Safety and Tolerability of Phenylbutyrate in Inclusion Body Myositis

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.

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Key information

Conditions

Inclusion Body Myositis Muscular Diseases Musculoskeletal Diseases Myositis Myositis, Inclusion Body Nervous System Diseases Neuromuscular Diseases Sporadic Inclusion Body Myositis

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

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.

Other names: PBA

Primary outcomes

Safety and Tolerability of Phenylbutyrate Measured by Incidence of Adverse Events

Time frame: 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 outcomes

Inclusion Body Myositis Functional Rating Scale (IBMFRS)

Time frame: 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.

Sponsors and collaborators

Lead sponsor

University of Kansas Medical Center

Other

Registry information

Important dates

Study start: 2020
Primary completion: 2022
Study completion: 2022
First posted: Jun 9, 2020
Registry last updated: Sep 19, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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