
| Concentration and Activity of Lapatinib in Vestibular Schwannomas lapatinib - Vestibular Schwannoma, NF2 | Early Phase 1 | Completed | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | 26 | 2009-06-01 | 2021-01-12 |
| Ranibizumab for Neurofibromas Associated With Neurofibromatosis 1 Ranibizumab - Neurofibromatosis Type 1, Cutaneous Neurofibromas | Early Phase 1 | Completed | Massachusetts General Hospital | 11 | 2008-03-01 | 2016-07-07 |
Huntington Disease (4) · Amyloid Neuropathies, Familial (2) · Friedreich Ataxia (2) · Neurofibroma (2) · Neurofibromatoses (2) · Neurofibromatosis 2 (2) · Neuroma, Acoustic (2) · Amyloidosis, Hereditary, Transthyretin-Related (1) · Depression (1) · Fragile X Syndrome (1) · Meningioma (1) · Neurofibroma, Plexiform (1) · Neurofibromatosis 1 (1) · Polyneuropathies (1) · Rett Syndrome (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (17) · Genetic Diseases, Inborn (17) · Nervous System Diseases (17) · Neurodegenerative Diseases (15) · Central Nervous System Diseases (8) · Brain Diseases (7) · Basal Ganglia Diseases (5) · Dyskinesias (5) · Mental Disorders (5) · Movement Disorders (5) · Neoplasms (5) · Neoplasms by Histologic Type (5) · Neoplasms, Nerve Tissue (5) · Neoplastic Syndromes, Hereditary (5)
2-(2-(3-(4-(2-fluoroethoxy)phenyl)-7-methyl-4-oxo-3,4-dihydroquinazolin-2-yl)ethyl)-4-isopropoxyisoindoline-1,3-dione (2) · 4-(N-methylamino)-4'-(2-(2-(2-fluoroethoxy)ethoxy)ethoxy)stilbene (1) · Acamprosate (1) · Baclofen (1) · Everolimus (1) · Lapatinib (1) · Lovastatin (1) · Magnetic Resonance Spectroscopy (1) · Methylprednisolone (1) · Minocycline (1) · Ranibizumab (1) · Rosuvastatin Calcium (1) · nilotinib (1) · patisiran (1) · pimavanserin (1)
CHDI Foundation, Inc. (2) · Children's Hospital of Philadelphia (2) · ExoRNA Bioscience (2) · Austin Neuromuscular Center (1) · Children's Hospital Medical Center, Cincinnati (1) · Children's Hospital of Fudan University (1) · Guangzhou Jiayin Biotech Ltd (1) · Indiana University (1) · Joseph Jankovic (1) · Massachusetts General Hospital (1) · NYU Langone Health (1) · National Cancer Institute (NCI) (1) · Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins (1) · Stanford University (1)
United States (11) · China (4) · Denmark (2) · Netherlands (2) · Norway (2) · Sweden (2)
Cristina Sampaio, MD, PhD (2) · Andrea Varrone, MD, PhD (1) · Andrei Iagaru, MD (1) · Bernhard Landwehrmeyer, MD, PhD (1) · Craig A Erickson, M.D. (1) · David Lynch, MD PhD (1) · David R Lynch, MD PhD (1) · Huiping Li (1) · Jaishri O Blakeley, MD (1) · Melissa Markel, MD (1) · Scott Plotkin, MD, PhD (1) · Staci M Peron, Ph.D. (1) · Theodore Nicolaides, MD (1) · Xia Meng, Ph.D (1)
U54HD082008 (1)