NCT04421677

Safety and Tolerability of Phenylbutyrate in Inclusion Body Myositis

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: University of Kansas Medical Center · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Inclusion Body Myositis
• Sporadic Inclusion Body Myositis

Interventions

Type	Name	Description
DRUG	Phenylbutyrate Oral Tablet	Phenylbutyrate, an orally active chemical chaperone approved by the US Food and Drug Administration for treatment of urea cycle disorders, mimics the function of intracellular molecular chaperones in preventing protein aggregation and oligomerization.

Timeline

• Start date: 2020-08-20
• Primary completion: 2022-01-20
• Completion: 2022-01-20
• First posted: 2020-06-09
• Last updated: 2024-09-19

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study