191 completed. 191 Phase 1, 65 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 191 | Completed 191 |
| Phase 2 | 65 | Completed 65 |
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 |
|---|---|---|---|---|---|---|
| Test-retest Study With [18F]FBB in Cardiac Amyloidosis Florbetaben F18 - Cardiac Amyloidosis | Phase 1 | Completed | Lantheus Germany GmbH | 15 | 2025-10-21 | 2026-10-23 |
| Phase 1, Open-label, Drug-drug Interaction Study to Assess Effect Between Radiprodil and the Co-administered Drugs. Radiprodil + co-administered drugs - Tuberous Sclerosis Complex (TSC), Focal Cortical Dysplasia | Phase 1 | Completed | GRIN Therapeutics, Inc. | 18 | 2025-05-20 | 2025-11-18 |
| Effect of Food on the Pharmacokinetics of LPM3770164 Sustained-release Tablets LPM3770164 sustained release tablet - Huntington Disease, Tardive Dyskinesia | Phase 1 | Completed | Luye Pharma Group Ltd. | 16 | 2024-07-31 | 2024-12-12 |
| Patients With ALS and Other Motor Disorders Will be Treated With Umbilical or Placental Mesenchymal Cell Exosome Solution Exosome Secretome - Motor Disorders | Phase 1 | Completed | The Foundation for Orthopaedics and Regenerative Medicine | 70 | 2024-07-14 | 2026-10-16 |
| Trimetazidine Efficacy in Attenuating Paclitaxel-Induced Peripheral Neuropathy Trimetazidine, Placebo - Peripheral Neuropathy Due to Chemotherapy | Phase 1 / Phase 2 | Completed | Minia University | 60 | 2024-06-23 | 2025-03-30 |
| Tuvusertib (M1774) Human Mass Balance and Absolute Bioavailability Study (DDRIVER Solid Tumors 303) Tuvusertib [14C]Tuvusertib microtracer, Tuvusertib, Tuvusertib + [14C]Tuvusertib microdose bolus injection - Solid Tumor | Phase 1 | Completed | Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany | 12 | 2024-03-25 | 2026-04-23 |
| Efficacy of Skin Cooling in Reducing Pain Associated With Non-invasive Treatments of Neurofibromatosis Type 1 Cutaneous Neurofibromas Deoxycholic Acid, Polidocanol, 1064nm Nd:YAG laser - Neurofibromatosis 1 | Phase 1 | Completed | Massachusetts General Hospital | 13 | 2024-03-01 | 2025-11-21 |
| Study Comparing Once Daily Dose of 900mg of TETA 4HCL Against Cuprior® (450mg Trientine Base, Twice Daily). 900mg TETA 4HCl Once Daily Formulation, 900mg TETA 4HCl Cuprior® - Wilson's Disease | Phase 1 | Completed | Orphalan | 26 | 2024-01-16 | 2026-08-26 |
| Safety, Tolerability and Pharmacokinetic of Multiple-ascending Doses of LPM3770164 in Healthy Subjects LPM3770164 sustained release tablet, LPM3770164 sustained release tablet simulant - Tardive Dyskinesia, Huntington Disease | Phase 1 | Completed | Luye Pharma Group Ltd. | 40 | 2023-12-19 | 2024-12-12 |
| Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1 PGN-EDODM1 for infusion, Placebo - Myotonic Dystrophy 1 | Phase 1 | Completed | PepGen Inc | 24 | 2023-12-12 | 2026-02-13 |
| Assessing the Safety and Efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) in the Treatment of Rett Syndrome (RTT) NTI164 - Rett Syndrome | Phase 1 / Phase 2 | Completed | Fenix Innovation Group | 14 | 2023-09-20 | 2026-09-09 |
| Tuvusertib (M1774) in Combination With Cemiplimab in Participants With Non-Squamous NSCLC (DDRiver NSCLC 322) M1774, Cemiplimab - Non-Small Cell Lung Cancer | Phase 1 / Phase 2 | Completed | EMD Serono Research & Development Institute, Inc. | 61 | 2023-09-13 | 2026-09-14 |
| Single and Multiple Ascending Dose Study of MTR-601 in Healthy Individuals MTR-601 - Muscle Spasticity, Hypertonia, Muscle | Phase 1 | Completed | Motric Bio | 89 | 2023-09-11 | 2026-06-05 |
| CTH120 First-in-Human Study: Single and Multiple Ascending Doses and Potential Food Interaction CTH120, Placebo - Fragile X Syndrome | Phase 1 | Completed | Connecta Therapeutics, S.L. | 76 | 2023-06-23 | 2025-01-23 |
| The Safety and Tolerability of CLZ-2002 in Patients With Charcot-Marie Tooth Disease. CLZ-2002 - Charcot Marie Tooth Disease, Type 1 | Phase 1 | Completed | Cellatoz Therapeutics, Inc | 9 | 2023-06-19 | 2025-02-17 |
| A Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers verenafusp alfa 6 mg/kg, verenafusp alfa 9 mg/kg, verenafusp alfa 12 mg/kg - Mucopolysaccharidosis Type II, Metabolic Diseases | Phase 1 | Completed | AO GENERIUM | 18 | 2023-04-06 | 2024-07-03 |
| A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Of AJ201 In Patients AJ201, Placebo - Spinal and Bulbar Muscular Atrophy, Kennedy's Disease | Phase 1 / Phase 2 | Completed | AnnJi Pharmaceutical Co., Ltd. | 25 | 2023-02-28 | 2024-08-15 |
| Dosing and Tolerability of Deoxycholic Acid vs. Polidocanol in the Treatment of Neurofibromatosis Type 1 Cutaneous Neurofibromas Kybella, Asclera - Neurofibromatosis 1 | Phase 1 | Completed | Massachusetts General Hospital | 20 | 2022-12-06 | 2025-02-21 |
| Study to Evaluate Multiple Ascending Dose and Multi-Dose of DT-216 in Adult Patients With Friedreich Ataxia DT-216, Placebo - Friedreich Ataxia | Phase 1 | Completed | Design Therapeutics, Inc. | 32 | 2022-09-27 | 2024-02-29 |
| DPCP to Treat Cutaneous Neurofibromas Associated With NF1 DPCP - Cutaneous Neurofibroma | Phase 1 | Completed | Nicholas Gulati | 17 | 2022-09-14 | 2024-05-06 |
Showing 20 of 191. Sign in to see all of them.
Huntington Disease (32) · Hereditary Sensory and Autonomic Neuropathies (27) · Neurofibromatosis 1 (14) · Friedreich Ataxia (13) · Fragile X Syndrome (12) · Mucopolysaccharidosis II (12) · Alzheimer Disease (11) · Tuberous Sclerosis (11) · Neuronal Ceroid-Lipofuscinoses (9) · Tourette Syndrome (9) · Bulbo-Spinal Atrophy, X-Linked (7) · Amyloid Neuropathies, Familial (6) · Genetic Diseases, Inborn (6) · Hepatolenticular Degeneration (6) · Myotonic Dystrophy (6)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (191) · Nervous System Diseases (188) · Genetic Diseases, Inborn (185) · Neurodegenerative Diseases (156) · Central Nervous System Diseases (87) · Brain Diseases (77) · Neuromuscular Diseases (69) · Nutritional and Metabolic Diseases (60) · Metabolic Diseases (57) · Peripheral Nervous System Diseases (55) · Congenital Abnormalities (54) · Movement Disorders (47) · Neoplasms (47) · Basal Ganglia Diseases (46)
latrepirdine (8) · Everolimus (6) · Sirolimus (4) · 6-chloro-2,3,4,9-tetrahydro-1H-carbazole-1-carboxamide (3) · Deoxycholic Acid (3) · PX-866 (3) · tetrathiomolybdate (3) · 5-(5-((2,4-dimethylthiazol-5-yl)methyl)-1,3,4-oxadiazol-2-yl)-1-ethyl-N-(tetrahydro-2H-pyran-4-yl)-1H-pyrazolo(3,4-b)pyridin-4-amine (2) · AZD 6244 (2) · Lasers, Solid-State (2) · Magnetic Resonance Spectroscopy (2) · Polidocanol (2) · berzosertib (2) · cerliponase alfa (2) · ecopipam (2)
Pfizer (9) · Children's Hospital Medical Center, Cincinnati (7) · AstraZeneca (6) · Massachusetts General Hospital (5) · National Institute of Neurological Disorders and Stroke (NINDS) (5) · Alexion Pharmaceuticals, Inc. (4) · National Cancer Institute (NCI) (4) · Cascadian Therapeutics Inc. (3) · Luye Pharma Group Ltd. (3) · Siena Biotech S.p.A. (3) · Stanford University (3) · University of Rochester (3) · Weill Medical College of Cornell University (3) · AO GENERIUM (2) · BioMarin Pharmaceutical (2)
United States (138) · United Kingdom (23) · Germany (15) · Canada (14) · Spain (11) · France (10) · Japan (8) · Australia (6) · Italy (6) · China (4) · Netherlands (4) · South Korea (4) · Belgium (3) · Switzerland (3) · Denmark (2)
Brigitte C Widemann, M.D. (2) · David Russell, MD (2) · Richard R Anderson, MD (2) · A. Reghan Foley, M.D. (1) · Ahmed Mostafa Abd-Elaziz, Lecturer (1) · Alan Hand, MD (1) · Allan L Reiss (1) · An N. Dang Do, MD, PhD (1) · Andrew Stephens, MD, PhD (1)
PMID 17562928, PMID 29688815, PMID 33980287, PMID 37171121, PMID 38101904, PMID 38776275, PMID 15923132, PMID 16278898, PMID 17482283, PMID 18256395, PMID 18473686, PMID 18625864, PMID 18752978, PMID 19194885, PMID 20497190, PMID 20652413, PMID 20837825, PMID 21047224, PMID 21482669, PMID 21764821, PMID 22134099, PMID 22262746, PMID 22934754, PMID 23325902, PMID 23497636, PMID 23581634, PMID 23798472, PMID 24296492, PMID 24327173, PMID 24398093, PMID 24457989, PMID 24878825, PMID 25223836, PMID 25281031, PMID 25342621, PMID 26336641, PMID 26381530, PMID 26606867, PMID 27072954, PMID 27631475, PMID 28647203, PMID 29023494, PMID 29066505, PMID 29091557, PMID 29480210, PMID 29725840, PMID 29766026, PMID 30153790, PMID 30194931, PMID 30390395
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