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Safety and Tolerability of Phenylbutyrate in Inclusion Body Myositis

Primary Purpose

Inclusion Body Myositis, Sporadic Inclusion Body Myositis

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Phenylbutyrate Oral Tablet
Sponsored by
University of Kansas Medical Center
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Inclusion Body Myositis focused on measuring Inclusion Body Myositis, IBM, PBA, Phenylbutyrate

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

  • University of Kansas Medical Center

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Phenylbutyrate

Arm Description

Open-label phenylbutyrate

Outcomes

Primary Outcome Measures

Safety and Tolerability of Phenylbutyrate Measured by Incidence of Adverse Events
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 Outcome Measures

Inclusion Body Myositis Functional Rating Scale (IBMFRS)
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.

Full Information

First Posted
May 18, 2020
Last Updated
May 19, 2022
Sponsor
University of Kansas Medical Center
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1. Study Identification

Unique Protocol Identification Number
NCT04421677
Brief Title
Safety and Tolerability of Phenylbutyrate in Inclusion Body Myositis
Official Title
Safety and Tolerability of Phenylbutyrate in Inclusion Body Myositis
Study Type
Interventional

2. Study Status

Record Verification Date
May 2022
Overall Recruitment Status
Completed
Study Start Date
August 20, 2020 (Actual)
Primary Completion Date
January 20, 2022 (Actual)
Study Completion Date
January 20, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University of Kansas Medical Center

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Inclusion Body Myositis, Sporadic Inclusion Body Myositis
Keywords
Inclusion Body Myositis, IBM, PBA, Phenylbutyrate

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Phenylbutyrate
Arm Type
Experimental
Arm Description
Open-label phenylbutyrate
Intervention Type
Drug
Intervention Name(s)
Phenylbutyrate Oral Tablet
Other Intervention Name(s)
PBA
Intervention Description
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.
Primary Outcome Measure Information:
Title
Safety and Tolerability of Phenylbutyrate Measured by Incidence of Adverse Events
Description
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.
Time Frame
Month 3 - Month 6
Secondary Outcome Measure Information:
Title
Inclusion Body Myositis Functional Rating Scale (IBMFRS)
Description
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.
Time Frame
Month 6

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Duaa Jabari, MD
Organizational Affiliation
University of Kansas Medical Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
Undecided

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Safety and Tolerability of Phenylbutyrate in Inclusion Body Myositis

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