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Participating in a research study or clinical trial is one way to take an active role in furthering understanding and treatment of Ataxia. It is also a way to get access to new treatment options before they are widely available. However, participating is a deeply personal choice to make. Look into the risks and benefits of each study before enrolling. You may also consider where research is being conducted, some studies are in-person and others are done remotely. To learn more about the clinical trial process, visit PrepRARE Clinical Trial Readiness.
NAF is committed to providing information to those affected by Ataxia including the availability of research studies and clinical trials for the study of Ataxia.
Below you will find clinical trials and research opportunities for those with various types of Ataxia. These trials have been approved and monitored by an Institutional Review Board (IRB), Research Ethics Board (REB), or Research Ethics Committee (REC) to make sure the risks are as low as possible and are worth any potential benefits. Click the name of the study to see detailed information.
If you would like to have your study listed here or request help with recruitment, download the Research Recruitment Application or email research@ataxia.org with your question.
Interventional clinical trials involve testing a drug, medical device, activity, or procedure. The study aims to figure out if a new treatment improves symptoms or slows disease progression.
STUDY NAME
CLINICAL TRIAL PHASE
CONDITION
LOCATION
AGE
Phase 1
Friedreich’s Ataxia
New York, NY, USA
Adult
18+
Phase 1
Friedreich Ataxia
Philadelphia, PA, USA
Children
Observational clinical studies collect information about disease progression over time. There is no drug or treatment involved in these types of studies. Observational studies are important for understanding symptoms and the patient experience. These studies support future drug development.
Did you know that NAF helps coordinate one of the longest-running Ataxia observational studies of all time? The Clinical Research Consortium for the Study of Cerebellar Ataxia (CRC-SCA) gives researchers crucial information to help design clinical trials and facilitate drug development. People with SCA1, 2, 3, 6, 7, 8, and 10 are eligible to participate.
STUDY NAME
CONDITION
LOCATION
AGE
Learn how to find clinical trial information using ClinicalTrials.gov.
Research studies are used to explore the causes and effects of ataxia. They are used to better understand how ataxia impacts part of the body, what causes ataxia, and how we can measure ataxia symptoms. This information is important for planning future interventional and observational clinical studies.
STUDY NAME
CONDITION
LOCATION
AGE
All Ataxias
Remote
Adult 18+
All Ataxias
Remote
Adult 18+
All Ataxias
Boston, MA, USA
Adult 18+
All Ataxias for which genetic testing was considered or performed
Remote
Children and Adult 18+
Cerebellar Ataxia of Unknown Etiology (CAUE)
Baltimore, MD, USA
New York, NY, USA
Los Angeles, CA, USA
Evenston, IL, USA
Adult 18+
SCA1, SCA2, SCA3, SCA6, SCA7, SCA8, SCA17, or SCA48
Remote
Adult 18+
Spinocerebellar Ataxia and Cerebellar Degeneration (Any Subtype)
Remote
Children and Adult 18+
Spinocerebellar Ataxia and Multiple System Atrophy
Remote
Adult 18+
SCA (all subtypes) and Friedreich’s Ataxia
Boulder, CO, USA
Adult 18+
Spinocerebellar Ataxias (confirmed with genetic testing)
Baltimore, MD, USA
Adult 18+
Spinocerebellar Ataxias (Any Type)
Remote
Adult 18+
For Clinicians Only – Caring for patients with SCAs 1,2,3,6,7,8,10,12,27B, FRDA, RFC1/CANVAS, FXTAS, AT, EA1/2
Remote
Adult 18+
Patient Registries are essential tools for Ataxia researchers. The purpose of a patient registry is to advance the development of treatments for a disease by connecting researchers with those who are affected with that disease. If you or a family member are affected by Ataxia or are at risk for Ataxia, we recommend that you sign up for one or more patient registries. Learn more about what patient registries are and why do they matter in this article.
REGISTRY NAME
CONDITION
LANGUAGE SUPPORTED
Autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS)
English, French, Spanish, and Italian
English, Polish, Hebrew, French, German, and Arabic
English
Dentatorubral-pallidoluysian atrophy (DRPLA)
English, French, Italian, Japanese, Korean and Portuguese
Friedreich Ataxia
English, Dutch, French, German, Greek, Italian, Portuguese, and Spanish
Fragile X-associated tremor/ataxia syndrome (FXTAS), Fragile X-associated primary ovarian insufficiency (FXPOI), Fragile X premutation carriers, Relatives of Fragile X premutation carriers
English and Spanish
The CoRDS Registry was established to facilitate the establishment of rare disease registries for all rare diseases with a specific questionnaire for those with any type of Ataxia or those at risk for Ataxia.
About CoRDS
How to Enroll in the CoRDS Registry
Donating tissue for medical research is an important and deeply personal decision. Proper planning can help ensure that wishes are accounted for and honored at the time of your or a loved one’s passing. Start your planning with the documents listed below. For additional information on tissue donation contact the National Ataxia Foundation at naf@ataxia.org or call 763-553-0020. If death is imminent or has occurred, rapid recovery is essential. Call 1-877-GoAtaxia (1-877-462-8294) as soon as death appears imminent or has occurred. Thank you for considering this important legacy gift.
Our generous donors help us fund promising Ataxia research and offer support services to people with Ataxia. Your gift today will help us continue to deliver on our mission to improve the lives of persons affected by Ataxia.
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