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 on the appropriate patient registry listed below (you can be included on more than one patient registry.)

Ataxia Patient Registry at CoRDS 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.

CoRDS Registry at Sanford Research Video

Friedreich’s Ataxia Research Alliance Patient Registry for those with Friedreich’s ataxia.

The Fragile X Research Registry is for families who have individuals with Fragile X-associated tremor/ataxia syndrome (FXTAS) in the United States.

Patient Databases

Autosomal recessive spastic ataxia of Charlevoix-Saguenay ARSACS The purpose of this website is to create a new database for the SACSIN gene mostly reported in the province of Quebec, Canada.

Ataxia-Telangiectasia (A-T) Children’s Project Family Data Base is to help A-T families stay in touch with the A-T Children’s Project for information about clinical studies.

Participating in a 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 in a clinical trial is a deeply personal choice to make. Look into risks and benefits of each trial before enrolling. The following links are great resources for finding research studies that are looking for volunteer participants.

• ClinicalTrials.gov: Provides updated information about federally and privately supported clinical research
• Clinical Research Consortium for the SCA’s

The following links will provide information about participating in a clinical trial.

• NIH Clinical Research Trials and You
• The Center for Information and Study on Clinical Research Participation (CISCRP)

The National Ataxia Foundation 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) to make sure the risks are as low as possible and are worth any potential benefits.

• Individuals with SCA6 and SCA8 are Needed to Participate in Research Studying Ataxic Movements at the Kennedy Krieger Institute
• Ataxia research project at the University of California, Berkeley
• Natural History of Spinocerebellar Ataxia Type 7
  
   
• Balance Research Study for SCA1, 2, 3, or 6

• A Phase III, Long-Term, Randomized, Double-blind, Placebo-controlled Trial of BHV-4157 in Adult Subjects With Spinocerebellar Ataxia

• Clinical Trial Readiness for SCA1 and SCA3 (READISCA)

• GeneDx is recruiting those with specific forms of Ataxia to participate in a study. Eligible participants will be financially compensated.
• Study for Individuals AT RISK for SCA1 or SCA3.

One of the challenges in developing a treatment for a rare disease, such as Ataxia, is the lack of natural history studies. A natural history study collects data that shows how a specific disease progresses in individuals over time. Natural history studies are essential for developing clinical trial designs that will facilitate drug development.With the recent funding by the National Ataxia Foundation of the Clinical Research Consortium for the Study of Cerebellar Ataxia (CRC-SCA), a natural history study will begin for SCAs 1, 2, 3, 6 and 7 (and SCA 8 and 10 at some sites). The institutions and principle investigators that are in the CRC-SCA are listed below:

• Columbia University – Dr. Sheng Han Kuo
• Emory University – Dr. George (Chip) Wilmot
• Houston Methodist Research Institute – Dr. Tetsuo Ashizawa
• Johns Hopkins University – Drs. Liana Rosenthal and Chiadikaobi Onyike
• Massachusetts General – Dr. Jeremy Schmahmann
• Northwestern University – Dr. Puneet Opal
  Contact: Karen Williams 312-503-2068
  k-williams8@northwestern.edu
• University of California – Dr. Susan Perlman
• University of Chicago – Dr. Christopher Gomez
• University of Florida – Dr. SH Subramony
• University of Michigan – Drs. Henry Paulson and Vikram Shakkottai
• University of Minnesota – Dr. Khalaf Bushara
• University of Utah – Dr. Stefan Pulst

The success of the natural history study is dependent on participation by individuals who have those forms of SCA. If you have SCA 1, 2, 3, 6 or 7 (and SCA 8 and 10 at some sites) and are a patient at one of those sites or can travel to a site, please contact the clinic office for more information. You may also contact Sue Hagen at NAF at 763-231-2742 or susan@ataxia.org for more information.

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. For additional information on tissue donation, contact the National Ataxia Foundation at naf@ataxia.org or call 763-553-0020. Thank you for considering this important legacy gift.

Brain Donation Donor Information Form (pdf)