A Randomized, Double-Blind, Controlled, Phase 2/3 Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Friedreich's Ataxia
Overview
- Phase
- Phase 3
- Intervention
- RT001
- Conditions
- Friedreich's Ataxia
- Sponsor
- Retrotope, Inc.
- Enrollment
- 65
- Locations
- 6
- Primary Endpoint
- Change From Baseline to 11 Months in Maximum Consumption of Oxygen (mlO2/kg/Min) Using Cardiopulmonary Exercise Testing (CPET)
- Status
- Completed
- Last Updated
- 3 years ago
Overview
Brief Summary
The purpose of this study is to assess the Efficacy, Long Term Safety and Tolerability of RT001 in subjects with Friedreich's Ataxia
Detailed Description
This is a randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, tolerability, in subjects with FRDA following the oral administration of study drug (active or placebo capsules). Sixty eligible patients will undergo various assessments at different time points during the the study. The study duration is 13 months which includes screening, treatment and safety follow up phone call.
Investigators
Eligibility Criteria
Inclusion Criteria
- •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
Exclusion Criteria
- •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)
Arms & Interventions
RT001
* 9 capsules daily (8.64 g total dose) given as 3 capsules three times a day (TID) with meals for the first month of treatment. * Six capsules daily (5.76 g total dose) given as 3 capsules (BID) with breakfast, and 3 capsules with dinner after the first month of treatment
Intervention: RT001
Placebo
* 9 capsules daily (8.64 g total dose) given as 3 capsules three times a day (TID) with meals for the first month of treatment. * Six capsules daily (5.76 g total dose) given as 3 capsules (BID) with breakfast, and 3 capsules with dinner after the first month of treatment
Intervention: Placebo
Outcomes
Primary Outcomes
Change From Baseline to 11 Months in Maximum Consumption of Oxygen (mlO2/kg/Min) Using Cardiopulmonary Exercise Testing (CPET)
Time Frame: 11 months
To measure the change in MVO2 from baseline to Month 11 using CPET
Secondary Outcomes
- Change From Baseline in the Timed 1 Minute Walk Distance(11 months)