CompletedNot Applicable

Neurology Measures in FA Children

United States108 participantsStarted 2017-11-20

Plain-language summary

The purpose of this study is to identify ways to follow progression of Friedreich's Ataxia (FA) and be able to measure changes over time in children with FA. Participants will have biannual visits to observe how the disease progresses over time and determine the rate of progression. Funding Source- Food and Drug Administration Office of Orphan Products Development (FDA OOPD).

Who can participate

Age range2 Years – 18 Years

SexALL

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Inclusion criteria

✓. Males or females age 2 to 18 years.
✓. Genetically confirmed diagnosis of Friedreich's Ataxia (FA) or clinically confirmed diagnosis of FA, pending confirmatory genetic testing through a commercial or research laboratory
✓. Parental/guardian permission (informed consent) and if appropriate, child assent.

What they're measuring

Change in mFARS (Modified Friedreich's Ataxia Rating Scale) Score

Timeframe: Baseline up to 36 Months

Trial details

NCT IDNCT03418740

SponsorChildren's Hospital of Philadelphia

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2023-02-02

Results submitted2024-02-02

View on ClinicalTrials.gov More Friedreich Ataxia trials