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

ClinicalTrials.gov Identifier: NCT04102501, September 25, 2019.

Retrotope, Inc, drug RT001 (9-cis, 12-cis-11,11-D2-linoleic acid ethyl ester), Phase 3
Ages Eligible for Study: 12 Years to 50 Years (Child, Adult).

Locations, United States: Collaborative Neuroscience Network, LLC (Long Beach, California), UCLA (Los Angeles, California), USF Ataxia Research Center (Tampa, Florida), University of Iowa Stead Family Children's Hospital (Iowa City, Iowa) and Children's Hospital of Philadelphia (Philadelphia, Pennsylvania)

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 washout period.

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