FA Clinical Outcome Measures

NCT03090789 · tracked via the Priya Life Science India tracker

Sponsor

Friedreich's Ataxia Research Alliance

Phase

Observational

Started

2001-01-01

Last updated

2024-10-04

Condition(s) studied

Friedreich AtaxiaNeuro-Degenerative Disease

Study summary

This multicenter natural history study aims to expand the network of clinical research centers in FA, and to provide a framework for facilitating therapeutic interventions. In addition, this study will lead to the development of valid yet sensitive clinical measures crucial to outcome assessment of patients with Friedreich's Ataxia. This study will support genetic modifier studies, biomarker studies, and frataxin protein level assessments by building a sample repository.

This natural history study is no longer recruiting under this protocol NCT03090789 but remains actively recruiting under the harmonized study (UNIFAI) NCT06016946.

Eligibility

Sex

ALL

Min age

4 Years

Max age

80 Years

Healthy volunteers

Accepted

Inclusion Criteria: 1. Males or females age 4 to 80 years. 2. Genetically confirmed diagnosis of FA (for carrier/control cheek swab and blood samples this is not required). 3. Clinically confirmed diagnosis of FA, pending confirmatory genetic testing through a commercial or research laboratory (for carrier/control cheek swab and blood samples this is not required). 4. Parental/guardian permission (informed consent) and if appropriate, child assent. Exclusion Criteria: 1\) Signs or symptoms of severe cardiomyopathy (such as congestive heart failure)

Primary outcome measure(s)

Friedreich Ataxia Rating Scale — once every 1 year
rating scale based on clinical neurologic examination

Trial sites (14)

Facility	City	Region
UCLA Ataxia Center	Los Angeles	California
University of Colorado	Denver	Colorado
University of Florida - Neurology	Gainesville	Florida
USF Ataxia Research Center	Tampa	Florida
Emory University Hospital - Neurology	Atlanta	Georgia
University of Iowa, Stead Family Children's Hospital	Iowa City	Iowa
Ohio State University - Neurology	Columbus	Ohio
Children's Hospital of Philadelphia - Neurology	Philadelphia	Pennsylvania
St. Jude Children's Research Hospital	Memphis	Tennessee
Murdoch Childrens Research Institute	Parkville	Victoria
The Hospital for Sick Children	Toronto	Ontario
CHUM - Hopital Notre-Dame	Montreal	Quebec
All India Institute of Medical Sciences (Aiims)	New Delhi	India
Auckland City Hospital	Auckland	New Zealand

More Friedreich's Ataxia Research Alliance trials in India

Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study Recruiting

Other trials for the same condition

A Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia Recruiting

Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study Recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT03090789 on ClinicalTrials.gov ↗ ← All trials in India