Children's Hospital of Philadelphia, University of Florida and University of California Los Angeles are seeking children with FA for a 3 yr study to help advance our understanding of disease progression and validate outcome measures that can be used in future clinical trials.
This study is funded by the Food and Drug Administration (FDA) and the Friedreich’s Ataxia Research Alliance (FARA).
It is a natural history study with the following objectives:
Collect data twice a year to understand progression of FA and be able to quantify change in progression over time. Develop and validate outcome measures (timed walk tests, vision tests) that can be used in future clinical trials Make design and implementation of clinical trials more efficient and effective Collect biological samples such as cheek swabs and blood to identify and study biomarkers involved with the disease process and quantify the change in these markers over time Analyze disease progression in FA affected tissues using innovative and noninvasive measures
Key Inclusion Criteria:
You/your child have a confirmed diagnosis of Friedreich’s ataxia
You/your child are between the ages of 2 and 18 years of age
To learn more about the Inclusion and Exclusion Criteria and the process for participation in the study, please contact the Clinical Research Coordinator with any questions.
Children's Hospital of Philadelphia, PA - View the CHOP Study Flyer
Contact: Cassandra Strawser, 215-590-2314 -
University of California Los Angeles, CA
Contact: Aaron Fisher, 310-206-8153 -
or Arjun Sarkar -
University of Florida, Gainesville, FL
Contact: Samantha Norman, 352-273-8218 -
For more information visit Clinicaltrials.gov - https://www.clinicaltrials.gov/ct2/show/NCT03418740
Anyone considering participating in a clinical trial should discuss the matter with his or her physician. FARA does not endorse or recommend any particular studies.
Neurological Measures of Progression in Children with FA