Inclusion Body Myositis, Sporadic Inclusion Body Myositis
Conditions
Keywords
Inclusion Body Myositis, IBM, PBA, Phenylbutyrate
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.
Interventions
DRUGPhenylbutyrate Oral Tablet
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.
Sponsors
University of Kansas Medical Center
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety and Tolerability of Phenylbutyrate Measured by Incidence of Adverse Events | 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
| Measure | Time frame | Description |
|---|---|---|
| Inclusion Body Myositis Functional Rating Scale (IBMFRS) | 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. |
Countries
United States
Outcome results
None listed