224 active not recruiting. 224 Phase 3, 39 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 224 | Active not recruiting 224 |
| Phase 2 | 39 | Active not recruiting 39 |
Follow Nervous System Diseases: new trials by email Literature Review of Nervous System Diseases trials Research Report on Nervous System Diseases
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 |
|---|---|---|---|---|---|---|
| Pivotal Study of N-acetyl-L-leucine for CACNA1A N-Acetyl-L-Leucine, Placebo - CACNA1A, Spinocerebellar Ataxia Type 6 | Phase 3 | Active not recruiting | IntraBio Inc | 60 | 2026-08-17 | 2026-11-03 |
| An Open Label Extension (OLE) Study (Following Completion of CTQJ230A12301) to Evaluate Long-term Safety and Tolerability of Pelacarsen (TQJ230) Pelacarsen (TQJ230) - Cardiovascular Disease and Lipoprotein(a) | Phase 3 | Active not recruiting | Novartis Pharmaceuticals | 5700 | 2026-05-11 | 2026-10-14 |
| Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome TSHA-102 - Rett Syndrome | Phase 3 | Active not recruiting | Taysha Gene Therapies, Inc. | 4 | 2026-05-08 | 2026-08-19 |
| Phase Ⅲ Trial of Group ACYW135 Meningococcal Conjugate Vaccine in Children Aged 2-6 Years experimental vaccine, active comparator vaccine - Epidemic Meningitis, Meningococcal Vaccines | Phase 3 | Active not recruiting | Sinovac Biotech Co., Ltd | 660 | 2026-03-22 | 2026-08-10 |
| HEALEY ALS Platform Trial - Regimen I NUZ-001 NUZ-001, Matching placebo - Amyotrophic Lateral Sclerosis (ALS) | Phase 2 / Phase 3 | Active not recruiting | Merit E. Cudkowicz, MD | 240 | 2026-02-25 | 2026-07-20 |
| Randomized Clinical Trial Evaluating the Efficacy and Safety of Vormatrigine in Adults With Focal Seizures 40 mg/day vormatrogine for 12 weeks, 30 mg/day vormatrogine for 12 weeks, 20 mg/day vormatrogine for 12 weeks - Focal Epilepsy | Phase 3 | Active not recruiting | Praxis Precision Medicines | 300 | 2026-01-29 | 2026-08-03 |
| A Trial of TAK-861 for the Treatment of Narcolepsy With Cataplexy TAK-861, Placebo - Narcolepsy Type 1 (NT1), Narcolepsy With Cataplexy | Phase 3 | Active not recruiting | Takeda | 98 | 2026-01-29 | 2026-10-30 |
| Maridebart Cafraglutide Versus Placebo in Adult Participants With Obstructive Sleep Apnea on Positive Airway Pressure Therapy Maridebart cafraglutide, Placebo - Obesity, Obstructive Sleep Apnea | Phase 3 | Active not recruiting | Amgen | 250 | 2025-12-19 | 2026-10-06 |
| Efficacy and Safety of Interleukin-6 Receptor Inhibitor Combined With Endovascular Treatment in Patients With Acute Anterior Circulation Large Vessel Occlusion Stroke -2 Tocilizumab, Placebo - Acute Ischemic Stroke | Phase 3 | Active not recruiting | Capital Medical University | 692 | 2025-12-05 | 2026-10-06 |
| Maridebart Cafraglutide Versus Placebo in Adult Participants With Obstructive Sleep Apnea Not on Positive Airway Pressure (PAP) Therapy Maridebart cafraglutide, Placebo - Obstructive Sleep Apnea | Phase 3 | Active not recruiting | Amgen | 250 | 2025-11-25 | 2026-09-29 |
| RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome) RGX-121-3102 - MPS II, Hunter Syndrome (MPS II) | Phase 3 | Active not recruiting | REGENXBIO Inc. | 2 | 2025-11-25 | 2026-06-04 |
| A Study to Learn About Different Dosing Schedules of a Lyme Disease Vaccine in Healthy Adults VLA15, Placebo - Lyme Borreliosis | Phase 3 | Active not recruiting | Pfizer | 207 | 2025-11-19 | 2026-05-06 |
| A Clinical Trial of Trontinemab in Participants With Early Symptomatic Alzheimer's Disease Trontinemab, Placebo - Alzheimers Disease | Phase 3 | Active not recruiting | Hoffmann-La Roche | 1060 | 2025-11-12 | 2026-09-25 |
| SPI-1005 for the Treatment of Meniere's Disease (Open Label) Ebselen - Meniere's Disease, Ménière's Disease | Phase 3 | Active not recruiting | Sound Pharmaceuticals, Incorporated | 200 | 2025-10-29 | 2026-07-13 |
| Study of COYA 302 for the Treatment of ALS COYA 302, Placebo - Amyotrophic Lateral Sclerosis (ALS) | Phase 2 / Phase 3 | Active not recruiting | Coya Therapeutics | 120 | 2025-10-01 | 2026-10-13 |
| A Study of Trontinemab in Participants With Early Symptomatic Alzheimer's Disease Trontinemab, Placebo - Alzheimers Disease | Phase 3 | Active not recruiting | Hoffmann-La Roche | 1101 | 2025-09-17 | 2026-09-25 |
| Effect of Different Anesthetic Drugs on Electrocorticography (ECOG). Fentanyl (IV), Ketamine (0.5 mg/kg) - Epilepsy, Brain Tumor | Phase 3 | Active not recruiting | Children's Cancer Hospital Egypt 57357 | 88 | 2025-08-18 | 2025-09-10 |
| Confirmatory Study 3 of KPS-0373 in Patients With Spinocerebellar Degeneration KPS-0373, Placebo - Spinocerebellar Degeneration | Phase 3 | Active not recruiting | Kissei Pharmaceutical Co., Ltd. | 142 | 2025-08-15 | 2026-09-04 |
| Air Versus Lignocaine for Endotracheal Tube Cuff Inflation and Effect on Postoperative Sore Throat and Intracuff Pressure Xylocaine - Postoperative Sore Throat, Cough, Hoarseness | Phase 2 / Phase 3 | Active not recruiting | PAEC General Hospital, Islamabad | 80 | 2025-08-02 | 2026-01-02 |
| A Clinical Trial for Participants With DEE to Assess Efficacy, Safety, Tolerability, and PK of Relutrigine 1.0mg/kg/day PRAX-562, 1.5mg/kg/day PRAX-562, Placebo - Developmental and Epileptic Encephalopathy 1 | Phase 3 | Active not recruiting | Praxis Precision Medicines | 160 | 2025-07-09 | 2026-08-04 |
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Alzheimer Disease (17) · Brain Neoplasms (14) · Multiple Sclerosis (14) · Myasthenia Gravis (12) · Dementia (11) · Cognitive Dysfunction (9) · Mucopolysaccharidosis II (7) · Multiple Sclerosis, Chronic Progressive (7) · Multiple Sclerosis, Relapsing-Remitting (7) · Epilepsy (6) · Genetic Diseases, X-Linked (6) · Ischemic Stroke (6) · Neurodevelopmental Disorders (6) · Neurologic Manifestations (6)
Central Nervous System Diseases (113) · Brain Diseases (95) · Pathological Conditions, Signs and Symptoms (74) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (61) · Neurodegenerative Diseases (60) · Genetic Diseases, Inborn (54) · Immune System Diseases (49) · Neurologic Manifestations (46) · Autoimmune Diseases (44) · Autoimmune Diseases of the Nervous System (43) · Neoplasms (43) · Signs and Symptoms (43) · Nutritional and Metabolic Diseases (41) · Neuromuscular Diseases (38)
ocrelizumab (13) · Radiosurgery (8) · Magnetic Resonance Spectroscopy (4) · Methylprednisolone (4) · Radiotherapy (4) · Vincristine (4) · donanemab (4) · lecanemab (4) · Cyclophosphamide (3) · Dexamethasone (3) · Fingolimod Hydrochloride (3) · Prednisolone (3) · Temozolomide (3) · idursulfase (3) · venglustat (3)
Hoffmann-La Roche (9) · Novartis Pharmaceuticals (8) · Sanofi (8) · Eli Lilly and Company (6) · M.D. Anderson Cancer Center (6) · Amgen (5) · Alexion Pharmaceuticals, Inc. (4) · NRG Oncology (4) · Biogen (3) · Capital Medical University (3) · Ionis Pharmaceuticals, Inc. (3) · Pfizer (3) · REGENXBIO Inc. (3) · Washington University School of Medicine (3) · argenx (3)
United States (172) · Canada (91) · Italy (88) · Spain (85) · United Kingdom (83) · France (81) · Germany (79) · Australia (71) · Poland (62) · Japan (57) · Brazil (49) · Belgium (47) · Netherlands (47) · China (44) · Denmark (37)
Randall J Bateman, MD (3) · David Hui (2) · Merit Cudkowicz, MD (2) · Robert J. Fox, MD (2)
PMID 42552749, PMID 38723617, PMID 42208560, PMID 28687375, PMID 28801186, PMID 29262276, PMID 29393183, PMID 30207920, PMID 31086941, PMID 31386061, PMID 31467033, PMID 31587995, PMID 32479786, PMID 33395032, PMID 33395098, PMID 33844429, PMID 34000245, PMID 34115419, PMID 34383289, PMID 34398588, PMID 34427473, PMID 34816762, PMID 34911199, PMID 35246398, PMID 35275197, PMID 36008843, PMID 36341846, PMID 36449413, PMID 36934371, PMID 37009976, PMID 37017737, PMID 37289451, PMID 37459141, PMID 37467180, PMID 37491196, PMID 37717787, PMID 37790359, PMID 37870112, PMID 37977713, PMID 37990597, PMID 38171926, PMID 38216511, PMID 38254921, PMID 38323738, PMID 38499015, PMID 38520467, PMID 38580946, PMID 38581616, PMID 38730496, PMID 38814532
2023 (25), 2018 (7), 2019 (3), 2025 (3), U10CA180868 (3), U10CA180886 (3), 2017 (2), R01AG046179 (2), R01AG053267 (2), R56AG053267 (2), U01AG042791 (2), U01AG059798 (2), 1UG1EY033293 (1), 2014 (1), P30CA016672 (1)