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.
180
Registered studies found
36
Currently open to enrolment
119
Testing an intervention
Registry data retrieved 4 November 2026 · refreshed automatically twice daily
Registered studies
36 studies
Studies currently recruiting or about to
- 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
- 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
- Not yet recruitingPhase 248 participants
GV101 for Friedreich's Ataxia (FA) Treatment
- Run by
- Graviton Bioscience Corporation
- Registry ID
- NCT07870590
- Testing
- GV101, Placebo
- Started
- November 2026
- Sites in
- United States
- Not yet recruitingPhase 160 participants
A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of ATI-105 in Healthy Adults
- Run by
- Adjuvia Therapeutics, Inc.
- Registry ID
- NCT07869654
- Testing
- ATI-105, Placebo
- Started
- October 2026
- Sites in
- United States
- RecruitingPhase 3150 participants
A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia
- Run by
- Larimar Therapeutics, Inc.
- Registry ID
- NCT07778836
- Testing
- Nomlabofusp, Placebo
- Started
- October 2026
- Sites in
- United States
- Not yet recruitingObservational25 participants
Multi-Modal Digital Monitoring of Disease Symptoms in Spinocerebellar Ataxia
- Run by
- BioSensics
- Registry ID
- NCT07851376
- Started
- December 2026
- Sites in
- United States
- RecruitingPhase 110 participants
A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
- Run by
- Solid Biosciences Inc.
- Registry ID
- NCT07180355
- Testing
- SGT-212
- Started
- October 2025
- 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
- Not yet recruitingNot applicable24 participants
Optimizing Parameters of Transcranial Temporal Interference Stimulation for Spinocerebellar Ataxia Type 3
- Run by
- First Affiliated Hospital of Fujian Medical University
- Registry ID
- NCT07743866
- Testing
- Transcranial Temporal Interference Stimulation (tTIS)
- Started
- August 2026
- Sites in
- China
- 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
- 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
- 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
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
- 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
- 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
- RecruitingObservational14 participants
Friedreich Ataxia Nerve Ultrasund
- Run by
- Centre Hospitalier Universitaire de Nice
- Registry ID
- NCT07508631
- Testing
- Nerve ultrasound - routine exam
- Started
- December 2025
- Sites in
- France
- RecruitingObservational200 participants
Patient-reported, Health Economic and Psychosocial Outcomes in Friedreich Ataxia
- Run by
- German Center for Neurodegenerative Diseases (DZNE)
- Registry ID
- NCT05943002
- Started
- June 2023
- Sites in
- Austria, France, Germany
- 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
- 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
- —
- RecruitingPhase 370 participants
A Randomized, Parallel-arm, Double Blind, Placebo-controlled Study to Assess the Efficacy of Fampridine for Patients With Spinocerebellar Ataxia SCA27B Caused by a GAA Expansion in the FGF14 Gene
- Run by
- Assistance Publique - Hôpitaux de Paris
- Registry ID
- NCT07185347
- Testing
- Fampridine 10 mg prolonged-release tablet (per os), Placebo (tablets per os)
- Started
- October 2025
- Sites in
- France
- 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
- 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
- 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
- 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 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
- RecruitingObservational4000 participants
Biomarker Research in Inherited Movement Disorders
- Run by
- Assistance Publique - Hôpitaux de Paris
- Registry ID
- NCT05034172
- Testing
- Clinical follow-up
- Started
- August 2021
- Sites in
- France
- 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
Betaine alleviates renal osteodystrophy by stabilizing ABCG1 mRNA to inhibit HMOX1-mediated ferroptosis in BMSCs
Li X, Xie Y, Ren W, Liu Q, Pang Q, Liu W, Li X, He B, He D, Wan X, Liu J, Cai J, He B, Huang W, He F
- May be paywalled2027
Lipid flip-flop in biological membranes: through the lens of TMEM16F
Shan KZ, Lowry AJ, González Torres MA, Seri MS, Yang H
- Free to read2027
Exosomes in brain diseases: Mechanisms, advances, and perspectives
Cheng Q, Zhu Y, Lv S, Chen R, Liu P, Ji X
- May be paywalled2026
A microbiota-metabolite-glial axis drives white matter repair via isorhamnetin-PSAP-GPR37 signaling after ischemic stroke
Zhang Y, Qiu L, Wu J, Shi Y, Niu S, Zhao C, Chen T, Cui C, Ma Y, Zhang X, Yao X, Meng Y, Zhao L, Xing Y, Wu X, Chen Z, Zheng K, Cai Y, Chen J, Wu J
- Free to read2026
Divergent epidemiologic, shared risks: a comparative global analysis of COPD, ischemic stroke, and intracerebral hemorrhage
Chen B, Liao Z, Lv C, Yang L, Sun F, Mei Z
- Free to read2026
The Place of Therapeutic Apheresis in Severe Immune-Related Adverse Events: A Scoping Review of Literature
Vuillet C, Moranne O, Daubin D, Quantin X, Palassin P, Kanouni T, Maria ATJ, MonRIO network (Montpellier Réseau Immuno‐Oncologie)
- Free to read2026
The impact of rising global temperatures on neurological diseases
Pereira GM, Rocha GWB, Li LM
- Free to read2026
Quantification of Neuromotor Control in <i>STXBP1</i>-Related Disorders With Wearable Sensors
Orlando JM, Bane B, Chisari T, Magielski JH, Pierce SR, Cunningham KG, Woo C, Tefft S, Nolan J, Ruggiero SM, Boland MJ, McKee JL, Helbig I, Prosser LA
- Free to read2026
Multiple strategies to improve the therapeutic efficacy of mesenchymal stem cell‑derived exosome transplantation in ischemic stroke (Review)
Xu Y, Zhou X, Zeng W, Wang J, Zhou L, Li L, Zou Z, Chen C, Zhang Y, He Z, Ye J
- Free to read2026
Stress granules at the crossroads of the proteostasis network
Kim R, Calle Urgiles EN, Moon SL
- May be paywalled2026
Nanopore sequencing combined with adaptive sampling and NanoExpansion enables accurate characterization of repeat expansion disorders
Morandi L, Casadei F, De Fanti S, Bonan L, Ravaioli F, Palombo F, Fiorentino A, de Pasqua S, Vacchiano V, Avoni P, Rizzo G, Capellari S, Ruggeri E, Donadio V, Castellani G, Bacalini MG, Lodi R, Liguori R, Carelli V, Tonon C
- Free to read2026
Multidomain risk profiles and documented polyvascular involvement across atherosclerotic vascular beds
Supriami K, Flores AM, Sui Y, Cho SMJ, Patel AP, Ruan Y, Aragam KG, Natarajan P, Ellinor PT, Fahed AC
- Free to read2026
Association of circulating miR-210 with post-stroke seizures and clinical outcome: A preliminary study
Enayati Y, Daneshvar Kakhki R, Kazemi M, Yazdani A, Motedayyen H, Noorbakhsh M
- Free to read2026
A comprehensive overview of genetic mutations in Iranian patients with Bardet-Biedl syndrome
Khalilian S, Fathi M, Entezam M, Farbood Z, Ghafouri-Fard S, Dastgheib SA, Miryounesi M
- May be paywalled2026
Predicting risk of ischemic stroke: A transformer model using genomic data
Yang Y, Guo K, Fang Z, Zhang Y, Lin H, Grosser M, Venter D, Cordato D, Zhang G, Lu J
- May be paywalled2026
Emerging genetics of sleep and circadian disorders
Mani H, Cuddapah VA
- 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
Data sources for this page
- ClinicalTrials.gov — US National Library of Medicine
- Europe PMC — EMBL-EBI
Next
How a drug gets approved
Why promising results do not always turn into medicines.