A Study to Assess Variation in Potential Biomarkers in Friedreich Ataxia

Completed

Conditions studied: Friedreich Ataxia

In brief

To test the variability of specific ribonucleic acid (RNA) and proteins as well as frataxin levels in samples of blood and buccal cells taken directly from patients with Friedreich's ataxia (FRDA) in order to confirm potential new biomarkers of disease in patients with FRDA.

Key facts

Study ID

NCT04255680

Run by

Larimar Therapeutics, Inc.

People needed

20

Starts

2020-01-14

Expected to finish

2020-06-30

Last updated by the study team

2020-08-18

Who can join

Age: 12 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Male and female patients with FRDA confirmed by genetic testing (FRDA subjects only).
• Children and adults between the ages of 12 and 65 (inclusive); age for controls will be +/- 2 years relative to FRDA subjects.
• Subject (and/or parent/legal guardian) has voluntarily signed consent form.
• Willingness and ability to comply with all study procedures.
• Functional Disability Stage (FDS) of 3, 4, or 5 (FRDA subjects only).

You may not qualify if…

• Treatment with an investigational product within 30 days of study.
• Use of gamma interferon or receiving any dose of gamma interferon within 90 days of the specimen collection day.
• Use of any statin medications within 90 days of the specimen collection day.
• Use of any lipid-lowering agents within 6 weeks of the specimen collection day.
• Use of daily biotin supplementation that exceeds 30 mcg/day, either as part of a multivitamin or as a standalone supplement, within 7 days of the study visit.
• Pregnant women.

Where it is running

• The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States

Full record on ClinicalTrials.gov

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