Conditions: Friedreich's Ataxia
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 19
Healthy volunteers: no

Brief 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.

Arms:
arm 1: RT001, oral, 1.8 g QD for 28 days or matching comparator arm 2: RT001, oral, 4.5 g BID for 28 days or matching comparator

Interventions: intervention 1: Low dose cohort intervention 2: High dose cohort
Intervention descriptions: intervention 1: RT001 is encapsulated di-deutero synthetic homologue of linoleic acid ethyl ester. Each capsule contains 900 mg of RT001. intervention 2: RT001 comparator is encapsulated non-deuterated linoleic acid ethyl ester.

Protocol document: