Safety and Tolerability of Phenylbutyrate in Inclusion Body Myositis
Status:
Active, not recruiting
Trial end date:
2022-01-01
Target enrollment:
Participant gender:
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.