RecruitingPhase 3NCT07221292
Neurological
Spinocerebellar Ataxia
Also known as SCA, autosomal dominant cerebellar ataxia, ADCA, SCA1/SCA2/SCA3/SCA6/SCA7 and others
Spinocerebellar ataxias (SCAs) are a heterogeneous group of autosomal dominant neurodegenerative disorders characterized by progressive cerebellar ataxia, with over 40 genetic subtypes identified. The most common types (SCA1, SCA2, SCA3, SC
ORPHA:99 ↗Gene ATXN1 (SCA1)Gene ATXN2 (SCA2)Gene ATXN3 (SCA3/MJD)Gene CACNA1A (SCA6)Gene ATXN7 (SCA7)Gene TBP (SCA17)Prevalence 1-5 per 10,000 (Orphanet, all types combined)Onset Adult, AdolescentAutosomal dominant genetic (most types, trinucleotide or other repeat expansion)
42
studies recruiting now
as of 7 Sept 2026
286
studies registered in total
as of 7 Sept 2026
12
countries with a recruiting site
as of 7 Sept 2026
22 Jul 2026
most recent study posted
among recruiting studies
Recruiting trials
RecruitingPhase 2NCT06447025
An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia
RecruitingNCT06865482
Clinical Course Of Disease In Participants With FA-CM
RecruitingPhase 2NCT07721025
Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy
RecruitingNCT00018889
Phenotype/Genotype Correlations in Movement Disorders
Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
See all 42 recruiting studiesWhere recruiting studies are running
Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.
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About Spinocerebellar Ataxia
Spinocerebellar ataxias (SCAs) are a heterogeneous group of autosomal dominant neurodegenerative disorders characterized by progressive cerebellar ataxia, with over 40 genetic subtypes identified. The most common types (SCA1, SCA2, SCA3, SCA6, SCA7) are caused by CAG trinucleotide repeat expansions in their respective genes, encoding polyglutamine tracts that cause toxic protein aggregation. Clinical features beyond cerebellar ataxia vary by subtype and include pyramidal signs, peripheral neuropathy, ophthalmoplegia, and cognitive decline.
Common clinical features
Progressive cerebellar ataxiaDysarthriaNystagmus and oculomotor abnormalitiesPyramidal signs (spasticity, hyperreflexia)Peripheral neuropathyCognitive declineDysphagia
From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.
Treatments being studied
6 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.
Phase 3Rovatirelin
Phase 2/3Troriluzole
Phase 2/3Riluzole (Exservan)
Phase 2Human Immunoglobulin G (Flebogamma dif (previously flebogammadif))
Phase 2Nilotinib
Phase 1Ubidecarenone
Before you apply
Things trial teams commonly ask about for Spinocerebellar Ataxia. Not eligibility rules; those are set by each study.
- SCA subtype must be genetically confirmed — repeat expansion length and subtype are required for all trials
- Scale for Assessment and Rating of Ataxia (SARA) score is the primary eligibility and outcome measure
- CAG repeat length predicts age of onset and progression rate — this affects trial stratification and eligibility windows
- Antisense oligonucleotide (ASO) and gene silencing trials are subtype-specific (e.g., SCA3 ASO trials differ from SCA1)
Related conditions
Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).