Safety and Tolerability of Phenylbutyrate in Inclusion Body Myositis

NCT04421677 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: PBA in IBM
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

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

Keywords

Inclusion Body Myositis; IBM; PBA; Phenylbutyrate

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Inclusion Body Myositis Functional Rating Scale (IBMFRS)

  Month 6

Study Arms (1)

Phenylbutyrate

EXPERIMENTAL

Open-label phenylbutyrate

Drug: Phenylbutyrate Oral Tablet

Interventions

Phenylbutyrate Oral Tablet 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.

Also known as: PBA

Phenylbutyrate

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University of Kansas Medical Center: lead

Study Sites (1)

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

MeSH Terms

Conditions

Myositis, Inclusion Body

Interventions

Phenylbutyrates

Condition Hierarchy (Ancestors)

Myositis; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases

Intervention Hierarchy (Ancestors)

Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals

Study Officials

• Duaa Jabari, MD

  University of Kansas Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 18, 2020
• First Posted: June 9, 2020
• Study Start: August 20, 2020
• Primary Completion: January 20, 2022
• Study Completion: January 20, 2022
• Last Updated: September 19, 2024

Record last verified: 2022-05

Locations

US (1)