Safety and Tolerability of Phenylbutyrate in Inclusion Body Myositis

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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

Trial design

Primary purpose

Treatment

Allocation

N/A

Interventional model

Single Group Assignment

Masking

None (Open label)

10 participants in 1 patient group

Phenylbutyrate
Experimental group
Description:
Open-label phenylbutyrate
Treatment:
Drug: Phenylbutyrate Oral Tablet

Trial contacts and locations

1

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Data sourced from clinicaltrials.gov

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