NCT02445794

A First in Human Study of RT001 in Patients With Friedreich's Ataxia

A Randomized, Double-blind, Controlled Study to Assess the Safety, Tolerability, and Pharmacokinetics of RT001 in Patients With Friedreich's Ataxia

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 19 (actual)
• Sponsor: Biojiva LLC · Industry
• Sex: All
• Age: 18 Years – 50 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of RT001 in patients with Friedreich's ataxia.

Detailed description

Study RT001-002 is a randomized, double-blind, controlled, ascending dose study to evaluate the safety, tolerability, pharmacokinetic, disease state, and exploratory endpoints in patients with Friedreich's ataxia after oral administration. The study includes 2 dose levels of RT001.

Conditions

• Friedreich's Ataxia

Interventions

Type	Name	Description
DRUG	Low dose cohort	RT001 is encapsulated di-deutero synthetic homologue of linoleic acid ethyl ester. Each capsule contains 900 mg of RT001.
DRUG	High dose cohort	RT001 comparator is encapsulated non-deuterated linoleic acid ethyl ester.

Timeline

• Start date: 2015-08-01
• Primary completion: 2016-06-01
• Completion: 2016-07-01
• First posted: 2015-05-15
• Last updated: 2020-11-27
• Results posted: 2020-11-27

Locations

2 sites across 1 country: United States