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

• Status: Active, not recruiting
• Trial end date: 2021-12-01
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Retrotope, Inc.

Criteria

Key Inclusion Criteria:

1. Male or female 12 to 50 years of age

2. Medical history consistent with the symptoms of FRDA at ≤ 25 years of age

3. Detection of biallelic pathogenic variants in frataxin gene (FXN)

4. Ambulatory (with or without assistive device) and capable of performing other assessments/evaluations.

5. Must be able to walk 25 feet during the timed 1-minute walk

Key Exclusion Criteria:

1. Received treatment with other experimental therapies within the last 30 days prior to the first dose

2. Previously participated in the RT001 trial

3. Refusal to discontinue fish oils or other oil-based supplements for the duration of the study (Screening till last study procedure completed)

4. History of malignancies (other than basal cell carcinomas)

5. Inability to complete CPET protocol

6. Female who is breastfeeding or has a positive pregnancy test

7. History of uncontrolled diabetes mellitus (Type 1 or 2)