Condition (MeSH)
Machado-Joseph Disease
ClinicalTrials.gov lists 32 trials for Machado-Joseph Disease or a narrower condition beneath it.
Search trials, drugs, companies and investigators, and follow each one to the others. Individual plan, $249/seat/mo. Card required.
Trials per phase
32 of the 32 trials are tagged with Machado-Joseph Disease itself; the rest are tagged with a narrower condition beneath it.
When did Machado-Joseph Disease trials start? (14)
Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year.
Trials over time, by phase
- Phase 1
- Phase 1/2
- Phase 2
- Phase 2/3
- Phase 3
- Not applicable
- Observational
Top of scale: 5
Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.
| Year | Trials started |
|---|---|
| 2026 (partial year) | 2 |
| 2023 | 2 |
| 2022 | 3 |
| 2021 | 1 |
| 2020 | 1 |
| 2019 | 4 |
| 2018 | 4 |
| 2017 | 5 |
| 2014 | 3 |
| 2012 | 1 |
Showing 10 of 14 earlier years.
No forecast is made for this condition: it averages 1.2 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: 1 trial still running at the 2026-10-09 snapshot, fewer than the 5 needed for a median. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Machado-Joseph Disease trials in? (7)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 1 |
| Phase 1/2 | 1 |
| Phase 2 | 6 |
| Phase 2/3 | 2 |
| Phase 3 | 2 |
| Not applicable | 10 |
| Observational | 10 |
How many Machado-Joseph Disease trials are recruiting? (7)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 2 |
| Recruiting | 3 |
| Active, not recruiting | 2 |
| Completed | 14 |
| Terminated | 2 |
Showing 5 of 7 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Machado-Joseph Disease trials? (6)
Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.
| Sponsor | Trials |
|---|---|
| Bioblast Pharma Ltd. | 1 |
| Biogen | 1 |
| Biohaven Pharmaceuticals, Inc. | 1 |
| Brainsway | 1 |
| Cadent Therapeutics | 1 |
Showing 5 of 6 sponsors.
Which drugs are being tested for Machado-Joseph Disease? (4)
Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.
| Drug | Trials |
|---|---|
| Trehalose | 2 |
| BHV-4157 | 2 |
| Maltose | 1 |
| CAD-1883 | 1 |
Interventions as named in the registry (11)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Intravenous Infusion | 1 |
| VO659 | 1 |
| BIIB132 | 1 |
| varenicline free base | 1 |
| 4-Phenylbutyrate | 1 |
Showing 5 of 11 interventions.
These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.
Which serious adverse events do Machado-Joseph Disease trials report? (10)
Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.
| Adverse event | Subjects affected | Trials |
|---|---|---|
| Suicide attempt | 2 | 1 |
| Urinary tract infection | 2 | 1 |
| Abscess | 1 | 1 |
| Asthenia | 1 | 1 |
| COVID-19 | 1 | 1 |
Showing 5 of 10 adverse events.
Which publications cover Machado-Joseph Disease? (15)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Transcranial alternating current stimulation for treating spinocerebellar ataxia type 3: A randomized controlled trial. | Cell reports. Medicine | 2025 | Yes |
| Fixel-Based Analysis of Diffusion Imaging as a Quantitative Marker of Disease State in Spinocerebellar Ataxia. | Annals of clinical and translational neurology | 2025 | Yes |
| Baseline Clinical and Blood Biomarkers in Patients With Preataxic and Early-Stage Disease Spinocerebellar Ataxia 1 and 3. | Neurology | 2023 | Yes |
| Regional Brain and Spinal Cord Volume Loss in Spinocerebellar Ataxia Type 3. | Movement disorders : official journal of the Movement Disorder Society | 2021 | Yes |
| Cerebellar transcranial direct current stimulation in spinocerebellar ataxia type 3 (SCA3-tDCS): rationale and protocol of a randomized, double-blind, sham-controlled study. | BMC neurology | 2019 | Yes |
Showing 5 of 15 publications.
Prevalence (9)
Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.
| Orphanet disease | Type | Class | Geography | Jurisdiction | Mapping relation |
|---|---|---|---|---|---|
| Spinocerebellar ataxia type 3 | Point prevalence | 1-9 / 100 000 | Portugal | Not stated | Exact |
| Spinocerebellar ataxia type 3 | Point prevalence | 1-9 / 100 000 | Worldwide | Not stated | Exact |
| Spinocerebellar ataxia type 3 | Point prevalence | 1-9 / 100 000 | Japan | Not stated | Exact |
| Spinocerebellar ataxia type 3 | Point prevalence | 1-9 / 100 000 | Japan | Not stated | Exact |
| Spinocerebellar ataxia type 3 | Point prevalence | 1-9 / 100 000 | Portugal | Not stated | Exact |
Showing 5 of 9 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Spinocerebellar ataxia type 3 | 98757 | Exact | Orphadata Science knowledge files, Product 1 |
| Spinocerebellar ataxia type 3 | 98757 | Exact | Orphanet Rare Disease Ontology (ORDO) |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: sub-conditions in mesh.