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

Study Overview
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Brief Summary
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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
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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.

Official Title
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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

Conditions
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Friedreich's Ataxia

Intervention / Treatment
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* Drug: RT001
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Key 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 Key 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)

Ages Eligible for Study
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Minimum Age: 12 Years
Maximum Age: 50 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: RT001<br>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 | Drug: RT001<br>* RT001 is encapsulated 9-cis, 12-cis-11,11-D2-linoleic acid ethyl ester, which is a site specific (C11) di-deutero synthetic homologue of LA ethyl ester. Each capsule contains 960 mg of RT001.<br>|
| Placebo Comparator: Placebo<br>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 | Drug: Placebo<br>* The placebo product is composed of encapsulated USP safflower oil. The placebo capsules are identical in appearance and size to RT001.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change From Baseline to 11 Months in Maximum Consumption of Oxygen (mlO2/kg/Min) Using Cardiopulmonary Exercise Testing (CPET) | To measure the change in MVO2 from baseline to Month 11 using CPET | 11 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change From Baseline in the Timed 1 Minute Walk Distance | Change from baseline Distance walked in 1 minute at 11 months | 11 months |