Treatment and research

Open trials and recent research

Live from the public registry and the biomedical literature. Nothing on this page is written by us — it is the raw record, tidied up and made readable.

175

Registered studies found

Currently open to enrolment

123

Testing an intervention

Registry data retrieved 5 August 2026 · refreshed automatically twice daily

Left: a randomised trial splits one group in two at the same time. Right: an external-control design compares against separate data — practical when a disease is rare, harder to trust.

Registered studies

37 studies

Studies currently recruiting or about to

RecruitingObservational1400 participants
Natural History Study of and Genetic Modifiers in Spinocerebellar Ataxias
Run by
Lauren Moore
Registry ID
NCT01060371
Testing
Genetic Testing, Blood Collection, Magnetic Resonance Imaging (MRI) Scan, Assessments and Questionnaires
Started
April 2010
Sites in
Canada, United States
RecruitingObservational100 participants
Characterization of the Cardiac Phenotype of Friedreich's Ataxia (FRDA)
Run by
Weill Medical College of Cornell University
Registry ID
NCT02316314
Testing
Cardiac magnetic resonance imaging (CMR), Exercise-stress test, Echocardiogram (ECHO), Cardiac-related blood studies
Started
January 2015
Sites in
United States
RecruitingPhase 226 participants
Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy
Run by
Lexeo Therapeutics
Registry ID
NCT07721025
Testing
LX2006, Usual Care
Started
June 2026
Sites in
United States
RecruitingNot applicable160 participants
Autosomal Dominant Spinocerebellar Ataxias and Social Cognition
Run by
University Hospital, Angers
Registry ID
NCT07099651
Testing
tests
Started
December 2025
Sites in
France
RecruitingNot applicable5 participants
Interposed Nucleus aDBS for Ataxia
Run by
University of Florida
Registry ID
NCT07325487
Testing
Deep Brain Stimulation
Started
June 2026
Sites in
United States
Not yet recruitingObservational56 participants
Biomarkers for Babies and Young Children With Ataxia Telangiectasia
Run by
University of Nottingham
Registry ID
NCT07709728
Started
September 2026
RecruitingNot applicable5 participants
Deep Brain Stimulation for Spinocerebellar Ataxia
Run by
University of California, San Francisco
Registry ID
NCT07288437
Testing
Deep Brain Stimulation (DBS)
Started
April 2026
Sites in
United States
RecruitingObservational10 participants
A Study to Learn How BIIB141 (Omaveloxolone) Affects the Health of Participants With Friedrich's Ataxia Who Took it During Pregnancy and/or During Breastfeeding and About the Health of Their Babies
Run by
Biogen
Registry ID
NCT06628687
Testing
Omaveloxolone
Started
October 2026
Sites in
United States
Not yet recruitingPhase 3120 participants
Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)
Run by
PTC Therapeutics
Registry ID
NCT07681713
Testing
Vatiquinone
Started
August 2026
Enrolling by invitationObservational60 participants
GABA and GSH in FRDA
Run by
Children's Hospital of Philadelphia
Registry ID
NCT07635030
Testing
MRI/ MRS (Magnetic Resonance Imaging /Magnetic Resonance Spectroscopy)
Started
May 2024
Sites in
United States
RecruitingObservational65 participants
Clinical Course Of Disease In Participants With FA-CM
Run by
Lexeo Therapeutics
Registry ID
NCT06865482
Started
September 2025
Sites in
Brazil, Canada, Czechia, France, Germany, Italy, Spain, United States
RecruitingPhase 285 participants
An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia
Run by
Larimar Therapeutics, Inc.
Registry ID
NCT06447025
Testing
CTI-1601
Started
January 2024
Sites in
United States
RecruitingObservational40 participants
Efficacy of Omaveloxolone Treatment for Dysphagia in French Patients With Friedreich's Ataxia
Run by
Centre Hospitalier Universitaire de Nice
Registry ID
NCT07013292
Testing
SKYCLARYS (omaveloxolone)
Started
June 2025
Sites in
France
RecruitingObservational300 participants
A Study to Learn More About the Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Who Are Prescribed it by Their Own Doctors
Run by
Biogen
Registry ID
NCT06623890
Testing
Omaveloxolone
Started
December 2024
Sites in
Austria, Belgium, Czechia, Germany, Italy, Netherlands, United States
Not yet recruitingPhase 245 participants
Umbilical Cord Mesenchymal Stem Cells Therapy (19#iSCLife®-SA) for Patients With Spinocerebellar Ataxia
Run by
Sclnow Biotechnology Co., Ltd.
Registry ID
NCT03378414
Testing
Intravenous infusion, Intrathecal injection, umbilical cord mesenchymal stem cell
Started
December 2026
RecruitingPhase 125 participants
Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia
Run by
Weill Medical College of Cornell University
Registry ID
NCT05302271
Testing
AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN, Prednisone
Started
February 2022
Sites in
United States
Not yet recruitingPhase 1/Phase 290 participants
Evaluation of the Safety and Preliminary Efficacy of Neuroinduced Mesenchymal Stem Cells and Exosome Therapy in Patients With Spinocerebellar Palsy
Run by
Biocells Medical
Registry ID
NCT07467733
Testing
Intrathecal infusion, Intranasal infusion
Started
June 2026
Sites in
Poland
Not yet recruitingNot applicable30 participants
Cardiac Output and Fatigue in Friedreich's Ataxia
Run by
Scott Barbuto
Registry ID
NCT07444333
Testing
Aerobic Exercise, Omeveloxolone
Started
April 2026
AvailablePhase not stated
Expanded Access Program (EAP) of Levacetylleucine for Ataxia-Telangiectasia (A-T)
Run by
IntraBio Inc
Registry ID
NCT07380165
Testing
Levacetylleucine
Started
—
AvailablePhase not stated
Expanded Access Protocol of Troriluzole in Patients With Spinocerebellar Ataxia (SCA)
Run by
Biohaven Pharmaceuticals, Inc.
Registry ID
NCT06034886
Testing
Troriluzole
Started
—
Not yet recruitingNot applicable24 participants
Telerehabilitation for Core Stability and Strength in Hereditary Ataxia
Run by
Universitat de Lleida
Registry ID
NCT07200505
Testing
TR Mixed CORE Exercices, TR Asyncronous CORE Exercices
Started
February 2026
Sites in
Spain
RecruitingObservational203 participants
Biomarkers in Friedreich's Ataxia
Run by
University of Florida
Registry ID
NCT02497534
Started
September 2015
Sites in
United States
Enrolling by invitationObservational108 participants
Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders
Run by
LMU Klinikum
Registry ID
NCT06605612
Testing
FBIndex
Started
September 2024
Sites in
Germany
RecruitingPhase 1/Phase 268 participants
A Safety and Pharmacokinetics Trial of VO659 in SCA1, SCA3 and HD
Run by
Vico Therapeutics B. V.
Registry ID
NCT05822908
Testing
VO659
Started
February 2023
Sites in
Denmark, France, Germany, Israel, Netherlands, United Kingdom
RecruitingNot applicable300 participants
Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology
Run by
Centre Hospitalier Universitaire de Liege
Registry ID
NCT07136844
Testing
Syde, Dynamometric measurements of muscle strength, The 6-minute walk test (6MWT), The 10-meter test (10MWT)
Started
March 2024
Sites in
Belgium
Not yet recruitingObservational64 participants
Structural Validity and Inter-rater Reliabitiliy of the Ataxia Trunk, Lower And Upper Extremity Scale (ATLAS)
Run by
Haute Ecole de Santé Vaud
Registry ID
NCT07079332
Testing
New Scale to evaluate ataxia motor symptoms
Started
September 2025
RecruitingNot applicable30 participants
Electroencephalogram in Patients With Friedreich's Ataxia for the Study of the Structural and Functional Connectome.
Run by
IRCCS Eugenio Medea
Registry ID
NCT07095062
Testing
HD-EEG recordings, combined with cognitive and motor assessment
Started
July 2025
Sites in
Italy
RecruitingObservational5000 participants
Hereditary Ataxia Research on Multi-Omics and Neuroclinical Insights in the Yangtze Delta
Run by
Second Affiliated Hospital, Zhejiang University, School of Medicine
Registry ID
NCT07092358
Testing
None of intervention
Started
June 2025
Sites in
China
Enrolling by invitationNot applicable30 participants
The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.
Run by
LMU Klinikum
Registry ID
NCT07072676
Testing
Assistive gait devices combined with physiotherapy
Started
September 2025
Sites in
Germany
RecruitingObservational20000 participants
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Run by
Sanford Health
Registry ID
NCT01793168
Started
July 2010
Sites in
Australia, United States
RecruitingPhase 1/Phase 220 participants
A Multiple Ascending Dose Study of DT-216P2 in Patients With Friedreich's Ataxia
Run by
Design Therapeutics, Inc.
Registry ID
NCT06874010
Testing
DT-216P2
Started
June 2025
Sites in
Australia
Not yet recruitingNot applicable80 participants
Efficacy of Cerebellar Transcranial Magnetic Stimulation to Treat Hereditary Spinocerebellar Ataxias
Run by
Xijing Hospital
Registry ID
NCT06898645
Testing
navigated iTBS (Intermittent theta-burst stimulation) targeting the cerebellum
Started
March 2025
Not yet recruitingPhase 136 participants
A Single Ascending Dose Study of DT-216P2 in Normal Healthy Participants
Run by
Design Therapeutics, Inc.
Registry ID
NCT06772870
Testing
DT-216P2, Saline
Started
February 2025
Sites in
Australia
RecruitingPhase 2/Phase 334 participants
Riluzole in Patients With Spinocerebellar Ataxia Type 7
Run by
S. Andrea Hospital
Registry ID
NCT03660917
Testing
Riluzole, Placebo
Started
June 2021
Sites in
Italy
RecruitingObservational300 participants
Hong Kong Spinocerebellar Ataxias Registry
Run by
Chinese University of Hong Kong
Registry ID
NCT03336008
Testing
no intervention
Started
December 2012
Sites in
Hong Kong
Not yet recruitingPhase 220 participants
Stemchymal® for Polyglutamine Spinocerebellar Ataxia
Run by
Steminent US, Inc.
Registry ID
NCT06397274
Testing
Stemchymal, Placebo
Started
June 2025
RecruitingObservational400 participants
The EUROSCA Natural History Study
Run by
Ataxia Study Group
Registry ID
NCT02440763
Started
July 2005
Sites in
Austria, Belgium, France, Germany, Hungary, Italy, Netherlands, Poland and 2 more

Recent papers

Published research mentioning spinocerebellar ataxia, most recent first, from Europe PMC. Titles are shown as published and we do not summarise them — a link to the real thing is more honest than our gloss on a paper.

If you are reading in this area for the first time, look for the ones tagged “Review”. Reviews summarise a whole field rather than reporting one experiment, and they are far more useful than a single study — which, taken alone, can easily point the wrong way.

May be paywalled2026
Influenza vaccination associates with reduced risk of incident ischemic stroke among older adults with diabetes: A population-based retrospective cohort study
Yang T, Ying X, Yang H, Wu X, Shao J, Ye L, Hong J
May be paywalled2026
Long-term causes of death in 888,003 ischemic stroke patients in Thailand: a nationwide retrospective study with 18-year follow-up
Sirikarn P, Pukthaisong P, Pattanapipitpaisal P, Vorasoot N, Kasemsap N, Tiamkao S, Kongbunkiat K
May be paywalled2026
Postoperative outcomes of on- vs off-pump CABG in patients with HFrEF: a nationwide cohort study in Taiwan
Tan YH, Tung HH, Chien SC, Chien CY
May be paywalled2026
Discovery of a mutation-containing circRNA in polyglutamine disease through systematic analysis of RNAs with CAG repeats
Pawlik W, Woźna-Wysocka M, Jazurek-Ciesiołka M, Dulski J, Witkoś TM, Ciołak A, Kozłowska E, Kościańska E, Bartelt LC, Philippe J, Sławek J, Świtoński PM, La Spada AR, Fiszer A
Free to read2026
Cardiovascular-kidney-metabolic syndrome through the lens of gut‑derived uremic toxins
Xu C, Snelson M, Marques FZ
Free to read2026
Analytical and clinical validation of a competitive ELISA for serum indoxyl sulfate and its clinical association with chronic kidney disease risk stratification
Hou YC, Chuang YC, Cheng YW, Huang HY, Chen LY, Tseng TL, Lu KC
Free to read2026
Scoping review on the relationship between microRNAs (miRNAs) and short sleep disorder or insomnia with short sleep duration
Perdigoto S, Meira E Cruz M, Remesal M, Scala M, González Soltero MDR, Noriega CA, de Pedro M
Free to read2026
Effect of hemoperfusion plus hemodialysis on uremic toxins and anemia in patients with maintenance hemodialysis
Zou LX, Wang X, Qian CH, Zheng XQ, Sun L
Free to read2026
Spontaneous intracranial artery dissection: risk factors, clinical features and imaging features
Wang Y, Jiang Q, Chen X, Liang Q, Cao S, Wang F
Free to read2026
The association between serum indoxyl sulfate and radial-cephalic arteriovenous fistulas dysfunction in ESKD patients: a prospective study
Shen Q, Zheng P, Wang Q, Lin Y, Qiu M, Weng X, Yu G
Free to read2026
Comparison of the predictive performance of systemic immune-inflammation index and neutrophil-to-lymphocyte ratio for three-month poor functional outcome in ischemic stroke: a systematic review and meta-analysis
Ma X, Zhou Y, Li Z, Mao G, Wei H, Zhao T
Free to read2026
The role of salvianolic acid B and benzoylpaeoniflorin in enhancing angiogenesis through Nrf2/HO-1/VEGFA signaling axis in ischemic stroke recovery
Zhao C, Bai X, Ding Y, Zeng A, Wen A, Fu Q, Wang J
May be paywalled2026
Neurometabolic alterations in the anterior cingulate gyrus and putamen: Insights from adolescents with insomnia and depression
Liu Y, Zhong Q, Liu P, Wang Y, Zhang Y, Huang D, Chen P, Yangyu W, Lai S, Jia Y
May be paywalled2026
Elevated serum miR-19a-3p serves as a diagnostic and prognostic biomarker for ischemic stroke and aggravates neuronal injury by targeting PTEN
Wang L, Ye W, Zhang H
May be paywalled2026
Application and prospect of bioinformatics in the prevention and treatment of ischemic stroke: A comprehensive narrative review
Fang L, Zhou Y, Yang J, Ban T, Shen W, Ma L, Song Y, Zhao F
May be paywalled2026
Sex-specific associations of social determinants of health and genetic risk factors with atherosclerotic cardiovascular diseases incidence in the general population
Paulin A, Ruel LJ, Thériault S, Arsenault BJ
May be paywalled2026
Opportunities and challenges for the application of single-cell RNA sequencing and spatial transcriptomics in ischemic stroke
Wang Z, Hao Z, Xu S, Wu H, Wu J, Zhao M, Wang R, Xing L, Wang J
May be paywalled2026
Circulating acidic α-glucosidase as a potential biomarker for lactate-related immunometabolism in ischemic stroke
Liao D, Zeng T, Zhang M, Yang Q, Gao C, Gao Z, Bai J, Jin L, Wang H, Luo P, Dai S, Li X
May be paywalled2026
From molecular mechanisms to clinical implications of clock genes in ischemic stroke: A scoping review
Li S, Liu K, Lv Y, Zhang Z, Liu Y, Yu M, Yang Q, Gao Q, Zhao Y, Wu H
May be paywalled2026
PARP1 modulates BBB function via regulating NLRC5 in cerebral endothelial cells during ischemic stroke
Yang X, Shu H, Liang M, Tao C, Zhu X
May be paywalled2026
Trends of Mortality and Years of Life Lost Due to Stroke Among Older Adults in China and Its Provinces, 2005-2020
Wang Z, Long Z, Li J, Chen Y, Yuan B, Wang W, Li J, Wang X, Sheng J, Zhao J, Wang L, Zhou M, Yin P, Ma Q
May be paywalled2026
Optimizing twin prime editing components for scalable genome editing and therapy in spinocerebellar ataxia type 3
Gwon LW, Seong JB, Yeo HG, Oh Y, Park J, Won J, Park SJ, Kim YH, Huh JW, Kim A, Park KH, Lee Y, Lee SH
May be paywalled2026
Endothelial-to-mesenchymal transition in ischemic diseases: Molecular mechanisms, pathophysiological roles, and emerging therapeutic strategies
He Z, Yang Q, Ji X, Li J, Xu M
May be paywalled2026
Systematic profiling of chemical components in Tribulus terrestris L. extract and its absorbed parent compounds/metabolites in rat plasma utilizing UPLC-Q-TOF/MS
Han Y, Cong X, Cai L, Zhao G, Sun X

Data sources for this page

ClinicalTrials.gov — US National Library of Medicine
Europe PMC — EMBL-EBI

How a drug gets approved

Why promising results do not always turn into medicines.