7 completed, 6 recruiting, 4 unknown, 2 terminated. 22 Phase 1, 8 Phase 2.
Follow Ataxia: new trials by email Literature Review of Ataxia trials Research Report on Ataxia
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 |
|---|---|---|---|---|---|---|
| Safety and Efficacy of Mutation-targeted Precision Genetic Therapy for Ataxia-Telangiectasia (A-T) Antisense oligonucleotide targeting the ATM gene - Ataxia Telangiectasia | Phase 1 / Phase 2 | Recruiting | Timothy Yu | 10 | 2026-08-01 | 2026-05-28 |
| Evaluation of the Safety and Preliminary Efficacy of Neuroinduced Mesenchymal Stem Cells and Exosome Therapy in Patients With Spinocerebellar Palsy Intrathecal infusion, Intranasal infusion - Spinocerebellar Ataxia - All Sub-types | Phase 1 / Phase 2 | Not yet recruiting | Biocells Medical | 90 | 2026-06-01 | 2026-03-12 |
| An Phase Ib/II Clinical Trial of TCC1727 Combination Therapy in Advanced Solid Tumors TCC1727 tablet 90mg, TCC1727 tablet 120mg, TCC1727 tablet 160mg - Solid Cancers, NSCLC (Advanced Non-small Cell Lung Cancer) | Phase 1 / Phase 2 | Recruiting | Beijing Tide Pharmaceutical Co., Ltd | 266 | 2025-12-03 | 2026-01-28 |
| A Study of SGT-212 Gene Therapy in Friedreich's Ataxia SGT-212 - Friedreich's Ataxia (FA) | Phase 1 | Recruiting | Solid Biosciences Inc. | 10 | 2025-10-22 | 2026-09-29 |
| Study of ARO-ATXN2 Injection in Adults With Spinocerebellar Ataxia Type 2 ARO-ATXN2 Injection, Placebo - Spinocerebellar Ataxia Type 2 | Phase 1 | Active not recruiting | Arrowhead Pharmaceuticals | 39 | 2024-12-17 | 2026-06-29 |
| Transplantation of Human iPS Cell-derived Dopaminergic Progenitors (CT1-DAP001) for Parkinson's Disease (Phase I/II) Human induced pluripotent stem cell-derived dopaminergic progenitors (CT1-DAP001) - PD - Parkinson's Disease | Phase 1 | Recruiting | University of California, San Diego | 7 | 2024-06-01 | 2026-08-17 |
| A Safety and Pharmacokinetics Trial of VO659 in SCA1, SCA3 and HD VO659 - Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 3 | Phase 1 / Phase 2 | Recruiting | Vico Therapeutics B. V. | 68 | 2023-02-14 | 2026-08-19 |
| Comparative Bioavailability of Betamethasone Oral Solution Metered Spray (GTX-102) in Healthy Subjects GTX-102 medium dose fast Period 1 and Period 2, GTX-102 medium dose slow Period 1 and Period 2, GTX-102 high dose fast Period 1 and Period 2 - Ataxia Telangiectasia | Phase 1 | Unknown | Grace Therapeutics Inc. | 48 | 2022-09-13 | 2023-01-23 |
| Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN, Prednisone - Friedreich Ataxia, Cardiomyopathies | Phase 1 | Recruiting | Weill Medical College of Cornell University | 25 | 2022-02-22 | 2026-09-28 |
| A Pharmacokinetics and Safety Study of BIIB132 in Adults With Spinocerebellar Ataxia 3 BIIB132, BIIB132-Matching Placebo - Spinocerebellar Ataxia Type 3 | Phase 1 | Terminated | Biogen | 8 | 2022-02-02 | 2023-08-04 |
| Promoting Physical Activity Engagement for People With Early-stage Cerebellar Ataxia Physical activity coaching - Cerebellar Ataxia | Phase 1 | Unknown | Teachers College, Columbia University | 30 | 2021-03-26 | 2024-05-03 |
| Study to Test How Well Patients With Advanced Solid Tumors Respond to Treatment With the Elimusertib in Combination With Pembrolizumab, to Find the Optimal Dose for Patients, How the Drug is Tolerated and the Way the Body Absorbs, Distributes and Discharges the Drug Elimusertib (BAY1895344), Pembrolizumab (Keytruda®) - Advanced Solid Tumors | Phase 1 | Completed | Bayer | 56 | 2019-09-30 | 2024-04-02 |
| A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome dantrolene sodium - Wolfram Syndrome, Diabetes Mellitus | Phase 1 / Phase 2 | Completed | Washington University School of Medicine | 21 | 2017-01-01 | 2024-03-13 |
| A TAK-831-1001, Single and Multiple Rising Dose Study in Healthy Participants TAK-831 Oral Suspension, TAK-831 Placebo, TAK-831 Tablet - Schizophrenia, Cerebellar Ataxia | Phase 1 | Terminated | Neurocrine Biosciences | 110 | 2015-09-23 | 2021-06-14 |
| An Open-label Trial of Intravenous Immune Globulin (IVIG)in Treating Spinocerebellar Ataxias Intravenous Immune Globulin (IVIG) - Spinocerebellar Ataxias | Phase 1 | Unknown | University of South Florida | 10 | 2015-04-01 | 2016-06-08 |
| Clinical Study to Evaluate the Safety and Efficacy BMMNC in Cerebellar Ataxia Intravenous transfer of of Bone Marrow derived Mono Nuclear Stem Cell (BMMNCs) - Cerebellar Ataxia | Phase 1 / Phase 2 | Unknown | Chaitanya Hospital, Pune | 20 | 2014-09-01 | 2014-09-17 |
| AMES Treatment of the Proximal Arm in Chronic Stroke 30 PAAD treatments - Stroke, Cerebrovascular Accident | Phase 1 | Withdrawn | Oregon Health and Science University | 2013-08-01 | 2019-03-29 | |
| A Safety, Pharmacokinetic and Pharmacodynamic Study of Kevetrin in Patients With Advanced Solid Tumors thioureidobutyronitrile - Solid Tumors | Phase 1 | Completed | Cellceutix Corporation | 48 | 2012-10-01 | 2016-02-24 |
| Treatment of Cerebellar Ataxia With Mesenchymal Stem Cells Allogeneic adult adipose-derived mesenchymal stem cells - Cerebellar Ataxia | Phase 1 / Phase 2 | Completed | National Yang Ming Chiao Tung University | 7 | 2012-05-01 | 2014-09-12 |
| Lithium Treatment for Patients With Spinocerebellar Ataxia Type I Lithium Carbonate - Spinocerebellar Ataxia Type I | Phase 1 | Completed | National Institute of Neurological Disorders and Stroke (NINDS) | 13 | 2008-05-18 | 2019-12-17 |
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Spinocerebellar Ataxias (5) · Ataxia Telangiectasia (4) · Cerebellar Ataxia (4) · Friedreich Ataxia (2) · Machado-Joseph Disease (2) · Movement Disorders (2) · Neurodegenerative Diseases (2) · Autoimmune Diseases (1) · Brain Diseases (1) · Carcinoma (1) · Carcinoma, Non-Small-Cell Lung (1) · Cardiomegaly (1) · Cardiomyopathies (1) · Central Nervous System Diseases (1)
Nervous System Diseases (22) · Neurologic Manifestations (22) · Dyskinesias (21) · Central Nervous System Diseases (18) · Brain Diseases (17) · Cerebellar Diseases (16) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (13) · Genetic Diseases, Inborn (13) · Pathological Conditions, Signs and Symptoms (12) · Signs and Symptoms (12) · Neurodegenerative Diseases (11) · Cerebellar Ataxia (10) · Heredodegenerative Disorders, Nervous System (10) · Spinal Cord Diseases (8)
BAY 1895344 (1) · Dantrolene (1) · Immunoglobulins, Intravenous (1) · Lithium Carbonate (1) · Prednisone (1) · Rituximab (1) · pembrolizumab (1)
Arrowhead Pharmaceuticals (1) · Bayer (1) · Beijing Tide Pharmaceutical Co., Ltd (1) · Biocells Medical (1) · Biogen (1) · Cellceutix Corporation (1) · Chaitanya Hospital, Pune (1) · Grace Therapeutics Inc. (1) · National Institute of Allergy and Infectious Diseases (NIAID) (1) · National Institute of Neurological Disorders and Stroke (NINDS) (1) · National Pediatric Neuroinflammation Organization, Inc. (1) · National Yang Ming Chiao Tung University (1) · Neurocrine Biosciences (1) · Oregon Health and Science University (1) · Solid Biosciences Inc. (1)
United States (14) · Germany (4) · United Kingdom (4) · Canada (2) · France (2) · Israel (2) · Netherlands (2) · Spain (2) · Taiwan (2) · Australia (1) · China (1) · Denmark (1) · India (1) · Italy (1) · New Zealand (1)
Barry Oken, MD (1) · Christelle Achkar, MD (1) · Fumihiko Urano, MD (1) · Geoffrey Shapiro, MD PhD (1) · Grisel J Lopez, M.D. (1) · Janice Faulknor, MD (1) · Joseph Ciacci, MD (1) · Michael R Pranzatelli, M.D. (1) · Paul J Cordo, Ph.D (1) · Ronald G Crystal, MD (1) · Samia J. Khoury, MD (1) · Theresa Zesiewicz, MD (1)
PMID 18794494, PMID 19838774, PMID 20606544, PMID 21211990, PMID 34185708, PMID 37171121, PMID 38489195, PMID 38615323, PMID 40739200, PMID 41171420, PMID 42307907
2015 (1), 2018 (1), R21DK113487 (1), R61HL151355 (1)