Randomized, clinical trial of RT001: Early signals of efficacy in Friedreich’s ataxia
RT001 is a deuterated ethyl linoleate that inhibits lipid peroxidation and is hypothesized to reduce cellular damage and recover mitochondrial function in degenerative diseases such as Friedreich’s ataxia.
To evaluate the safety, pharmacokinetics, and preliminary efficacy of RT001 in Friedreich’s ataxia patients.
We conducted a phase I/II double‐blind, comparator‐controlled trial with 2 doses of RT001 in Friedreich’s ataxia patients (9 subjects each cohort). Subjects were randomized 2:1 to receive either RT001 (1.8 or 9.0 g/day), or a matching dose of nondeuterated ethyl linoleate as comparator for 28 days. The primary endpoints were safety, tolerability, and pharmacokinetic analysis. Secondary endpoints included cardiopulmonary exercise testing and timed 25‐foot walk.