19 completed, 3 recruiting, 3 unknown, 2 not yet recruiting. 32 Phase 2, 5 Phase 1, 4 Phase 3.
Follow Cerebellar Ataxia: new trials by email Literature Review of Cerebellar Ataxia trials Research Report on Cerebellar 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| Efficacy and Safety Study of Stemchymal® in Polyglutamine Spinocerebellar Ataxia Stemchymal®, Excipients - Cerebellar Ataxia | Phase 2 | Completed | Steminent Biotherapeutics Inc. | 56 | 2015-09-01 | 2022-10-27 |
| 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 |
| Study To Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta in Patients With Machado-Joseph Disease Cabaletta for IV infusion once weekly during 24 weeks - Machado-Joseph Disease / Spinocerebellar Ataxia 3 | Phase 2 | Completed | Bioblast Pharma Ltd. | 15 | 2014-07-01 | 2016-11-22 |
| The Influence of Deep TMS on Cerebellar Signs in Patients With Machado Joseph Disease Deep TMS - Machado Joseph Disease (SCA3) | Phase 2 | Completed | Brainsway | 20 | 2014-02-01 | 2018-01-25 |
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Spinocerebellar Ataxias (13) · Ataxia Telangiectasia (10) · Machado-Joseph Disease (9) · Ataxia (4) · Spinocerebellar Degenerations (4) · Blast Crisis (1) · Breast Neoplasms (1) · Burkitt Lymphoma (1) · Carcinoma, Non-Small-Cell Lung (1) · Carcinoma, Ovarian Epithelial (1) · Congenital Abnormalities (1) · Endometrial Neoplasms (1) · Friedreich Ataxia (1) · Genetic Diseases, Inborn (1)
Brain Diseases (32) · Central Nervous System Diseases (32) · Cerebellar Diseases (32) · Dyskinesias (32) · Nervous System Diseases (32) · Neurologic Manifestations (32) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (30) · Genetic Diseases, Inborn (29) · Ataxia (28) · Heredodegenerative Disorders, Nervous System (21) · Neurodegenerative Diseases (21) · Spinal Cord Diseases (20) · Spinocerebellar Degenerations (16) · Spinocerebellar Ataxias (14)
Immunoglobulins, Intravenous (2) · Lithium Carbonate (2) · Riluzole (2) · nicotinamide-beta-riboside (2) · 4-phenylbutyric acid (1) · Buprenorphine (1) · Caspofungin (1) · Cell- and Tissue-Based Therapy (1) · Excipients (1) · IB1001 (1) · Infusions, Intravenous (1) · Injections, Spinal (1) · Morphine (1) · Niacinamide (1) · Oxycodone (1)
Hospital de Clinicas de Porto Alegre (2) · S. Andrea Hospital (2) · University of South Florida (2) · Beijing Tide Pharmaceutical Co., Ltd (1) · BioDelivery Sciences International (1) · Bioblast Pharma Ltd. (1) · Biohaven Pharmaceuticals, Inc. (1) · Brainsway (1) · Cadent Therapeutics (1) · Chaitanya Hospital, Pune (1) · Changhua Christian Hospital (1) · European Organisation for Research and Treatment of Cancer - EORTC (1) · Federico II University (1) · General Hospital of Chinese Armed Police Forces (1) · IntraBio Inc (1)
United States (10) · Germany (4) · Italy (4) · Brazil (3) · Israel (3) · Taiwan (3) · United Kingdom (3) · Australia (2) · China (2) · France (2) · Netherlands (2) · South Korea (2) · Spain (2) · Belgium (1) · Denmark (1)
Carlos Gordon, Prof. (2) · Theresa Zesiewicz, MD (2) · Alessandro Filla, MD (1) · Christelle Achkar, MD (1) · Claudio Viscoli, MD (1) · David Coman, MBBS FRACP (1) · Giovanni Ristori, MD (1) · Hilde L Nilsen (1) · Hong Jiang (1) · Jonas AM Saute, MD (1) · Kon Chu, MD, PhD (1) · Laura B Jardim, MD PhD (1) · Laura B Jardim, PhD (1)
PMID 16763896, PMID 16934683, PMID 19841031, 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 34349180, PMID 34620022, PMID 38489195, PMID 38615323, PMID 39009709, PMID 40739200