Natural History Study for Friedreich Ataxia
(UNIFAI Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to better understand Friedreich ataxia, a rare genetic disorder affecting muscle coordination and movement. Researchers seek to track the disease's progression over time to aid in developing safe and effective future treatments. The trial is observational, focusing on gathering detailed information about the condition rather than testing new treatments. Individuals with a confirmed genetic diagnosis of Friedreich ataxia who can provide informed consent are suitable candidates for this study. As an unphased trial, it offers participants the chance to contribute valuable insights into the disease, potentially aiding future treatment development.
Why are researchers excited about this trial?
Researchers are excited about this trial because it aims to build a comprehensive understanding of Friedreich Ataxia's natural progression, which is crucial for developing effective treatments in the future. Unlike most studies that focus on specific drugs or therapies, this trial collects extensive data from patients worldwide to identify patterns and potential therapeutic targets. By consolidating global efforts, the trial has the potential to accelerate the discovery of new treatments and improve the quality of life for those affected by this condition.
Who Is on the Research Team?
David Lynch, MD
Principal Investigator
Children's Hospital of Philadelphia
Jorg B Schulz, Prof
Principal Investigator
University Hospital, Aachen
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Baseline Assessment
Initial data collection including demographics, medical history, and baseline clinical assessments
Annual Follow-up
Participants undergo annual assessments to monitor disease progression and collect clinical outcome data
Data Analysis and Reporting
Analysis of collected data to identify patterns and inform future clinical trial designs
What Are the Treatments Tested in This Trial?
Find a Clinic Near You
Who Is Running the Clinical Trial?
Friedreich's Ataxia Research Alliance
Lead Sponsor