73 recruiting. 73 Phase 2, 32 Phase 1, 5 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 73 | Recruiting 73 |
| Phase 1 | 32 | Recruiting 32 |
| Phase 3 | 5 | Recruiting 5 |
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 |
|---|---|---|---|---|---|---|
| Neoadjuvant Pembrolizumab and Porustobart in Resectable pMMR/MSS Colon Cancer Pembrolizumab, Porustobart - Colon Cancer | Phase 2 | Recruiting | Solstice Oncology, Inc. | 100 | 2026-09-30 | 2026-10-27 |
| A Study of the Safety and Efficacy of Prime Editing (PM577) in Participants With Wilson Disease (WD) PM577a - Wilson Disease, Wilson's Disease | Phase 1 / Phase 2 | Recruiting | Prime Medicine, Inc. | 42 | 2026-09-01 | 2026-10-01 |
| A Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents With Rett Syndrome Rett-T, Placebo - Rett Syndrome | Phase 2 | Recruiting | Holland Bloorview Kids Rehabilitation Hospital | 35 | 2026-08-24 | 2026-09-02 |
| NORTHERA (DROXIDOPA) for Dysautonomia in Pediatric Survivors of Menkes Disease Droxidopa Oral Product, Placebo Control - Menkes Disease | Phase 1 / Phase 2 | Recruiting | Stephen G. Kaler | 6 | 2026-08-10 | 2026-10-05 |
| Trial of Selumetinib and Bromodomain Inhibitor With Durvalumab for Sarcomas Selumetinib + ZEN-3694 ± Durvalumab - MPNST, NF1 | Phase 2 | Recruiting | University of Alabama at Birmingham | 41 | 2026-08-04 | 2026-08-24 |
| A Study to Evaluate ARV-6723 Alone and With Pembrolizumab in Participants With Advanced Solid Tumors ARV-6723, Pembrolizumab, SOC - Advanced Solid Tumor | Phase 1 / Phase 2 | Recruiting | Arvinas Inc. | 499 | 2026-07-29 | 2026-10-13 |
| A Phase I/II Clinical Study to Evaluate SKG0201 Injection in Subjects With Spinal Muscular Atrophy Type I SKG0201 Injection - Spinal Muscular Atrophy 1 | Phase 1 / Phase 2 | Recruiting | Lanyue Biotech (Hangzhou) Co., Ltd. | 11 | 2026-07-15 | 2026-10-14 |
| Safety, Tolerability, and Pharmacokinetics of SVG103 (Paxalisib) in Focal Cortical Dysplasia Type II (FCD-II), Tuberous Sclerosis Complex (TSC) or Hemimegalencephaly (HME) SVG103 - Focal Cortical Dysplasia, Tuberous Sclerosis Complex (TSC) | Phase 1 / Phase 2 | Recruiting | Sovargen | 12 | 2026-07-14 | 2026-08-10 |
| Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy LX2006, Usual Care - Friedreich Ataxia, Cardiomyopathy, Secondary | Phase 2 | Recruiting | Lexeo Therapeutics | 26 | 2026-06-25 | 2026-09-02 |
| Early Phase Study Evaluating MEK and MDM2 Inhibition in Patients With NF1 and MPNST APG-115, Selumetinib - Malignant Peripheral Nerve Sheath Tumor (MPNST), Neurofibromatosis 1 (NF1) | Phase 1 / Phase 2 | Recruiting | AeRang Kim | 45 | 2026-06-01 | 2026-07-27 |
| Safety, Tolerability, and Preliminary Effectiveness of CTH120 in Fragile X Syndrome CTH120, CTH120 placebo hard capsules - Fragile X Syndrome (FXS), Fragile X Syndrome | Phase 2 | Recruiting | Connecta Therapeutics, S.L. | 30 | 2026-05-21 | 2026-07-17 |
| Sapanisertib and Serabelisib (PIKTOR) in Various Combinations in Patients With HR+/HER2- Advanced/Metastatic Breast Cancer Serabelisib, Sapanisertib, Fulvestrant - HR+ HER2- Breast Cancer | Phase 1 / Phase 2 | Recruiting | Faeth Therapeutics | 32 | 2026-04-22 | 2026-07-02 |
| Safety and Tolerability Study of Human Neural Stem Cells for Huntington's Disease hNSC-01 - Huntington Disease | Phase 1 / Phase 2 | Recruiting | Leslie Thompson | 21 | 2026-04-06 | 2026-04-13 |
| A Phase II Study of Sintilimab Combined With Ipilimumab N01, Cetuximab and Dabrafenib in Patients With Microsatellite-Stable, BRAF V600E-Mutated Metastatic Colorectal Cancer Ipilimumab N01, Sintilimab, Cetuximab - BRAF V600E, Colorectal Cancer | Phase 2 | Recruiting | Tianjin Medical University Cancer Institute and Hospital | 49 | 2026-03-01 | 2026-04-07 |
| Efficacy and Safety Study of Bilateral IVT Injection of GS010 at Two Dose Levels in LHON Patients GS010 High dose, GS010 Low dose - Leber Hereditary Optic Disease | Phase 2 | Recruiting | GenSight Biologics | 14 | 2026-01-22 | 2026-03-02 |
| TulmiSTAR-02: A Phase I/II Open-label Study of Tulmimetostat in Combination With Darolutamide vs. Darolutamide, and Tulmimetostat With Abiraterone in Patients With Metastatic Hormone-sensitive Prostate Cancer (mHSPC) Tulmimetostat, Darolutamide, Abiraterone - Metastatic Hormone-Sensitive Prostate Cancer (mHSPC) | Phase 1 / Phase 2 | Recruiting | Novartis Pharmaceuticals | 181 | 2026-01-13 | 2026-09-29 |
| Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1 GB221 - Spinal Muscular Atrophy Type I | Phase 1 / Phase 2 | Recruiting | Gemma Biotherapeutics | 22 | 2026-01-06 | 2026-06-29 |
| Study to Evaluate the Pharmacodynamics, Safety and Efficacy of SKY-0515 in Participants With Huntington's Disease SKY-0515, SKY-0515 Placebo - Huntington Disease | Phase 2 / Phase 3 | Recruiting | Skyhawk Therapeutics, Inc. | 400 | 2026-01-06 | 2026-10-29 |
| An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE) PGN-EDODM1 - Myotonic Dystrophy 1 | Phase 2 | Recruiting | PepGen Inc | 48 | 2025-12-23 | 2026-03-30 |
| Study of MRM-3379 in Male Participants With Fragile X Syndrome (BLOOM) Low dose of MRM-3379, Middle Dose of MRM-3379, High dose of MRM-3379 - Fragile X Syndrome | Phase 2 | Recruiting | Mirum Pharmaceuticals, Inc. | 60 | 2025-11-22 | 2026-09-29 |
Showing 20 of 73. Sign in to see all of them.
Hereditary Sensory and Autonomic Neuropathies (14) · Neurofibromatosis 1 (8) · Huntington Disease (7) · Myotonic Dystrophy (5) · Bulbo-Spinal Atrophy, X-Linked (4) · Endometrial Neoplasms (4) · Fragile X Syndrome (4) · Ovarian Neoplasms (4) · Spinal Muscular Atrophies of Childhood (4) · Tourette Syndrome (4) · Breast Neoplasms (3) · Colorectal Neoplasms (3) · Friedreich Ataxia (3) · Neoplasms (3) · Neurodevelopmental Disorders (3)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (73) · Genetic Diseases, Inborn (71) · Nervous System Diseases (71) · Neurodegenerative Diseases (64) · Neuromuscular Diseases (37) · Central Nervous System Diseases (31) · Peripheral Nervous System Diseases (25) · Brain Diseases (23) · Neoplasms (22) · Congenital Abnormalities (21) · Nervous System Malformations (18) · Neoplasms by Histologic Type (16) · Polyneuropathies (15) · Metabolic Diseases (14)
AZD 6244 (3) · Fulvestrant (3) · abemaciclib (3) · fludarabine (3) · Alpelisib (2) · Melphalan (2) · fludarabine phosphate (2) · pembrolizumab (2) · sapanisertib (2) · serabelisib (2) · Acetylcysteine (1) · Alemtuzumab (1) · Anastrozole (1) · Androgen Antagonists (1) · Azacitidine (1)
Faeth Therapeutics (2) · Holland Bloorview Kids Rehabilitation Hospital (2) · National Cancer Institute (NCI) (2) · PepGen Inc (2) · University of Alabama at Birmingham (2) · University of Minnesota (2) · AMO Pharma Limited (1) · ARTHEx Biotech S.L. (1) · Acrivon Therapeutics (1) · AeRang Kim (1) · AllRock Bio, Inc. (1) · Artios Pharma Ltd (1) · Arvinas Inc. (1) · Aspa Therapeutics (1) · Assistance Publique - Hôpitaux de Paris (1)
United States (41) · Canada (13) · France (12) · Spain (12) · United Kingdom (10) · Australia (8) · China (8) · Italy (7) · Germany (6) · Netherlands (5) · Belgium (4) · New Zealand (4) · Brazil (3) · South Korea (3) · Argentina (2)
AeRang Kim, MD, PhD (1) · Amy Xu, MD, PhD (1) · Antonio Gonzalez, MD, PHD (1) · Berge Minassian, MD (1) · Bin Li, MD (1) · Brigitte C Widemann, M.D. (1) · Carolyn Orr (1) · Christine Conelea (1) · Christine Conelea, PhD, LP (1) · Clement Loy (1)
PMID 32634238, PMID 32750127, PMID 38489195, PMID 38615323, PMID 38904277, PMID 39806603
2025 (5), 2023 (3)