37 completed, 9 unknown, 6 recruiting, 5 terminated. 61 Phase 4.
| Phase | Trials | Status |
|---|---|---|
| Phase 4 | 61 | Completed 37, Unknown 9, Recruiting 6, Terminated 5, Active not recruiting 3, Withdrawn 1 |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 |
|---|---|---|---|---|---|---|
| Cardiac Magnetic Resonance and Echocardiography Study With Acoramidis Acoramidis (AG10) oral tablet - Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM), Amyloidosis | Phase 4 | Recruiting | Eidos Therapeutics, a BridgeBio company | 150 | 2026-07-01 | 2026-07-29 |
| Enavogliflozin for the Management of Patients With Amyloid CardiomyopaThy Enavogliflozin 0.3mg, Placebo - Amyloid Cardiomyopathy | Phase 4 | Recruiting | Seoul St. Mary's Hospital | 68 | 2026-01-06 | 2026-06-18 |
| To Evaluate the Efficacy and Safety of Tafamidis Meglumine Soft Capsules in the Treatment of Adult Patients With Transthyretin Amyloid Polyneuropathy Tafamidis Meglumine Soft Capsules - Transthyretin Amyloid Polyneuropathy | Phase 4 | Recruiting | Qilu Pharmaceutical Co., Ltd. | 15 | 2025-06-19 | 2025-07-30 |
| Effect of Dapagliflozin Administration on the Apoptosis Levels of Patients With Acute Myocardial Infarction Forxiga® 10 mg Film-Coated Tablet, manufactured by AstraZeneca Pharmaceuticals LP, USA for AstraZeneca Pharmaceuticals Co. Ltd., China imported by PT AstraZeneca Indonesia, Indonesia - Acute Myocardial Infarction (AMI), STEMI - ST Elevation Myocardial Infarction (MI) | Phase 4 | Completed | Yoga Yudhistira | 40 | 2024-09-21 | 2025-02-27 |
| D-Cycloserine for Serine Palmitoyltransferase Inhibition D-cycloserine - Hereditary Spastic Paraplegia | Phase 4 | Completed | Massachusetts General Hospital | 1 | 2024-07-10 | 2026-04-21 |
| Real World Effectiveness and Safety of Deutetrabenazine in Adult Chinese Participants With Huntington's Disease (HD) Chorea in China Deutetrabenazine - Huntington's Disease, Chorea | Phase 4 | Completed | Teva Branded Pharmaceutical Products R&D LLC | 50 | 2024-02-02 | 2026-08-27 |
| N-of-1 for Beta-Blockers in Cardiac Amyloidosis Beta blocker - Cardiac Amyloidosis, Heart Diseases | Phase 4 | Completed | Weill Medical College of Cornell University | 14 | 2024-01-31 | 2026-07-29 |
| Ultrasound Therapy In Cardiac Amyloidosis Sonotherapy - Amyloidosis Cardiac | Phase 4 | Recruiting | Brigham and Women's Hospital | 70 | 2023-08-31 | 2026-08-21 |
| A Study to Investigate Behavioral and Other Co-Occurring Outcomes With Epidiolex as Add-On Therapy in Participants Aged 1 to 65 Years of Age With Tuberous Sclerosis Complex Cannabidiol Oral Solution [Epidiolex] - Tuberous Sclerosis Complex Associated Neuropsychiatric Disease | Phase 4 | Completed | Jazz Pharmaceuticals | 79 | 2023-06-29 | 2026-04-27 |
| A Phase IV Study of Safety and Efficacy of Everolimus in Taiwanese Patients With Tuberous Sclerosis Complex Who Have Renal Angiomyolipoma (TSC-AML) Everolimus - Renal Angiomyolipoma | Phase 4 | Completed | Novartis Pharmaceuticals | 4 | 2023-05-01 | 2026-10-29 |
| Treating Leg Symptoms in Women With X-linked Adrenoleukodystrophy Pramipexole, Placebo - Adrenoleukodystrophy, Restless Legs Syndrome | Phase 4 | Active not recruiting | Massachusetts General Hospital | 24 | 2023-04-01 | 2026-07-01 |
| A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II) ELAPRASE, Rituximab, Methotrexate - Mucopolysaccharidosis (MPS), Hunter Syndrome | Phase 4 | Withdrawn | Takeda | 2023-02-28 | 2025-12-09 | |
| Gandouling in the Treatment of Wilson's Disease Gandouling - Wilson's Disease | Phase 4 | Unknown | Jun Li | 240 | 2023-01-01 | 2024-02-09 |
| Real-World Effects of MC4R Agonist Therapy in BBS and Severe Genetic Obesity Setmelanotide - Bardet Biedl Syndrome (BBS), Bardet Biedl Syndrome | Phase 4 | Recruiting | Tom Hühne | 200 | 2023-01-01 | 2026-06-29 |
| A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India Elaprase - Hunter Syndrome | Phase 4 | Completed | Takeda | 5 | 2022-04-21 | 2025-01-23 |
| The Impact of Electroacupuncture Combined With NGF on Clinical Effect and Functional Changes on Ischemic Stroke Nerver growth factor (NGF) injection, Placebo injection, EA intervention - Ischemic Stroke | Phase 4 | Unknown | The Third Affiliated hospital of Zhejiang Chinese Medical University | 288 | 2022-04-01 | 2022-07-28 |
| Metformin in Children With Fragile X Syndrome Metformin, Placebo - Fragile X Syndrome, Metformin | Phase 4 | Completed | Children's Hospital of Fudan University | 37 | 2021-12-29 | 2026-08-25 |
| Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA) OAV101 - Muscular Atrophy, Spinal | Phase 4 | Completed | Novartis Pharmaceuticals | 16 | 2021-11-04 | 2024-10-09 |
| Evaluation of the Effects of Calcitriol's in the Neurological Symptoms of Friedreich's Ataxia Patients Calcitriol, Blood analysis for frataxin's level measurement, Blood analysis for hypercalcemia's control - Friedreich Ataxia | Phase 4 | Completed | Berta Alemany | 20 | 2021-08-25 | 2023-10-23 |
| Assessment of Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution Cannabidiol - Lennox Gastaut Syndrome, Dravet Syndrome | Phase 4 | Active not recruiting | Jazz Pharmaceuticals | 154 | 2021-07-07 | 2026-08-26 |
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Amyloid Neuropathies, Familial (9) · Tourette Syndrome (9) · Hereditary Sensory and Autonomic Neuropathies (7) · Tuberous Sclerosis (6) · Fragile X Syndrome (4) · Huntington Disease (4) · Mucopolysaccharidosis II (4) · Tic Disorders (4) · Tics (4) · Hepatolenticular Degeneration (3) · Amyloidosis (2) · Angiomyolipoma (2) · Bulbo-Spinal Atrophy, X-Linked (2) · Friedreich Ataxia (2) · Ischemic Stroke (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (61) · Nervous System Diseases (61) · Genetic Diseases, Inborn (60) · Neurodegenerative Diseases (52) · Central Nervous System Diseases (32) · Brain Diseases (29) · Neuromuscular Diseases (23) · Metabolic Diseases (21) · Neurologic Manifestations (21) · Nutritional and Metabolic Diseases (21) · Peripheral Nervous System Diseases (20) · Congenital Abnormalities (19) · Pathological Conditions, Signs and Symptoms (19) · Mental Disorders (17)
idursulfase (4) · Everolimus (3) · Aripiprazole (2) · Cannabidiol (2) · Fluoxetine (2) · Methylphenidate (2) · Riluzole (2) · Sirolimus (2) · florbetapir (2) · Acetaminophen (1) · Adrenergic beta-Antagonists (1) · Bupropion (1) · Calcitriol (1) · Citalopram (1) · Clonidine (1)
Novartis Pharmaceuticals (4) · Brigham and Women's Hospital (2) · Jazz Pharmaceuticals (2) · Massachusetts General Hospital (2) · Medical College of Wisconsin (2) · NYU Langone Health (2) · Peking Union Medical College Hospital (2) · Shire (2) · Takeda (2) · All India Institute of Medical Sciences, Bhubaneswar (1) · Berta Alemany (1) · Children's Hospital of Fudan University (1) · Eidos Therapeutics, a BridgeBio company (1) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (1) · HLB Pharmaceutical Co., Ltd. (1)
United States (32) · China (10) · Germany (5) · United Kingdom (5) · Canada (4) · Brazil (3) · Italy (3) · Poland (3) · Spain (3) · Taiwan (3) · Argentina (2) · Belgium (2) · Denmark (2) · France (2) · India (2)
Harvey S Singer, MD (2) · Ahmad Yasa MD (1) · Anil B Mukherjee, M.D. (1) · Barbara J Coffey, M.D, M.S. (1) · Barbara J Coffey, MD, MS (1) · Berta Alemany Perna (1) · Chris Mueller, MD (1) · Craig A. Erickson, MD (1) · Debasish Hota, MD, DM (1) · Donald S Higgins, M.D. (1) · Emily Stern, PhD (1) · Eva Kohegyi, MD, MS (1) · Florian S Eichler, MD (1)
PMID 1135616, PMID 11383973, PMID 16829550, PMID 17006758, PMID 17159456, PMID 17459549, PMID 18791076, PMID 20218790, PMID 20568658, PMID 20579510, PMID 21059347, PMID 21357874, PMID 21639796, PMID 23152677, PMID 24595507, PMID 24799137, PMID 24841414, PMID 24997880, PMID 25205361, PMID 25681004, PMID 25770310, PMID 25870613, PMID 25902842, PMID 26077459, PMID 2855531, PMID 28626052, PMID 29487073, PMID 29729645, PMID 29984566, PMID 30762779, PMID 30782512, PMID 32316911, PMID 32740676, PMID 33262243, PMID 33305808, PMID 33488759, PMID 34770113, PMID 35482910, PMID 37621153, PMID 38428428, PMID 38866445, PMID 39033586, PMID 40104015, PMID 40538652, PMID 40903014, PMID 41291951, PMID 42435131, PMID 42477731, PMID 8054244
2023 (3), 2018 (2), 2014 (1), R01MH045802 (1), R24AG064025 (1), R34MH085844 (1), T32GM007748 (1)