Active — Not RecruitingNot Applicable

FA Clinical Outcome Measures

United States2,000 participantsStarted 2001-01-01

Plain-language 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.

Who can participate

Age range4 Years – 80 Years

SexALL

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Inclusion criteria

✓. Males or females age 4 to 80 years.
✓. Genetically confirmed diagnosis of FA (for carrier/control cheek swab and blood samples this is not required).
✓. 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).
✓. Parental/guardian permission (informed consent) and if appropriate, child assent.

What they're measuring

Friedreich Ataxia Rating Scale

Timeframe: once every 1 year

Trial details

NCT IDNCT03090789

SponsorFriedreich's Ataxia Research Alliance

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2030-01-01

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