<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT04421677</url>
  </required_header>
  <id_info>
    <org_study_id>PBA in IBM</org_study_id>
    <nct_id>NCT04421677</nct_id>
  </id_info>
  <brief_title>Safety and Tolerability of Phenylbutyrate in Inclusion Body Myositis</brief_title>
  <official_title>Safety and Tolerability of Phenylbutyrate in Inclusion Body Myositis</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>University of Kansas Medical Center</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>University of Kansas Medical Center</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      This is a pilot study (phase 1 clinical trial) to evaluate the safety and tolerability of&#xD;
      phenylbutyrate in IBM. In this open label study, 10 patients with sporadic inclusion body&#xD;
      myositis will be treated with phenylbutyrate (3 gm twice daily) for 3 months. There will be a&#xD;
      run-in period, during which certain biomarkers will be measured at baseline and at the end of&#xD;
      the run-in period in addition to final measurement at the end of the treatment period.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">August 20, 2020</start_date>
  <completion_date type="Actual">January 20, 2022</completion_date>
  <primary_completion_date type="Actual">January 20, 2022</primary_completion_date>
  <phase>Phase 1</phase>
  <study_type>Interventional</study_type>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Safety and Tolerability of Phenylbutyrate Measured by Incidence of Adverse Events</measure>
    <time_frame>Month 3 - Month 6</time_frame>
    <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.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Inclusion Body Myositis Functional Rating Scale (IBMFRS)</measure>
    <time_frame>Month 6</time_frame>
    <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.</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">10</enrollment>
  <condition>Inclusion Body Myositis</condition>
  <condition>Sporadic Inclusion Body Myositis</condition>
  <arm_group>
    <arm_group_label>Phenylbutyrate</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Open-label phenylbutyrate</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Phenylbutyrate Oral Tablet</intervention_name>
    <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.</description>
    <arm_group_label>Phenylbutyrate</arm_group_label>
    <other_name>PBA</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Fulfill ENMC 2011 diagnostic criteria for IBM&#xD;
&#xD;
          -  Age &gt; 18 years&#xD;
&#xD;
          -  Women must be post-menopausal (no menses in &gt;12 months) or status post hysterectomy&#xD;
&#xD;
          -  Able to give informed consent&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Presence of any one of the following medical conditions: chronic infection; chronic&#xD;
             renal insufficiency; cancer other than skin cancer less than five years prior;&#xD;
             multiple sclerosis or prior episode of central nervous system demyelination; or other&#xD;
             chronic serious medical illnesses&#xD;
&#xD;
          -  Presence of any of the following on routine blood screening: WBC&lt;3000; Platelets &lt;&#xD;
             100,000; hematocrit &lt; 30%; BUN &gt; 30 mg %; creatine &gt; 1.5 mg%; liver disease with serum&#xD;
             albumin &lt; 3 G/DL&#xD;
&#xD;
          -  Women who are pregnant or lactating&#xD;
&#xD;
          -  History of non-compliance with other therapies&#xD;
&#xD;
          -  Coexistence of other muscular disease&#xD;
&#xD;
          -  Drug or alcohol abuse within past three months&#xD;
&#xD;
          -  Known bleeding disorder&#xD;
&#xD;
          -  Known liver disease&#xD;
&#xD;
          -  Known congestive heart failure&#xD;
&#xD;
          -  Known hypernatremia&#xD;
&#xD;
          -  Inability to give informed consent&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Duaa Jabari, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>University of Kansas Medical Center</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>University of Kansas Medical Center</name>
      <address>
        <city>Kansas City</city>
        <state>Kansas</state>
        <zip>66160</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>May 2022</verification_date>
  <study_first_submitted>May 18, 2020</study_first_submitted>
  <study_first_submitted_qc>June 4, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">June 9, 2020</study_first_posted>
  <last_update_submitted>May 19, 2022</last_update_submitted>
  <last_update_submitted_qc>May 19, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">May 23, 2022</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Inclusion Body Myositis</keyword>
  <keyword>IBM</keyword>
  <keyword>PBA</keyword>
  <keyword>Phenylbutyrate</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Myositis</mesh_term>
    <mesh_term>Myositis, Inclusion Body</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>4-phenylbutyric acid</mesh_term>
  </intervention_browse>
  <patient_data>
    <sharing_ipd>Undecided</sharing_ipd>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT04421677
org study id: PBA in IBM
nct id: NCT04421677
lead sponsor: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
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.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Safety and Tolerability of Phenylbutyrate Measured by Incidence of Adverse Events
time frame: Month 3 - Month 6
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.
measure: Inclusion Body Myositis Functional Rating Scale (IBMFRS)
time frame: Month 6
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.
arm group label: Phenylbutyrate
arm group type: Experimental
description: Open-label phenylbutyrate
intervention type: Drug
intervention name: Phenylbutyrate Oral Tablet
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.
arm group label: Phenylbutyrate
other name: PBA
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Duaa Jabari, MD
role: Principal Investigator
affiliation: University of Kansas Medical Center
facility: 
country: United States
responsible party type: Sponsor
mesh term: Myositis
mesh term: Myositis, Inclusion Body
mesh term: 4-phenylbutyric acid
sharing ipd: Undecided