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

Friedreich's Ataxia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04102501
• Other study ID #: RT001-006
• Status: Completed
• Phase: Phase 3
• Start date: October 30, 2019
• Est. completion date: August 23, 2021

Study information

• Verified date: April 2022
• Source: Retrotope, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 65
• Est. completion date: August 23, 2021
• Est. primary completion date: August 23, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years to 50 Years

Eligibility

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)

Related Conditions & MeSH terms

• Ataxia
• Cerebellar Ataxia
• Friedreich Ataxia
• Friedreich's Ataxia

Intervention

Drug:
• RT001
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.
• Placebo
The placebo product is composed of encapsulated USP safflower oil. The placebo capsules are identical in appearance and size to RT001.

Locations

Country	Name	City	State
United States	University of Florida	Gainesville	Florida
United States	University of Iowa Stead Family Children's Hospital	Iowa City	Iowa
United States	Collaborative Neuroscience Network, LLC	Long Beach	California
United States	UCLA	Los Angeles	California
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	USF Ataxia Research Center	Tampa	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Retrotope, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Change From Baseline in the Timed 1 Minute Walk Distance	Change from baseline Distance walked in 1 minute at 11 months	11 months