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

Phase 3

Completed

• Conditions: Friedreich's Ataxia
• Interventions: Drug: RT001; Drug: Placebo

• Registration Number: NCT04102501
• Lead Sponsor: Retrotope, Inc.

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.

Study Timeline

• Study First Submitted: 2019-09-23
• Study First Submitted QC: 2019-09-23
• Study First Posted: 2019-09-25
• Study Start: 2019-10-30
• Primary Completion: 2021-08-23
• Study Completion: 2021-08-23
• Status Verified: 2022-04
• Results First Submitted: 2022-04-06
• Results First Submitted QC: 2022-05-04
• Last Update Submitted: 2022-05-04
• Results First Posted: 2022-05-27
• Last Update Posted: 2022-05-27

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 65

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)

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
RT001	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
Placebo	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

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
Change From Baseline in the Timed 1 Minute Walk Distance	11 months	Change from baseline Distance walked in 1 minute at 11 months

Trial Locations

Locations (6)

Children's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States

Collaborative Neuroscience Network, LLC; 🇺🇸 Long Beach, California, United States

University of Iowa Stead Family Children's Hospital; 🇺🇸 Iowa City, Iowa, United States

UCLA; 🇺🇸 Los Angeles, California, United States

University of Florida; 🇺🇸 Gainesville, Florida, United States

USF Ataxia Research Center; 🇺🇸 Tampa, Florida, United States