Safety and Tolerability of Phenylbutyrate in Inclusion Body Myositis

NCT ID: NCT04421677

Last Updated: 2024-09-19

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 10 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-08-20
• Study Completion Date: 2022-01-20

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.

Conditions

Inclusion Body Myositis; Sporadic Inclusion Body Myositis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Phenylbutyrate

Open-label phenylbutyrate

Group Type: EXPERIMENTAL

Phenylbutyrate Oral Tablet

Intervention Type: DRUG

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.

Interventions

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.

Intervention Type: DRUG

Other Intervention Names

PBA

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University of Kansas Medical Center

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Duaa Jabari, MD

Role: PRINCIPAL_INVESTIGATOR

University of Kansas Medical Center

Locations

University of Kansas Medical Center

Kansas City, Kansas, United States

Countries

United States

Other Identifiers

PBA in IBM

Identifier Type: -

Identifier Source: org_study_id