Pilot trial of inosine to elevate urate levels in amyotrophic lateral sclerosis.

ABSTRACT:

Objective

To test the safety, tolerability, and urate-elevating capability of the urate precursor inosine taken orally or by feeding tube in people with amyotrophic lateral sclerosis (ALS).

Methods

This was a pilot, open-label trial in 25 participants with ALS. Treatment duration was 12 weeks. The dose of inosine was titrated at pre-specified time points to elevate serum urate levels to 7-8 mg/dL. Primary outcomes were safety (as assessed by the occurrence of adverse events [AEs]) and tolerability (defined as the ability to complete the 12-week study on study drug). Secondary outcomes included biomarkers of oxidative stress and damage. As an exploratory analysis, observed outcomes were compared with a virtual control arm built using prediction algorithms to estimate ALSFRS-R

SUBMITTER: Nicholson K 

PROVIDER: S-EPMC6292193 | biostudies-literature | 2018 Dec

REPOSITORIES: biostudies-literature