CompletedPhase 1

Safety and Tolerability of Phenylbutyrate in Inclusion Body Myositis

United States10 participantsStarted 2020-08-20

Plain-language 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.

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Safety and Tolerability of Phenylbutyrate Measured by Incidence of Adverse Events

Timeframe: Month 3 - Month 6

Trial details

NCT IDNCT04421677

SponsorUniversity of Kansas Medical Center

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2022-01-20

View on ClinicalTrials.gov More Inclusion Body Myositis trials