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.
176
Registered studies found
38
Currently open to enrolment
117
Testing an intervention
Registry data retrieved 18 September 2026 · refreshed automatically twice daily
Registered studies
38 studies
Studies currently recruiting or about to
- RecruitingPhase 360 participants
Pivotal Study of N-acetyl-L-leucine for CACNA1A
- Run by
- IntraBio Inc
- Registry ID
- NCT07221292
- Testing
- N-Acetyl-L-Leucine, Placebo
- Started
- August 2026
- Sites in
- Austria, Germany, Greece, Italy, Switzerland, United Kingdom, 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
- 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
- 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
- RecruitingObservational4000 participants
TreatHSP Platform: Adaptive Natural History Platform for Ataxias, HSPs, and Spastic Ataxias
- Run by
- Heidelberg University
- Registry ID
- NCT07798674
- Started
- July 2024
- Sites in
- Austria, Czechia, France, Germany, Ireland, Italy, Netherlands, Poland and 1 more
- RecruitingObservational3000 participants
Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study
- Run by
- Friedreich's Ataxia Research Alliance
- Registry ID
- NCT06016946
- Started
- June 2023
- Sites in
- Australia, Austria, Belgium, Brazil, Canada, Czechia, France, Germany and 9 more
- 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
- 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
- Sites in
- Belgium, Brazil, Canada, France, Spain, United States
- 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
- Not yet recruitingObservational40 participants
Digital Outcome Assessment Using AI Active Gaming and Motion Capture in Friedreich Ataxia
- Run by
- University of Exeter
- Registry ID
- NCT07731971
- Testing
- Laboratory validation session (VSimulator), Home-based active gaming assessment
- Started
- July 2026
- Sites in
- United Kingdom
- 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
- RecruitingPhase 3255 participants
A Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia
- Run by
- Biogen
- Registry ID
- NCT06953583
- Testing
- Omaveloxolone, Placebo
- Started
- June 2025
- Sites in
- Australia, Austria, Brazil, Canada, Denmark, France, Germany, India and 8 more
- 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
- RecruitingPhase 1/Phase 210 participants
Safety and Efficacy of Mutation-targeted Precision Genetic Therapy for Ataxia-Telangiectasia (A-T)
- Run by
- Timothy Yu
- Registry ID
- NCT07215416
- Testing
- Antisense oligonucleotide targeting the ATM gene
- Started
- August 2026
- 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
- RecruitingObservational300 participants
Spinocerebellar Ataxia Type 27B Natural History Study (SCA27B-NHS)
- Run by
- University Hospital Tuebingen
- Registry ID
- NCT06472557
- Started
- June 2024
- Sites in
- Czechia, Germany, Italy, Spain
- 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
- —
- RecruitingNot applicable60 participants
Ophthalmological Disorders in Dominant Spinal-cerebellar Ataxias
- Run by
- University Hospital, Montpellier
- Registry ID
- NCT07019558
- Testing
- Neurological assessment, Ophthalmological assessment
- Started
- October 2025
- Sites in
- France
- 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
- 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
- RecruitingNot applicable24 participants
Efficacy of Stabilometric Platform to Improve Standing Balance in Patients With Friedreich's Ataxia
- Run by
- IRCCS Eugenio Medea
- Registry ID
- NCT06692296
- Testing
- Balance training over stabilometric platform, Balance training by means of conventional rehabilitation
- Started
- February 2025
- Sites in
- Italy
- 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.
- Free to read2027
Exosomes in brain diseases: Mechanisms, advances, and perspectives
Cheng Q, Zhu Y, Lv S, Chen R, Liu P, Ji X
- Free to read2026
PSMA1, PSMA5, and PSMB2 serve as prognostic biomarkers and are correlated with tumor-infiltrating leukocytes in HCC
Islam B, Xu W, Rehman ZU, Zeng R, Khushafa T, Yu H, Huang J
- Free to read2026
NF-κB across the Alzheimer disease spectrum: Context-dependent protective and pathogenic roles
Azargoonjahromi A, Abutalebian F, Nasiri H
- Free to read2026
LncRNA HOTAIR/miR-9-5p/FOXP1 axis modulates cerebral ischemia-reperfusion injury via NLRP3 inflammasome activation
Liu T, Zhu X, Zhuo F, Peng S
- Free to read2026
Electroacupuncture at Neiguan (PC6) and Shuigou (GV26) regulates microglial polarization and neuroinflammation through the cGAS-STING signaling pathway in ischemic stroke
Li X, Duan Y, Ma Y, Ma Y, Ma X, Liang X, Xin D
- Free to read2026
Mitochondrial targeting strategies for mitigating oxidative damage and enhancing neuroprotection in ischemic stroke: A Review
Trujillo-Rangel WÁ, Escutia-Gutiérrez R, García-Benavides L, Zamudio-Ojeda A, Delgado-Lara DLC, García-Mercado FG, Padilla-Ponce DJ, Magallón-Chávez O, Rivas-Loaiza JA, Pérez-Cisneros M, Guevara-Martínez SJ
- Free to read2026
Indoxyl sulfate exacerbates chronic inflammation and susceptibility to severe infection via modulating T-cell CD73/adenosine signaling in chronic kidney disease
Cao J, Nie Y, Cao X, Chen X, Bo J, Wang Y, Zou J, Ding X, Shen B, Xiang F
- Free to read2026
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
- Free to read2026
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
- Free to read2026
Two dipeptide repeat proteins are produced from mammalian telomeric RNA previously thought to be a long non-coding RNA
Al-Turki TM, Griffith JD
- Free to read2026
Impact of dual antiplatelet therapy duration after percutaneous coronary intervention on outcomes in acute coronary syndrome patients with and without chronic kidney disease: a nationwide cohort study
Chang KW, Liao PL, Huang JY
- Free to read2026
Investigating interdisciplinary learning outcomes in a forensic science programme
Lim XX, Low YN, Gan JS, Tan WS
- 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
- Free to read2026
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
Direct quantification of S100β and BNP in whole blood via a GHNP-enhanced SERS plasmonic sensor for ultra-early ischemic stroke diagnosis and clinical stratification
Wang M, Wu J, Wang C, Liu D, Wang Y, Zhao P
- May be paywalled2026
Ruxolitinib preserves endothelial function in middle-aged and old mice after ischemia/reperfusion injury
Saemann L, Soyer HS, Pohl S, Hagewiesche S, Sarow L, Wambrauw S, Kuru-Schors M, Simm A
Data sources for this page
- ClinicalTrials.gov — US National Library of Medicine
- Europe PMC — EMBL-EBI
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