NAF Ataxia Research Studies Currently Underway

George R Wilmot, MD, PhD

Emory University
Cooperative Ataxia Registry

Summary: This registry is designed for patients with ataxia. Ataxia is a group of rare disorders that affect movement and coordination. Because these disorders are rare, it will require a collaborative effort between scientist across North America and patients across North America to complete sound research. This registry will serve in matching patients interested in participating in research projects with researchers looking for eligible subjects to enroll in their studies. After signing an informed consent form and an authorization to obtain medical records form, potential subjects will complete a patient information form and mail them or fax them to the registry office. After review of the data, eligibility will be determined by the principal investigator. The data will be entered by hand and stored in a secure database on a computer.

The registry will support two different types of subsequent studies. First, patient data that is stripped of all identifying information can be accessed directly by researchers who have submitted qualifying study protocols to the registry. Second, ataxia researchers can submit research protocols requiring identifying information, and if a protocol is approved by the registry principal investigator, potentially eligible patients will be mailed information on the study. The information sheet will provide a brief description of the study and a phone number to contact the study coordinator. It will be up to the patient to determine if they are interested in calling to get more information about the study. At no time will patient contact information be provided to the researcher by the registry.

One purpose of the study is to collect data and personal contact information, on an ongoing basis, about patients diagnosed with ataxia. This identifiable data will enable us to contact patients to provide information on research studies available. Another purpose will be to use this data to study the natural history of these disorders and will allow identification of the features which may be most sensitive to change, and/or interventions that may be tested in collaborative studies in the future. Patients enrolled in the registry will have the opportunity to enroll in future treatment protocols, if they are eligible and so desire, but will sign a different, specific, consent form for those studies at that time. After the information listed above is obtained, it will be submitted to the cooperative registry. Patients can refuse any part of the data collection they do not wish to participate in.

This research may help us to learn more about this group of rare diseases and may help to form a basis for future treatment protocols that have not been available to date.