The UNIFAI Study
Research type
Research Study
Full title
Friedreich's Ataxia Global Clinical Consortium UNIFIED Natural History Study
IRAS ID
355948
Contact name
Paola Giunti
Contact email
Sponsor organisation
UCL- University College London
Clinicaltrials.gov Identifier
FG0015293, Data Protection Number for FARA Europe; Z6364106/2025/06/249, UCL's Data Protection Policy Number
Duration of Study in the UK
24 years, 11 months, 30 days
Research summary
This project is a global, multicenter, prospective, observational natural history study that can be used to understand the disease progression and support the development of safe and effective drugs and biological products for Friedreich’s Ataxia.
Primary Objective:
Unify the global FA community (clinicians, researchers, and patients) contributing to the understanding of disease natural history; minimize duplicate efforts, create a sustainable data platform to support innovative clinical studies, and facilitate interventional therapy development.Secondary Objectives:
• Track health problems and progression of symptoms that occur throughout the disease course.
• Evaluate change in natural history as new disease specific therapies are approved.
• Optimize and improve the panel of outcome measures that can be used in FA clinical trials.
• Support genetic modifier studies and biomarker studies. Build in parallel a DNA and sample repository for use in large scale translational research.
• Create a global mechanism for sharing data and resources with basic and clinical investigators.
• To generate a registry of individuals with Friedreich Ataxia to aid recruitment to future trials and studies.REC name
London - Bromley Research Ethics Committee
REC reference
25/PR/0847
Date of REC Opinion
21 Oct 2025
REC opinion
Further Information Favourable Opinion