17 completed, 5 terminated, 4 recruiting, 2 unknown. 31 Early Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Early Phase 1 | 31 | Completed 17, Terminated 5, Recruiting 4, Unknown 2, Active not recruiting 1, Not yet recruiting 1, Withdrawn 1 |
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 |
|---|---|---|---|---|---|---|
| SPX-01 Gene Therapy in Pediatric Patients With Spinal Muscular Atrophy Type 1 (RISE) SPX-01 - Spinal Muscular Atrophy (SMA) | Early Phase 1 | Not yet recruiting | Exir Gene Pharmed | 1 | 2027-01-01 | 2026-10-29 |
| iMagemHTT-009- FIH Evaluation of Novel Mutant Huntingtin PET Radioligand [11C]CHDI-00491009 Radioligand [11C]CHDI-00491009 - Huntington Disease, HD | Early Phase 1 | Recruiting | CHDI Foundation, Inc. | 27 | 2026-02-24 | 2026-03-13 |
| A Clinical Study on the Treatment of Wilson Disease With ATP7B mRNA/LNP (DSL101) Group 1: DSL101 Low dose, Group 2: DSL101 Medium dose, Group 3: DSL101 High dose - Wilsons Disease | Early Phase 1 | Recruiting | DSciLab Co., Ltd. | 18 | 2025-12-31 | 2026-01-20 |
| Single-Dose AAV-MECP2 Safety/Tolerability and Efficacy in Rett Syndrome Intrathecal injection of AAV-MECP2 for the treatment of Rett syndrome - Rett Syndrome | Early Phase 1 | Active not recruiting | Guangzhou Women and Children's Medical Center | 8 | 2025-01-14 | 2026-05-28 |
| A Clinical Study to Evaluate the Safety and Efficacy of LY-M003 Injection in Patients With Wilson Disease LY-M003 - Wilson Disease | Early Phase 1 | Recruiting | Chaohui Yu | 18 | 2024-09-24 | 2026-01-23 |
| Calcium Folinate Treatment of Spastic Paraplegia 56 calcium folinate - Hereditary Spastic Paraplegia | Early Phase 1 | Recruiting | Shanghai 6th People's Hospital | 10 | 2024-07-01 | 2024-06-27 |
| 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 |
| D-cycloserine Augmented Treatment for Youth With Tic Disorders d-cycloserine, Placebo pill, Behavior Therapy - Tourette Syndrome | Early Phase 1 | Withdrawn | Johns Hopkins University | 2023-04-15 | 2023-04-14 | |
| 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 |
| Recombinant Adeno-associated Virus Administration for Patients With Menkes Syndrome Recombinant adeno-associated virus administration - Menkes Syndrome | Early Phase 1 | Terminated | Kunming Hope of Health Hospital | 1 | 2022-08-18 | 2022-11-30 |
| 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 |
| iMagemHTT: FIH Evaluation of Novel Mutant Huntingtin PET Radioligands [¹¹C]CHDI-00485180-R and [¹¹C]CHDI-00485626 Radioligand [¹¹C]CHDI-00485180-R, Radioligand [¹¹C]CHDI-00485626 - Huntington Disease | Early Phase 1 | Terminated | CHDI Foundation, Inc. | 24 | 2021-02-09 | 2023-05-06 |
| Molecular Imaging of the Underlying Mechanism of Cardiotoxicity in Patients With Light Chain Amyloidosis Using PET/CT FNOS, Florbetaben - Cardiac Amyloidosis | Early Phase 1 | Terminated | University of Pennsylvania | 3 | 2019-10-01 | 2024-11-12 |
| The Individual Therapy for Patients With Wilson's Disease DMPS, Penicillamine, DMSA - Wilson's Disease | Early Phase 1 | Unknown | Second Affiliated Hospital, Zhejiang University, School of Medicine | 400 | 2019-03-15 | 2019-05-21 |
| Pimavanserin Treatment in TS Pimavanserin - Tourette Syndrome, Tardive Dyskinesia | Early Phase 1 | Completed | Joseph Jankovic | 10 | 2018-11-12 | 2021-03-12 |
| Assessment of Cardiac Fixation During PET Using a New Drug Within Amyloid Cardiac Injuries. PET with 18 F-Flutemetamol - Cardiac Amyloidosis | Early Phase 1 | Terminated | University Hospital, Toulouse | 7 | 2017-03-15 | 2020-08-26 |
| The Safety and Efficacy of Long-term Treatment of PINS Stimulator System for Tourette Syndrome deep brain stimulation (DBS) - Tourette Syndrome | Early Phase 1 | Unknown | Beijing Pins Medical Co., Ltd | 20 | 2016-12-01 | 2016-10-14 |
| 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 |
Showing 20 of 31. Sign in to see all of them.
Huntington Disease (6) · Amyloid Neuropathies, Familial (4) · Hepatolenticular Degeneration (3) · Neurofibromatosis 2 (3) · Neuroma, Acoustic (3) · Tourette Syndrome (3) · Friedreich Ataxia (2) · Meningioma (2) · Neurofibroma (2) · Neurofibromatoses (2) · Rett Syndrome (2) · Spinal Muscular Atrophies of Childhood (2) · Amyloidosis, Hereditary, Transthyretin-Related (1) · Depression (1) · Fragile X Syndrome (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (31) · Genetic Diseases, Inborn (31) · Nervous System Diseases (31) · Neurodegenerative Diseases (27) · Central Nervous System Diseases (17) · Brain Diseases (15) · Basal Ganglia Diseases (12) · Movement Disorders (12) · Metabolic Diseases (10) · Nutritional and Metabolic Diseases (10) · Mental Disorders (9) · Neuromuscular Diseases (9) · Metabolism, Inborn Errors (8) · Dyskinesias (7)
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 (2) · 2-phenyl-6-(2'-(4'-(ethoxycarbonyl)thiazolyl))thiazolo(3,2-b)(1,2,4)triazole (1) · Acamprosate (1) · Baclofen (1) · Behavior Therapy (1) · Cycloserine (1) · Deep Brain Stimulation (1) · Everolimus (1) · HDAC-42 (1) · Lapatinib (1) · Leucovorin (1) · Lovastatin (1) · Magnetic Resonance Spectroscopy (1) · Methylprednisolone (1)
CHDI Foundation, Inc. (4) · Children's Hospital of Philadelphia (2) · ExoRNA Bioscience (2) · Austin Neuromuscular Center (1) · Beijing Pins Medical Co., Ltd (1) · Chaohui Yu (1) · Children's Hospital Medical Center, Cincinnati (1) · Children's Hospital of Fudan University (1) · DSciLab Co., Ltd. (1) · Exir Gene Pharmed (1) · Guangzhou Jiayin Biotech Ltd (1) · Guangzhou Women and Children's Medical Center (1) · Indiana University (1) · Johns Hopkins University (1) · Joseph Jankovic (1)
United States (14) · China (10) · Belgium (2) · Denmark (2) · Netherlands (2) · Norway (2) · Sweden (2) · France (1) · Iran (1)
Cristina Sampaio, MD, PhD (2) · Andrea Varrone, MD, PhD (1) · Andrei Iagaru, MD (1) · Bernhard Landwehrmeyer, MD, PhD (1) · Brad Welling, MD, PhD (1) · Chaohui Yu, 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) · Joseph McGuire, PhD (1) · Li Li (1) · Li Luming, PhD (1)
PMID 26943915, PMID 30043115, PMID 34667843, PMID 36167501, PMID 40527969
U54HD082008 (1)