NCT04102501

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

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

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 65 (actual)
• Sponsor: Biojiva LLC · Industry
• Sex: All
• Age: 12 Years – 50 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Friedreich's Ataxia

Interventions

Type	Name	Description
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.
DRUG	Placebo	The placebo product is composed of encapsulated USP safflower oil. The placebo capsules are identical in appearance and size to RT001.

Timeline

• Start date: 2019-10-30
• Primary completion: 2021-08-23
• Completion: 2021-08-23
• First posted: 2019-09-25
• Last updated: 2022-05-27
• Results posted: 2022-05-27

Locations

6 sites across 1 country: United States

Regulatory

• FDA-regulated drug study