Safety and Tolerability of Phenylbutyrate in Inclusion Body Myositis

Inclusion Body Myositis Clinical Trial

Official title:

Safety and Tolerability of Phenylbutyrate in Inclusion Body Myositis

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04421677
• Other study ID #: PBA in IBM
• Status: Completed
• Phase: Phase 1
• Start date: August 20, 2020
• Est. completion date: January 20, 2022

Study information

• Verified date: May 2022
• Source: University of Kansas Medical Center
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 10
• Est. completion date: January 20, 2022
• Est. primary completion date: January 20, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Inclusion Body Myositis
• Myositis
• Myositis, Inclusion Body
• Sporadic Inclusion Body Myositis

Intervention

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.

Locations

Country	Name	City	State
United States	University of Kansas Medical Center	Kansas City	Kansas

Sponsors (1)

Lead Sponsor	Collaborator
University of Kansas Medical Center

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety and Tolerability of Phenylbutyrate Measured by Incidence of Adverse Events	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.	Month 3 - Month 6
Secondary	Inclusion Body Myositis Functional Rating Scale (IBMFRS)	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.	Month 6