Safety and Tolerability of Phenylbutyrate in Inclusion Body Myositis

University of Kansas

Status and phase

Completed

Phase 1

Conditions

Sporadic Inclusion Body Myositis

Inclusion Body Myositis

Treatments

Drug: Phenylbutyrate Oral Tablet

Study type

Interventional

Funder types

Other

Identifiers

NCT04421677

PBA in IBM

Details and patient eligibility

About

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.

Enrollment

10 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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