26 completed, 4 unknown, 3 not yet recruiting, 3 recruiting. 42 Phase 2, 8 Phase 1, 5 Phase 3.
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 |
|---|---|---|---|---|---|---|
| Umbilical Cord Mesenchymal Stem Cells Therapy (19#iSCLife®-SA) for Patients With Spinocerebellar Ataxia Intravenous infusion, Intrathecal injection, umbilical cord mesenchymal stem cell - Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2 | Phase 2 | Not yet recruiting | Sclnow Biotechnology Co., Ltd. | 45 | 2026-12-31 | 2026-04-23 |
| 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 to Assess the Efficacy and Safety of Weekly Doses of GLM101 in Participants With PMM2-CDG GLM101 (Part A, Double-blind), Placebo (Part A, Double-blind), GLM101 (Part B, Open-label) - Phosphomannomutase 2 Deficiency, PMM2-CDG | Phase 2 / Phase 3 | Active not recruiting | Glycomine, Inc. | 50 | 2025-07-09 | 2026-08-14 |
| Stemchymal® for Polyglutamine Spinocerebellar Ataxia Stemchymal, Placebo - Spinocerebellar Ataxias | Phase 2 | Not yet recruiting | Steminent US, Inc. | 20 | 2025-06-01 | 2024-05-02 |
| Sirolimus Treatment in Pediatric Patients With Ataxia-Telangiectasia Rapamune (sirolimus/rapamycin) - Ataxia-Telangiectasia (A-T), Ataxia-Telangiectasia | Phase 2 | Completed | Sara Elva Espinosa Padilla, MD | 6 | 2024-07-11 | 2026-09-15 |
| 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 |
| STRIDES - a Clinical Research Study of an Investigational New Drug to Treat Spinocerebellar Ataxia SLS-005, Placebo - Spinocerebellar Ataxia Type 3 | Phase 2 / Phase 3 | Terminated | Seelos Therapeutics, Inc. | 23 | 2022-06-03 | 2024-04-24 |
| Ataxia-telangiectasia: Treating Mitochondrial Dysfunction With a Novel Form of Anaplerosis Triheptanoin - Ataxia Telangiectasia | Phase 2 | Completed | The University of Queensland | 30 | 2022-03-15 | 2023-07-20 |
| MBM-01 (Tempol) for the Treatment of Ataxia Telangiectasia MBM-01 - Ataxia Telangiectasia Louis-Bar, Ataxia Telangiectasia in Children | Phase 2 | Unknown | Matrix Biomed, Inc. | 20 | 2021-07-01 | 2021-05-17 |
| Study of CAD-1883 for Spinocerebellar Ataxia CAD-1883, Placebos - Spinocerebellar Ataxias, Spinocerebellar Ataxia Type 1 | Phase 2 | Withdrawn | Cadent Therapeutics | 2021-06-01 | 2021-04-08 | |
| Chinese Medicine WT for Spinocerebellar Ataxia Type 3 Chinese medicine WT - Ataxia, Spinocerebellar, Insulin-Like Growth Factor I | Phase 2 | Completed | Changhua Christian Hospital | 33 | 2021-05-31 | 2023-01-31 |
| Efficacy of Dual Task Training on Children With Ataxia After Medulloblastoma Resection Dual Task Training (Motor + Cognitive), Traditional Regular Exercise - Ataxia, Medulloblastoma | Phase 2 | Unknown | Cairo University | 30 | 2020-10-01 | 2021-11-18 |
| N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T) IB1001 - Ataxia Telangiectasia | Phase 2 | Terminated | IntraBio Inc | 17 | 2020-01-08 | 2026-03-25 |
| NAD Supplementation to Prevent Progressive Neurological Disease in Ataxia Telangiectasia Nicotinamide ribonucleoside - Ataxia Telangiectasia | Phase 2 | Active not recruiting | University Hospital, Akershus | 13 | 2019-06-05 | 2022-08-17 |
| Effects of Vitamin B3 in Patients With Ataxia Telangiectasia Vitamin B3 - Ataxia Telangiectasia, ATM Gene Mutation | Phase 2 | Completed | Radboud University Medical Center | 24 | 2019-03-18 | 2022-07-25 |
| Effect of Nilotinib in Cerebellar Ataxia Patients Nilotinib - Ataxia, Cerebellar, Ataxia, Progressive | Phase 2 | Completed | Seoul National University Hospital | 70 | 2018-11-19 | 2021-09-29 |
| 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 |
| Trial in Adult Participants With Spinocerebellar Ataxia (SCA) Troriluzole, Placebo - Spinocerebellar Ataxias, Spinocerebellar Ataxia Genotype Type 1 | Phase 2 / Phase 3 | Completed | Biohaven Pharmaceuticals, Inc. | 141 | 2016-12-15 | 2025-10-08 |
Showing 20 of 42. Sign in to see all of them.
Spinocerebellar Ataxias (13) · Ataxia Telangiectasia (10) · Machado-Joseph Disease (9) · Cerebellar Ataxia (8) · Friedreich Ataxia (4) · Spinocerebellar Degenerations (4) · Autistic Disorder (2) · Rett Syndrome (2) · Blast Crisis (1) · Breast Neoplasms (1) · Burkitt Lymphoma (1) · Carcinoma, Non-Small-Cell Lung (1) · Carcinoma, Ovarian Epithelial (1) · Congenital Abnormalities (1)
Dyskinesias (42) · Nervous System Diseases (42) · Neurologic Manifestations (42) · Central Nervous System Diseases (36) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (36) · Brain Diseases (35) · Cerebellar Diseases (35) · Genetic Diseases, Inborn (35) · Heredodegenerative Disorders, Nervous System (27) · Neurodegenerative Diseases (26) · Cerebellar Ataxia (24) · Spinal Cord Diseases (23) · Pathological Conditions, Signs and Symptoms (22) · Signs and Symptoms (21)
Immunoglobulins, Intravenous (2) · Lithium Carbonate (2) · Riluzole (2) · nicotinamide-beta-riboside (2) · trofinetide (2) · 4-phenylbutyric acid (1) · Buprenorphine (1) · Caspofungin (1) · Cell- and Tissue-Based Therapy (1) · Dantrolene (1) · Double-Blind Method (1) · Epoetin Alfa (1) · Excipients (1) · IB1001 (1) · Infusions, Intravenous (1)
Federico II University (2) · Hospital de Clinicas de Porto Alegre (2) · National Institute of Neurological Disorders and Stroke (NINDS) (2) · Neuren Pharmaceuticals Limited (2) · S. Andrea Hospital (2) · University of South Florida (2) · Beijing Tide Pharmaceutical Co., Ltd (1) · BioDelivery Sciences International (1) · Bioblast Pharma Ltd. (1) · Biocells Medical (1) · Biohaven Pharmaceuticals, Inc. (1) · Brainsway (1) · Cadent Therapeutics (1) · Cairo University (1) · Chaitanya Hospital, Pune (1)
United States (17) · Italy (6) · Germany (5) · United Kingdom (4) · Brazil (3) · France (3) · Israel (3) · Spain (3) · Taiwan (3) · Australia (2) · Belgium (2) · China (2) · Netherlands (2) · Poland (2) · Portugal (2)
Alan Percy, MD (2) · Carlos Gordon, Prof. (2) · Theresa Zesiewicz, MD (2) · Timothy Feyma, MD (2) · Alessandro Filla, MD (1) · Arthur Beisang, MD (1) · Christelle Achkar, MD (1) · Claudio Viscoli, MD (1) · Daniel G Glaze, M.D. (1) · Daniel Glaze, MD (1) · David Coman, MBBS FRACP (1) · Eric Marsh, MD (1)
PMID 37460385, PMID 38363467, PMID 16763896, PMID 16934683, PMID 17826341, PMID 19838774, PMID 19841031, PMID 20090835, PMID 20599042, PMID 20606544, PMID 21211990, PMID 22144182, PMID 22875082, PMID 22927201, PMID 24236946, PMID 24399647, PMID 25656498, PMID 26297649, PMID 26321318, PMID 26433381, PMID 26547562, PMID 26851119, PMID 28601294, PMID 28871440, PMID 28969699, PMID 29110179, PMID 30586396, PMID 30796268, PMID 30948626, PMID 33437944, PMID 33482890, PMID 34115419, PMID 34185708, PMID 34349180, PMID 34620022, PMID 38489195, PMID 38615323, PMID 39009709, PMID 40739200
R21DK113487 (1)