An expanded access protocol of RT001 in amyotrophic lateral sclerosis-Initial experience with a lipid peroxidation inhibitor.

ABSTRACT:

Introduction/aims

Lipid peroxidation is thought to play a biologically important role in motor neuron death in amyotrophic lateral sclerosis (ALS). 11,11 Di-deuterated linoleic ethyl ester (RT001) prevents lipid peroxidation in cellular and mitochondrial membranes. Herein we report on the use of RT001 under expanded access (EA).

Methods

We provided RT001 to patients with ALS via EA at a single site. The starting dose was 2.88 g/day, which was increased to to 8.64 g/day as tolerated. Participants were not eligible for alternative clinical trials. Participants were followed for adverse events and pharmacokinetic (PK) parameters were measured approximately 3 months after RT001 initiation.

Results

Sixteen participants received RT001 (5.6 ± 1.6 g/day; dose range, 1.92 to 8

SUBMITTER: Yerton M 

PROVIDER: S-EPMC9796343 | biostudies-literature | 2022 Oct

REPOSITORIES: biostudies-literature