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Safety and Tolerability of Phenylbutyrate in Inclusion Body Myositis

NCT04421677 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2024-09-19

No results posted yet for this study

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

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.

Sponsors & Collaborators

  • University of Kansas Medical Center

    lead OTHER

Principal Investigators

  • Duaa Jabari, MD · University of Kansas Medical Center

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-08-20
Primary Completion
2022-01-20
Completion
2022-01-20
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04421677 on ClinicalTrials.gov