Safety and Tolerability of Phenylbutyrate in Inclusion Body Myositis

Summary

This is a pilot study (phase 1 clinical trial) to evaluate the safety and tolerability of phenylbutyrate in IBM. In this open label study, 10 patients with sporadic inclusion body myositis will be treated with phenylbutyrate (3 gm twice daily) for 3 months. There will be a run-in period, during which certain biomarkers will be measured at baseline and at the end of the run-in period in addition to final measurement at the end of the treatment period.

Timeline

Start

2020-08-20

Primary completion

2022-01-20

Completion

2022-01-20

Drugs

Indications