10 completed, 5 active not recruiting, 4 terminated, 1 recruiting. 20 Phase 3, 5 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 20 | Completed 10, Active not recruiting 5, Terminated 4, Recruiting 1 |
| Phase 2 | 5 | Completed 3, Active not recruiting 1, Terminated 1 |
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 |
|---|---|---|---|---|---|---|
| Pivotal Study of N-acetyl-L-leucine for CACNA1A N-Acetyl-L-Leucine, Placebo - CACNA1A, Spinocerebellar Ataxia Type 6 | Phase 3 | Active not recruiting | IntraBio Inc | 60 | 2026-08-17 | 2026-11-03 |
| A Randomized, Parallel-arm, Double Blind, Placebo-controlled Study to Assess the Efficacy of Fampridine for Patients With Spinocerebellar Ataxia SCA27B Caused by a GAA Expansion in the FGF14 Gene Fampridine 10 mg prolonged-release tablet (per os), Placebo (tablets per os) - Spinocerebellar Ataxia 27B (SCA27B) | Phase 3 | Recruiting | Assistance Publique - Hôpitaux de Paris | 70 | 2025-10-21 | 2025-12-10 |
| 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 |
| A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T) N-Acetyl-L-Leucine, Placebo - Ataxia-Telangiectasia, Ataxia-Telangiectasia (A-T) | Phase 3 | Active not recruiting | IntraBio Inc | 60 | 2025-03-18 | 2025-07-03 |
| Open-Label Extension of EryDex Study IEDAT-04-2022 Dexamethasone sodium phosphate - Ataxia Telangiectasia | Phase 3 | Terminated | Quince Therapeutics S.p.A. | 101 | 2024-12-11 | 2026-05-05 |
| Evaluate the Neurological Effects of EryDex on Subjects With A-T Dexamethasone sodium phosphate, Placebo - Ataxia Telangiectasia | Phase 3 | Completed | Quince Therapeutics S.p.A. | 105 | 2024-06-24 | 2026-04-30 |
| 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 |
| Tllsh2910 for Ataxia and Gut Microbiota Alteration in Patients of Multiple System Atrophy Tllsh2910, Placebo - Ataxia, Cerebellar, Multiple System Atrophy | Phase 3 | Terminated | National Taiwan University Hospital | 18 | 2019-04-02 | 2023-04-07 |
| Troriluzole in Adult Participants With Spinocerebellar Ataxia troriluzole, Placebo - Spinocerebellar Ataxias, Spinocerebellar Ataxia Type 1 | Phase 3 | Active not recruiting | Biohaven Pharmaceuticals, Inc. | 299 | 2019-03-08 | 2026-06-24 |
| Extension Treatment Using EryDex System in Patients With AT Who Participated in the ATTeST-IEDAT-02-2015 Study EryDex System - Ataxia Telangiectasia, Genetic Syndrome | Phase 3 | Terminated | Quince Therapeutics S.p.A. | 104 | 2018-06-12 | 2024-10-08 |
| Clinical Trial With Riluzole in Spinocerebellar Ataxia Type 2 (ATRIL) Riluzole, Placebo - Spinocerebellar Ataxia Type 2 | Phase 3 | Completed | Assistance Publique - Hôpitaux de Paris | 42 | 2018-01-17 | 2021-06-21 |
| Open Pilot Trial of BHV-4157 BHV-4157 - Spinocerebellar Ataxias, Spinocerebellar Ataxia Type 1 | Phase 3 | Active not recruiting | University of California, Los Angeles | 24 | 2017-12-15 | 2024-05-09 |
| 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 |
| A Confirmatory Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD) KPS-0373, High dose, KPS-0373, Low dose, Placebo - Spinocerebellar Degeneration | Phase 3 | Completed | Kissei Pharmaceutical Co., Ltd. | 374 | 2013-10-09 | 2018-12-14 |
| Safety and Efficacy of Lithium Carbonate in Patients With Spinocerebellar Ataxia Type 3 Lithium Carbonate, Placebo - Spinocerebellar Ataxia Type 3, Machado Joseph Disease | Phase 2 / Phase 3 | Completed | Hospital de Clinicas de Porto Alegre | 62 | 2011-05-01 | 2013-01-30 |
| Efficacy of Riluzole in Hereditary Cerebellar Ataxia riluzole, Placebo comparator - Cerebellar Ataxia | Phase 2 / Phase 3 | Completed | S. Andrea Hospital | 60 | 2010-04-01 | 2015-04-21 |
| Conjugate Pneumococcal Vaccine in Ataxia Telangiectasia (AT) Conjugated pneumococcal vaccine (Prevenar) - Ataxia Telangiectasia (AT) | Phase 3 | Completed | Institute of Child Health | 30 | 2006-06-01 | 2008-04-11 |
| A Long-Term Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD) KPS-0373, High dose, KPS-0373, Low dose - Spinocerebellar Degeneration | Phase 3 | Completed | Kissei Pharmaceutical Co., Ltd. | 2016-04-18 | ||
| A 24-week Open-label Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD) KPS-0373, High dose, KPS-0373, Low dose - Spinocerebellar Degeneration | Phase 3 | Completed | Kissei Pharmaceutical Co., Ltd. | 2016-04-18 | ||
| An Extension Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD) KPS-0373, High dose, KPS-0373, Low dose - Spinocerebellar Degeneration | Phase 3 | Completed | Kissei Pharmaceutical Co., Ltd. | 2016-04-18 |
Spinocerebellar Ataxias (11) · Ataxia Telangiectasia (5) · Machado-Joseph Disease (4) · Spinocerebellar Degenerations (4) · Cerebellar Ataxia (2) · Congenital disorder of glycosylation type 1A (1) · Diplopia (1) · Episodic Ataxia, Type 2 (1) · Friedreich Ataxia (1) · Genetic Diseases, Inborn (1) · Heart Arrest (1) · Multiple System Atrophy (1) · Spinocerebellar Ataxia 10 (1) · Spinocerebellar ataxia 8 (1)
Dyskinesias (20) · Nervous System Diseases (20) · Neurologic Manifestations (20) · Brain Diseases (18) · Central Nervous System Diseases (18) · Cerebellar Diseases (18) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (17) · Cerebellar Ataxia (16) · Genetic Diseases, Inborn (16) · Neurodegenerative Diseases (13) · Heredodegenerative Disorders, Nervous System (12) · Spinal Cord Diseases (12) · Spinocerebellar Degenerations (8) · Cardiovascular Diseases (6)
Riluzole (2) · acetylleucine (2) · dexamethasone 21-phosphate (2) · 4-Aminopyridine (1) · Double-Blind Method (1) · Heptavalent Pneumococcal Conjugate Vaccine (1) · IB1001 (1) · Lithium Carbonate (1)
Kissei Pharmaceutical Co., Ltd. (4) · Quince Therapeutics S.p.A. (3) · Assistance Publique - Hôpitaux de Paris (2) · Biohaven Pharmaceuticals, Inc. (2) · IntraBio Inc (2) · Glycomine, Inc. (1) · Hospital de Clinicas de Porto Alegre (1) · Institute of Child Health (1) · National Taiwan University Hospital (1) · S. Andrea Hospital (1) · Seelos Therapeutics, Inc. (1) · University of California, Los Angeles (1)
United States (10) · United Kingdom (8) · Germany (7) · Italy (6) · Spain (6) · Japan (4) · Poland (4) · Switzerland (4) · France (3) · Norway (3) · Australia (2) · Belgium (2) · Brazil (2) · Denmark (2) · Portugal (2)
Dirk Thye, MD (2) · Chun-Hwei Tai (1) · Guenter R Janhofer, MD, PhD (1) · Jonas AM Saute, MD (1) · Laura B Jardim, MD PhD (1) · Silvia Romano, MD, PhD (1) · Tatsuro Takei (1)
PMID 34115419, PMID 24399647, PMID 26297649, PMID 26321318, PMID 31937586, PMID 35063116, PMID 38814532, PMID 39806095, PMID 39917433, PMID 42309084, PMID 42600624
2017 (1), 2018 (1), 2025 (1)