Machado-Joseph Disease clinical trials
The registry lists 33 studies for Machado-Joseph Disease across more than 10 countries. 5 are recruiting or about to start. 30 of them are mainly about Machado-Joseph Disease (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-12.
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Who runs them
Hospital de Clinicas de Porto Alegre (5) · First Affiliated Hospital of Fujian Medical University (2) · Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia (2) · National University of Malaysia (2) · Vico Therapeutics B. V. (1) · Lauren Moore (1) · University of Mississippi Medical Center (1) · Meir Medical Center (1)
What is being tested here
Drugs under test in Machado-Joseph Disease studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
| Drug | Open | Phase 3 | All |
|---|---|---|---|
| Troriluzole | 0 | 2 | 2 |
| Trehalose | 0 | 0 | 2 |
| Lithium Carbonate | 0 | 1 | 1 |
| Varenicline | 0 | 0 | 1 |
| SLS-005 | 0 | 1 | 1 |
| Phenylbutyric acid | 0 | 0 | 1 |
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 2
- Nov 2026Optimizing Parameters of Transcranial Temporal Interference Stimulation for Spinocerebellar Ataxia Type 3 · First Affiliated Hospital of Fujian Medical University
- Jun 2027Umbilical Cord Mesenchymal Stem Cells Therapy (19#iSCLife®-SA) for Patients With Spinocerebellar Ataxia · Sclnow Biotechnology Co., Ltd.
Who these studies take
Of the 3 Machado-Joseph Disease studies recruiting now, 2 (67%) set no upper age limit and 1 (33%) stop at 75 or younger. 2 (67%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 1 (33%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 2 (67%) | 2 (67%) |
| 18 | 2 (67%) | 2 (67%) |
| 30 | 3 (100%) | 3 (100%) |
| 40 | 3 (100%) | 3 (100%) |
| 50 | 3 (100%) | 3 (100%) |
| 65 | 2 (67%) | 2 (67%) |
| 75 | 2 (67%) | 2 (67%) |
| 85 | 2 (67%) | 2 (67%) |
Across all 33 studies, recruiting or not: 36% stop at 75 or younger and 12% at 65; 21% take children; 3% are for women only and 0% for men only; 27% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
Studies
Recruiting first, then most recently updated. Showing 33 of 33. search them.
- Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at SanfordRecruitingNCT017931681 country
- Optimizing Parameters of Transcranial Temporal Interference Stimulation for Spinocerebellar Ataxia Type 3Not yet recruitingNCT07743866Not applicable1 country
- A Safety and Pharmacokinetics Trial of VO659 in SCA1, SCA3 and HDRecruitingNCT05822908Phase 1/26 countries
- Natural History Study of and Genetic Modifiers in Spinocerebellar AtaxiasRecruitingNCT010603712 countries
- Umbilical Cord Mesenchymal Stem Cells Therapy (19#iSCLife®-SA) for Patients With Spinocerebellar AtaxiaNot yet recruitingNCT03378414Phase 2
- Troriluzole in Adult Participants With Spinocerebellar AtaxiaCompletedNCT03701399Phase 32 countries
- NIPD on CFTC for Triplet Repeat DiseasesCompletedNCT03087526Not applicable1 country
- Instrumented Data Exchange for Ataxia StudyCompletedNCT042681471 country
- Treatment of Transcranial Alternating Current Stimulation(tACS)on Cerebellar AtaxiaCompletedNCT05557786Not applicable1 country
- Open Pilot Trial of BHV-4157Active, not recruitingNCT03408080Phase 31 country
- Priming Motor Learning Through Exercise in People With Spinocerebellar AtaxiaActive, not recruitingNCT05826171Not applicable1 country
- STRIDES - a Clinical Research Study of an Investigational New Drug to Treat Spinocerebellar AtaxiaTerminatedNCT05490563Phase 2/38 countries
- Identification of Biomarkers in Spinocerebellar Ataxia 3CompletedNCT038851671 country
- A Pharmacokinetics and Safety Study of BIIB132 in Adults With Spinocerebellar Ataxia 3TerminatedNCT05160558Phase 16 countries
- Repetitive Transcranial Magnetic Stimulation in SCA3 PatientsCompletedNCT05502432Not applicable1 country
- Efficacy Of Oral Trehalose In Spinocerebellar Ataxia 3Status unknownNCT04399265Not applicable1 country
- Clinical Trial Readiness for SCA1 and SCA3Status unknownNCT034873671 country
- Study of CAD-1883 for Spinocerebellar AtaxiaWithdrawnNCT04301284Phase 21 country
- Cerebello-Spinal tDCS as Rehabilitative Intervention in Neurodegenerative AtaxiaCompletedNCT04153110Not applicable1 country
- Neuropsychiatry and Cognition in SCA3/MJDStatus unknownNCT047143071 country
- Natural History of Oculomotor Neurophysiology in Ataxic and Pre-ataxic Carriers of SCA3/MJDStatus unknownNCT042298231 country
- Clinical Effects of Oral Trehalose In Patients With Spinocerebellar Ataxia 3CompletedNCT04426149Not applicable1 country
- Astrocytic Markers and the Pre-ataxic Period of SCA3/MJD - BIGPRO Study AstrocytesStatus unknownNCT044199741 country
- Rehabilitative Trial With Cerebello-Spinal tDCS in Neurodegenerative AtaxiaCompletedNCT03120013Not applicable1 country
- The Influence of Deep TMS on Cerebellar Signs in Patients With Machado Joseph DiseaseCompletedNCT02039206Phase 21 country
- Machado-Joseph Disease in IsraelStatus unknownNCT021752901 country
- Study To Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta in Patients With Machado-Joseph DiseaseCompletedNCT02147886Phase 21 country
- Weight in Lower Limbs Improves Gait Ataxia of in Machado-Joseph Disease PatientsCompletedNCT02906046Not applicable
- Dalfampridine and Gait in Spinocerebellar AtaxiasCompletedNCT01811706Not applicable1 country
- Safety and Efficacy of Lithium Carbonate in Patients With Spinocerebellar Ataxia Type 3CompletedNCT01096082Phase 2/31 country
- Pilot Study of Safety and Efficacy of Sodium Phenylbutyrate in Spinocerebellar Ataxia Type 3WithdrawnNCT01096095Phase 21 country
- Study to Determine the Safety and Tolerability of Varenicline (Chantix®) in Treating Spinocerebellar Ataxia Type 3CompletedNCT00992771Phase 21 country
- Measuring Neurological Impairment and Functional Visual Assessment In Spinocerebellar AtaxiasCompletedNCT006542511 country