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National Ataxia Foundation


Ataxia Clinical Trials

Partnering with researchers to accelerate treatment development for Ataxia.

There is an urgent need for the Ataxia community to be “clinical trial ready” because many therapies are in the drug development pipeline. To successfully bring a treatment to the market, patient involvement is crucial. The Ataxia community is needed to participate in research studies and clinical trials, but that is not their only role in drug development. Drug developers need to understand how Ataxia symptoms impact a person’s daily life in order to find meaningful solutions. NAF developed PrepRARE as a program to educate members about all stages of the clinical trial process. Check out our series of webinars, articles, and other information to make sure that you are ready to make informed decisions about participation when a new clinical trial begins recruitment.

CURRENT CLINICAL TRIALS FOR ATAXIA is a public listing of clinical trials. The trials listed below are actively recruiting participants. To learn more about the study, visit their clinical trial listing. 


Interventional studies 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.

Interventional Drug or Study Name

Eligible Ataxia Type(s)

Link to Learn More

A Pharmacokinetics and Safety Study of BIIB132 in Adults with Spinocerebellar Ataxia 3


STRIDES – a Clinical Research Study of an Investigational New Drug to Treat Spinocerebellar Ataxia (STRIDES)



Observational 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.

Observational Study Name

Eligible Ataxia Type(s)

Link to Learn More

Natural History of Spinocerebellar Ataxia Type 7 (SCA7)


Identification of Biomarkers in Spinocerebellar Ataxia 3 (SCA3)


If you would like to have your study listed here or request help with recruitment, download the Research Recruitment Application or email with your question.


PrepRARE Webinars

How to Build Your Clinical Trial Resume

MERA Study for BIIB132 – A first in human clinical trial for SCA3

STRIDES Study for SCA3 – A Clinical Research Study of an Investigational New Drug

PrepRARE Articles

Clinical Trial Phases

Let’s chat about Clinical Trial Readiness with a focus on Ataxia. Clinical trials ensure that treatments are safe and effective before they gain approval for widespread use in humans. Clinical Read More…

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A clinical trial is a process to test a potential new treatment’s safety and effectiveness. The treatment, often referred to as an “intervention,” may be a medicine, device, procedure, or changes to the participants behavior – such as diet. The clinical trial is designed to measure the outcome for participants when the intervention is introduced. In the United States, the Food and Drug Administration (FDA) is the regulatory agency that oversees all clinical trials. The FDA is also responsible for reviewing applications for new interventions to treat a disease or condition. The FDA gives approval if the data from the clinical trial proves that benefits of the intervention outweigh the known risks. 

Before an intervention is tested in people, it must be successful in animal tests and proven that it will likely be safe for humans. After a clinical trial is ready for human participants, how does the process work? Keep reading to learn! 

Investigational New Drug

When a researcher or pharmaceutical company want to test their intervention on humans, they must submit an Investigation New Drug (IND) application to the FDA. After they receive approval, they can begin recruiting volunteers to participate in their clinical trial.

Recruitment for a Clinical Trial

After IND approval, the clinical trial can start enrolling patients to try their intervention. Clinical trials are listed at The listing will include detailed information about where the study will take place, for how long, and eligibility criteria for participants. 

Researchers and pharmaceutical companies will often contact a patient advocacy organization for the disease or condition to help with their recruiting efforts. Staying connected with NAF is a great way to learn about new clinical trials for Ataxia. Become a member to receive notices when new clinical trials that might interest you are recruiting participants.

Learn More:
How Do I Know if I Qualify for a Research Study

Clinical Trial Phases

The next thing to know is that clinical trials are classified by their phase, which include Phase I, Phase II, and Phase III. Phase I is typically a small number of participants to determine safety of the intervention. Phase II  begins after a successful Phase I and evaluates effectiveness of the intervention. If the treatment benefits enough patients in Phase II, then the trial moves to Phase III. Phase III tests the treatment in a larger population and compares it against current treatments available.

Learn More: 
Clinical Trial Phases

Be Ready to Participate in New Clinical Trials

Even if there aren’t clinical trials available right now, there are steps you can take to be ready when a new trial starts recruiting!

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