CompletedPhase 3

A Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Friedreich's Ataxia

United States65 participantsStarted 2019-10-30

Plain-language summary

The purpose of this study is to assess the Efficacy, Long Term Safety and Tolerability of RT001 in subjects with Friedreich's Ataxia

Age range12 Years – 50 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Male or female 12 to 50 years of age
✓. Medical history consistent with the symptoms of FRDA at ≤ 25 years of age
✓. Detection of biallelic pathogenic variants in frataxin gene (FXN)
✓. Ambulatory (with or without assistive device) and capable of performing other assessments/evaluations.
✓. Must be able to walk 25 feet during the timed 1-minute walk

✕. Received treatment with other experimental therapies within the last 30 days prior to the first dose
✕. Previously participated in the RT001 trial
✕. Refusal to discontinue fish oils or other oil-based supplements for the duration of the study (Screening till last study procedure completed)
✕. History of malignancies (other than basal cell carcinomas)
✕. Inability to complete CPET protocol
✕. Female who is breastfeeding or has a positive pregnancy test
✕. History of uncontrolled diabetes mellitus (Type 1 or 2)

Change From Baseline to 11 Months in Maximum Consumption of Oxygen (mlO2/kg/Min) Using Cardiopulmonary Exercise Testing (CPET)

Timeframe: 11 months

NCT IDNCT04102501

SponsorBiojiva LLC

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2021-08-23

Results submitted2022-04-06