37 completed. 37 Phase 4.
| Phase | Trials | Status |
|---|---|---|
| Phase 4 | 37 | Completed 37 |
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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
| 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 |
| 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 |
| The Effect Of Tafamidis Meglumine In Transthyretin Amyloid Polyneuropathy Patients tafamidis meglumine - Transthyretin Amyloid Polyneuropathy (ATTR-PN) | Phase 4 | Completed | Pfizer | 15 | 2021-04-28 | 2024-03-12 |
| C-Trelin Orally Disintegrated(OD) Tablet 5mg in Ataxia Due to Spinocerebellar Degeneration C-Trelin OD Tab(5mg Taltirelin Hydrate), Placebo - Spinocerebellar Degeneration | Phase 4 | Completed | HLB Pharmaceutical Co., Ltd. | 160 | 2019-03-04 | 2023-07-24 |
| Safety and Efficacy of Everolimus (Afinitor®) in Chinese Adult Patients With Angiomyolipoma Associated With Tuberous Sclerosis Complex. Everolimus - Renal Angiomyolipoma | Phase 4 | Completed | Novartis Pharmaceuticals | 40 | 2018-11-09 | 2021-09-09 |
| Dexmedetomidine and Propofol for Pediatric MRI Sedation propofol, Dexmedetomidine, Glycopyrrolate - Headache, Tumor | Phase 4 | Completed | Medical College of Wisconsin | 40 | 2018-03-04 | 2020-02-21 |
| Lurasidone Vs Olanzapine on Neurotrophic Biomarkers and Cardiometabolic Parameters in Unmedicated Schizophrenia Olanzapine, Lurasidone - Schizophrenia | Phase 4 | Completed | All India Institute of Medical Sciences, Bhubaneswar | 100 | 2017-08-25 | 2020-03-31 |
| Effects of Ondansetron in Obsessive-compulsive and Tic Disorders Ondansetron, Placebo - Obsessive-Compulsive Disorder, Tic Disorders | Phase 4 | Completed | NYU Langone Health | 110 | 2017-06-16 | 2024-05-03 |
| Study to Assess the Efficacy and Safety of Raxone in LHON Patients Idebenone - Leber's Hereditary Optic Neuropathy (LHON) | Phase 4 | Completed | Santhera Pharmaceuticals | 199 | 2016-05-01 | 2023-04-21 |
| Effects of Intranasal Nerve Growth Factor for Acute Ischemic Stroke Nerve Growth Factors, normal saline - Acute Ischemic Stroke | Phase 4 | Completed | Jinling Hospital, China | 106 | 2016-01-01 | 2021-08-31 |
| Combining Lovastatin and a Parent-Implemented Language Intervention for Fragile X Syndrome Lovastatin, Placebo - Fragile X Syndrome, Genetic Diseases | Phase 4 | Completed | University of California, Davis | 30 | 2016-01-01 | 2019-09-11 |
| Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age Elaprase for intravenous (IV) infusion - Hunter Syndrome | Phase 4 | Completed | Shire | 21 | 2015-10-28 | 2026-04-02 |
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Tourette Syndrome (6) · Tuberous Sclerosis (4) · Amyloid Neuropathies, Familial (3) · Fragile X Syndrome (3) · Hereditary Sensory and Autonomic Neuropathies (3) · Huntington Disease (3) · Mucopolysaccharidosis II (3) · Tic Disorders (3) · Tics (3) · Angiomyolipoma (2) · Bulbo-Spinal Atrophy, X-Linked (2) · Friedreich Ataxia (2) · Lymphangioleiomyomatosis (2) · Obsessive-Compulsive Disorder (2) · Abdominal Neoplasms (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (37) · Nervous System Diseases (37) · Genetic Diseases, Inborn (36) · Neurodegenerative Diseases (31) · Central Nervous System Diseases (19) · Brain Diseases (16) · Neurologic Manifestations (15) · Pathological Conditions, Signs and Symptoms (13) · Congenital Abnormalities (11) · Mental Disorders (11) · Metabolic Diseases (11) · Nutritional and Metabolic Diseases (11) · Basal Ganglia Diseases (10) · Movement Disorders (10)
Everolimus (3) · idursulfase (3) · Riluzole (2) · Adrenergic beta-Antagonists (1) · Aripiprazole (1) · Bupropion (1) · Calcitriol (1) · Cannabidiol (1) · Citalopram (1) · Clonidine (1) · Cycloserine (1) · Cysteamine (1) · Dexmedetomidine (1) · Doxycycline (1) · Fluoxetine (1)
Novartis Pharmaceuticals (3) · NYU Langone Health (2) · Shire (2) · All India Institute of Medical Sciences, Bhubaneswar (1) · Berta Alemany (1) · Brigham and Women's Hospital (1) · Children's Hospital of Fudan University (1) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (1) · HLB Pharmaceutical Co., Ltd. (1) · Harvey S. Singer (1) · Hospices Civils de Lyon (1) · Indiana University (1) · Jazz Pharmaceuticals (1) · Jinling Hospital, China (1) · Jody Corey-Bloom, MD, PhD (1)
United States (20) · China (5) · Poland (3) · United Kingdom (3) · Brazil (2) · Germany (2) · India (2) · Spain (2) · Taiwan (2) · Thailand (2) · Argentina (1) · Austria (1) · Belgium (1) · Bulgaria (1) · Canada (1)
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) · Craig A. Erickson, MD (1) · Debasish Hota, MD, DM (1) · Emily Stern, PhD (1) · George J. Brewer (1) · Lawrence D Scahill, MSN, PhD (1)
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 24595507, PMID 24841414, PMID 24997880, PMID 25205361, PMID 25681004, PMID 25902842, PMID 30782512, PMID 32316911, PMID 32740676, PMID 33305808, PMID 38428428, PMID 41291951, PMID 42435131
2023 (2), 2014 (1), R01MH045802 (1), R24AG064025 (1), R34MH085844 (1), T32GM007748 (1)