FARA - Clinical Trials

The Friedreich’s Ataxia Health Index Study (FA-HI)

Title of Study: The Friedreich's Ataxia Health Index Study

Principal Investigator: Chad Heatwole, MD, MS-CI

Institutional Affiliation: University of Rochester, Department of Neurology

Study Description: This study aims to identify the symptoms that have the greatest impact on quality-of-life for individuals with FA. The results of the study will help guide future research involving FA patients through the development of a patient-reported measure of health.

The study involves completing a survey to include questions on demographics and symptoms of FA.

WHO CAN PARTICIPATE?

U.S. residents
Participants with a confirmed diagnosis of FA
Adults, ages 18 or older
Children, ages 8-17, with parental consent; if the child is age 13-17, only the child should complete the survey
Parents of children age 0-12; both parent and child can complete the survey for children ages 8-12.

Please see this Recruitment flyer to access the survey links.

Part 2 of the Phase II MOXIe study (RTA 408 or omaveloxolone)

Description: FARA is excited to share news that Part 2 of the Phase II MOXIe study (RTA 408 or omaveloxolone), sponsored by Reata, is open, and needs 100 individuals with FA to participate. This is a randomized, placebo-controlled, double-blind, parallel-group study to evaluate the safety and efficacy of omaveloxolone (RTA 408) 150 mg in patients with Friedreich's ataxia. Participants will be randomized 1:1 to either receive omaveloxolone (RTA 408) 150 mg or placebo.

Key Criteria: Please see the study flyer below to learn about Inclusion and Exclusion Criteria.

Location: This is an international study with sites in the United States, Europe, and Australia.

Latest Friedreich's Ataxia - MOXIe Study Update:

Webinar with Dr. Colin Meyer of Reata and FARA reviewing MOXIe Part 1 study results and plan for Part 2 of the study:

Please review the study flyer below, if you are interested in the study or have questions about the study, we encourage you identify the site that is geographically closest to you and contact the study coordinator.

Magnetic Resonance Imaging (MRI) Muscle Phenotyping in Mitochondrial Disease

Description: The purpose of the study is to use a new research imaging technique, a kind of magnetic resonance imaging (MRI), to measure important metabolic features of muscle, including mitochondrial function, in people with mitochondrial disease and in healthy individuals.

Key Criteria: Ages 18-65 years

Location: University of Pennsylvania

ClinicalTrials.gov - https://clinicaltrials.gov/ct2/show/NCT02154711

It is hoped that this new strategy will help physicians to understand better the health problems of people with mitochondrial disease. Eventually, this could lead to better diagnostic and treatment approaches.

Clinical Trials

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