Safety and Tolerability of Phenylbutyrate in Inclusion Body Myositis

Phase 1

Completed

• Conditions: Sporadic Inclusion Body Myositis; Inclusion Body Myositis
• Interventions: Drug: Phenylbutyrate Oral Tablet

• Registration Number: NCT04421677
• Lead Sponsor: University of Kansas Medical Center

Brief 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2020-05-18
• Study First Submitted QC: 2020-06-04
• Study First Posted: 2020-06-09
• Study Start: 2020-08-20
• Primary Completion: 2022-01-20
• Study Completion: 2022-01-20
• Status Verified: 2022-05
• Last Update Submitted: 2022-05-19
• Last Update Posted: 2022-05-23

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

• Fulfill ENMC 2011 diagnostic criteria for IBM
• Age > 18 years
• Women must be post-menopausal (no menses in >12 months) or status post hysterectomy
• Able to give informed consent

Exclusion Criteria

• Presence of any one of the following medical conditions: chronic infection; chronic renal insufficiency; cancer other than skin cancer less than five years prior; multiple sclerosis or prior episode of central nervous system demyelination; or other chronic serious medical illnesses
• Presence of any of the following on routine blood screening: WBC<3000; Platelets < 100,000; hematocrit < 30%; BUN > 30 mg %; creatine > 1.5 mg%; liver disease with serum albumin < 3 G/DL
• Women who are pregnant or lactating
• History of non-compliance with other therapies
• Coexistence of other muscular disease
• Drug or alcohol abuse within past three months
• Known bleeding disorder
• Known liver disease
• Known congestive heart failure
• Known hypernatremia
• Inability to give informed consent

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Phenylbutyrate	Phenylbutyrate Oral Tablet	Open-label phenylbutyrate

Primary Outcome Measures

Name	Time	Method
Safety and Tolerability of Phenylbutyrate Measured by Incidence of Adverse Events	Month 3 - Month 6	Since this is a Phase I pilot trial, the primary outcome will be testing the safety and tolerability of phenylbutyrate in patients with Inclusion Body Myositis. This will be measured by the incidence of adverse events reported in subjects throughout the treatment period of the trial.

Secondary Outcome Measures

Name	Time	Method
Inclusion Body Myositis Functional Rating Scale (IBMFRS)	Month 6	The IBMFRS is a quickly administered (10-minute) ordinal rating scale (ratings 0-40) used to determine patients' assessment of their capability and independence in 10 functional activities. The higher a subject scores on the scale, the better functional ability the subject has.

Trial Locations

Locations (1)

University of Kansas Medical Center; 🇺🇸 Kansas City, Kansas, United States