60 active not recruiting. 60 Phase 2, 30 Phase 1, 9 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 60 | Active not recruiting 60 |
| Phase 1 | 30 | Active not recruiting 30 |
| Phase 3 | 9 | Active not recruiting 9 |
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 |
|---|---|---|---|---|---|---|
| A Trial to Learn How Safe AZD9750 is and How Well it Works in People With Metastatic Prostate Cancer When Given With or Without Other Anticancer Drugs AZD9750, AZD5305 - Prostate Cancer | Phase 1 / Phase 2 | Active not recruiting | AstraZeneca | 6 | 2026-01-27 | 2026-10-02 |
| Study of SKY-0515 for Safety, Efficacy, and Pharmacodynamics in Participants With Huntington's Disease SKY-0515, SKY-0515 Placebo - Huntington Disease, Huntingtons Disease | Phase 2 / Phase 3 | Active not recruiting | Skyhawk Therapeutics, Inc. | 120 | 2025-05-27 | 2026-05-01 |
| Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient VCA-894A - Charcot Marie Tooth Disease (CMT), Neuromuscular Diseases (NMD) | Phase 1 / Phase 2 | Active not recruiting | Vanda Pharmaceuticals | 1 | 2025-05-13 | 2026-08-19 |
| Trial of Atezolizumab, Bevacizumab, and Tiragolumab in Patients With Microsatellite Stable, Metastatic Colorectal Cancer Pre-Treatment Biopsy, On-Treatment Biopsy, Tiragolumab - Microsatellite Stable (MSS) Colorectal Cancer (CRC), Metastatic Colorectal Cancer (CRC) | Phase 2 | Active not recruiting | University of Colorado, Denver | 13 | 2025-03-25 | 2025-10-02 |
| A Phase I Clinical Study of ER2001 Injection for the Treatment of Early Manifest Huntington's Disease. ER2001 intravenous injection, Placebo - Huntington Disease | Phase 1 / Phase 2 | Active not recruiting | ExoRNA Bioscience | 27 | 2025-02-11 | 2026-01-14 |
| Efficacy and Safety of Gemlapodect (NOE-105) in Adults and Adolescents With Tourette Syndrome Gemlapodect, Placebo - Tourette Syndrome | Phase 2 | Active not recruiting | Noema Pharma AG | 164 | 2024-09-10 | 2026-03-09 |
| Astroscape: A Study of Radiprodil on Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms in Patients With TSC or FCD Type II Radiprodil - Tuberous Sclerosis Complex, Focal Cortical Dysplasia | Phase 1 / Phase 2 | Active not recruiting | GRIN Therapeutics, Inc. | 30 | 2024-07-10 | 2026-04-01 |
| Pilot Study of Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia Dexmedetomidine Sublingual - Familial Dysautonomia | Phase 2 | Active not recruiting | NYU Langone Health | 5 | 2024-06-01 | 2026-03-04 |
| Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment Idursulfase-IT, Elaprase - Hunter Syndrome | Phase 2 / Phase 3 | Active not recruiting | Takeda | 6 | 2024-03-05 | 2025-11-06 |
| A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1) VX-670, Placebo - Myotonic Dystrophy Type 1 (DM1) | Phase 1 / Phase 2 | Active not recruiting | Vertex Pharmaceuticals Incorporated | 47 | 2024-02-20 | 2026-08-20 |
| Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study) TSHA-102 - Rett Syndrome | Phase 1 / Phase 2 | Active not recruiting | Taysha Gene Therapies, Inc. | 6 | 2023-12-12 | 2025-10-15 |
| An Extension Study to Evaluate the Long-Term Safety and Efficacy of Votoplam in Participants With Huntington's Disease (HD) Votoplam - Huntington Disease | Phase 2 | Active not recruiting | Novartis Pharmaceuticals | 144 | 2023-08-25 | 2025-11-21 |
| Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 2 Patients GC101 - SMA II | Phase 1 / Phase 2 | Active not recruiting | GeneCradle Inc | 33 | 2023-08-01 | 2025-07-03 |
| Safety and Efficacy of the PAINLESS Nerve Growth Factor CHF6467 in Optic Pathway Glioma (OPG) CHF6467, Placebo - Optic Pathway Glioma | Phase 2 | Active not recruiting | Benedetto Falsini | 36 | 2023-06-12 | 2026-10-27 |
| Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type II Autologous CD34+ HSCs transduced ex vivo with CD11B LV encoding human IDS tagged with ApoEII - Mucopolysaccharidosis II | Phase 1 / Phase 2 | Active not recruiting | University of Manchester | 5 | 2023-06-01 | 2026-03-27 |
| Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND4 Mutations NR082 Injection, Injection needle - Leber Hereditary Optic Neuropathy (LHON) | Phase 1 / Phase 2 | Active not recruiting | Neurophth Therapeutics Inc | 12 | 2023-05-22 | 2024-09-04 |
| AMX0035 in Adult Patients With Wolfram Syndrome AMX0035 - Wolfram Syndrome | Phase 2 | Active not recruiting | Amylyx Pharmaceuticals Inc. | 12 | 2023-03-03 | 2026-04-14 |
| GENERATION HD2. A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared With Placebo in Participants With Prodromal and Early Manifest Huntington's Disease Tominersen, Placebo - Huntington Disease | Phase 2 | Active not recruiting | Hoffmann-La Roche | 301 | 2023-02-03 | 2026-08-10 |
| Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT FBX-101 - Krabbe Disease | Phase 1 / Phase 2 | Active not recruiting | Duke University | 9 | 2023-02-03 | 2026-11-04 |
| A Study to Evaluate AB-1001 Striatal Administration in Adults With Early Manifest Huntington's Disease AB-1001 Gene Therapy - Huntington Disease | Phase 1 / Phase 2 | Active not recruiting | AskBio France, SAS, a subsidiary of AskBio Inc. | 5 | 2022-10-12 | 2026-05-14 |
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Mucopolysaccharidosis II (10) · Neurofibromatosis 1 (9) · Huntington Disease (8) · Hereditary Sensory and Autonomic Neuropathies (6) · Neurofibroma, Plexiform (5) · Genetic Diseases, Inborn (4) · Nervous System Diseases (4) · Sudden Infant Death (4) · Lysosomal Storage Diseases (3) · Neurodegenerative Diseases (3) · Neuronal Ceroid-Lipofuscinoses (3) · Tuberous Sclerosis (3) · Brain Diseases (2) · Breast Neoplasms (2) · Bulbo-Spinal Atrophy, X-Linked (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (60) · Genetic Diseases, Inborn (56) · Nervous System Diseases (56) · Neurodegenerative Diseases (45) · Neuromuscular Diseases (20) · Nutritional and Metabolic Diseases (20) · Neoplasms (19) · Central Nervous System Diseases (18) · Metabolic Diseases (18) · Neurologic Manifestations (17) · Peripheral Nervous System Diseases (17) · Brain Diseases (15) · Congenital Abnormalities (15) · Metabolism, Inborn Errors (15)
AZD 6244 (4) · idursulfase (2) · pembrolizumab (2) · salicylhydroxamic acid (2) · trametinib (2) · 11-(2-pyrrolidin-1-ylethoxy)-14,19-dioxa-5,7,26-triazatetracyclo(19.3.1.1(2,6).1(8,12))heptacosa-1(25),2(26),3,5,8,10,12(27),16,21,23-decaene (1) · 3-(5-fluorobenzofuran-3-yl)-4-(5-methyl-5H-(1,3)dioxolo(4,5-f)indol-7-yl)-pyrrole-2,5-dione (1) · 4-phenylbutyric acid (1) · AZD5305 (1) · Adalimumab (1) · Albumin-Bound Paclitaxel (1) · Aspirin (1) · Baclofen (1) · Bevacizumab (1) · Biopsy (1)
National Cancer Institute (NCI) (4) · AstraZeneca (2) · Denali Therapeutics Inc. (2) · JCR Pharmaceuticals Co., Ltd. (2) · Massachusetts Eye and Ear Infirmary (2) · UniQure Biopharma B.V. (2) · AO GENERIUM (1) · Actuate Therapeutics Inc. (1) · Amylyx Pharmaceuticals Inc. (1) · AskBio France, SAS, a subsidiary of AskBio Inc. (1) · Benedetto Falsini (1) · Brigham and Women's Hospital (1) · Children's Hospital Medical Center, Cincinnati (1) · Darcy Krueger (1) · David L Rogers, MD (1)
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Brigitte C Widemann, M.D. (2) · D. Bradley Welling, MD, PhD (2) · Alejandra Gonzalez-Duarte, MD (1) · Amy E Brown, MD (1) · Anita Scheuber, MD (1) · Benedetto Falsini, MD (1) · Camille Bedrosian, MD (1) · Carmen P Priolo, MD PhD (1) · Craig A. Erickson, M.D. (1) · Darcy A Krueger, MD, PhD (1) · David H. Margolin, MD, PhD (1) · David Margolin, MD, PhD (1)
PMID 36463457, PMID 38489195, PMID 12473512, PMID 21343523, PMID 21444653, PMID 24327518, PMID 25408174, PMID 26767384, PMID 27424289, PMID 28029918, PMID 28053024, PMID 28672195, PMID 29383130, PMID 29846250, PMID 30405781, PMID 30627694, PMID 30975030, PMID 31101621, PMID 31151904, PMID 31401903, PMID 31533931, PMID 31831767, PMID 31881853, PMID 31882719, PMID 31894292, PMID 32187457, PMID 32250312, PMID 33395098, PMID 33536190, PMID 33728588, PMID 35007270, PMID 35467749, PMID 35815408, PMID 36196048, PMID 37035740, PMID 37115514, PMID 37400844, PMID 37614821, PMID 37942487, PMID 38055260, PMID 38058356, PMID 38867349, PMID 38869947, PMID 39499628, PMID 39514826, PMID 39762421, PMID 40235521, PMID 40709016, PMID 40739200, PMID 41139346
2023 (7), 2018 (1), 2019 (1), R01AG064802 (1), R01HD089474 (1), U01CA081457 (1), U10CA180886 (1), U54CA196519 (1), U54HD104461 (1), UM1CA081457 (1)