Status:
COMPLETED
Safety and Tolerability of Phenylbutyrate in Inclusion Body Myositis
Lead Sponsor:
University of Kansas Medical Center
Conditions:
Inclusion Body Myositis
Sporadic Inclusion Body Myositis
Eligibility:
All Genders
18+ years
Phase:
PHASE1
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 tre...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
August 20 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 20 2022
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT04421677
Start Date
August 20 2020
End Date
January 20 2022
Last Update
September 19 2024
Active Locations (1)
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1
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160