<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/NCT03014219</url>
  </required_header>
  <id_info>
    <org_study_id>15-005574</org_study_id>
    <nct_id>NCT03014219</nct_id>
  </id_info>
  <brief_title>Phase 1 Crohn's Pediatric Sub-study of MSC AFP</brief_title>
  <official_title>A Phase I Study of Autologous Mesenchymal Stromal Cell Coated Fistula Plug in Patients With Fistulizing Crohn's Disease: Pediatric Sub-study</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>William A. Faubion, M.D.</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Mayo Clinic</source>
  <oversight_info>
    <has_dmc>No</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>
      The investigators propose to study the safety of autologous mesenchymal stromal cell transfer&#xD;
      using a biomatrix (the Gore Bio-A Fistula Plug) in a Phase I study using a single dose of 20&#xD;
      million cells. 20 patients (age 12 to 17 years) with Crohns perianal fistulas will be&#xD;
      enrolled.&#xD;
&#xD;
      Subjects will undergo standard adjuvant therapy including drainage of infection and placement&#xD;
      of a draining seton. Six weeks post placement of the draining seton, the seton will be&#xD;
      replaced with the MSC loaded Gore fistula plug as per current clinical practice. The subjects&#xD;
      will be subsequently followed for fistula response and closure for 24 months. This is an&#xD;
      autologous product derived from the patient and used only for the same patient.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Visit 1: Patients will be evaluated for eligibility (inclusion/exclusion checklist) and&#xD;
      written, informed consent will be obtained. Patients will undergo general exam with vital&#xD;
      signs. Patients will be scheduled for a fat biopsy to collect the tissue needed to grow MSC.&#xD;
      In the event there is no cell growth from the tissue obtained from the first biopsy, one&#xD;
      further attempt will made from a second tissue sample from this patient. However, if the&#xD;
      second attempt fails to grow cells, no further attempts will be made, and the subject will&#xD;
      not continue in the study.&#xD;
&#xD;
      The subjects will be subsequently followed for fistula response and closure for 24 months.&#xD;
      Study visits are Day 1, Week 2, Week 4, Week 8, Week 12, Week 24, Week 52, and Week 104. This&#xD;
      is an autologous product derived from the patient and used only for the same patient.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Withdrawn</overall_status>
  <why_stopped>
    FDA approval was not obtained until adult trial completes accrual&#xD;
  </why_stopped>
  <start_date type="Anticipated">July 2017</start_date>
  <completion_date type="Anticipated">July 2020</completion_date>
  <primary_completion_date type="Anticipated">July 2019</primary_completion_date>
  <phase>Phase 1</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <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>Number of Subjects with any Adverse Events that are related to study drug</measure>
    <time_frame>[Time Frame: 2-24 months</time_frame>
    <description>The primary endpoint of this study is to determine the safety of using adipose derived, autologous mesenchymal stromal cells (MSC) bound to the Gore Bio-A Fistula Plug for treatment of refractory CD perianal fistulae.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Number of Subjects with healing in response to the study drug treatment</measure>
    <time_frame>Time Frame: 2-24 months</time_frame>
    <description>The Outcome Measure will be the presence or absence of fistula drainage (Yes vs. No)</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">0</enrollment>
  <condition>Perianal Fistula</condition>
  <arm_group>
    <arm_group_label>Only 1 arm: treatment with MSC-AFP</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Single Treatment Group: Eligible patients will be treated with a Gore Bio-A Fistula Plug that has been coated with autologous mesenchymal stromal cells. This is a drug study, specifically phase 1 study of autologous mesenchymal stromal cells. Single dose of 20 million cells.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Only 1 arm: treatment with MSC-AFP</intervention_name>
    <description>Eligible patients will be treated with a fistula plug that has been coated with autologous mesenchymal stromal cell. Drug study. Single dose of 20 million cells.</description>
    <arm_group_label>Only 1 arm: treatment with MSC-AFP</arm_group_label>
    <other_name>mesenchymal stromal cells</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria&#xD;
&#xD;
          1. Males and females 12-17 years of age.&#xD;
&#xD;
          2. Residents of the United States.&#xD;
&#xD;
          3. Crohn's disease with single or multiple draining complex perianal fistulae (definition&#xD;
             as below) for at least three months despite standard therapy (definition below).&#xD;
&#xD;
          4. Concurrent therapies with corticosteroids, 5-ASA drugs, thiopurines, MTX, antibiotics,&#xD;
             and anti-TNF therapy are permitted.&#xD;
&#xD;
          5. All patients should have undergone a colonoscopy in last 12 months to rule out&#xD;
             malignant or premalignant condition&#xD;
&#xD;
          6. Have no contraindications to MR evaluations: e.g. pacemaker or magnetically active&#xD;
             metal fragments, claustrophobia&#xD;
&#xD;
          7. Ability to comply with protocol&#xD;
&#xD;
          8. Competent and able to provide written informed consent (and assent where appropriate).&#xD;
&#xD;
          9. Must have failed standard medical therapy including anti-TNF agents&#xD;
&#xD;
        Exclusion Criteria&#xD;
&#xD;
          1. Inability to obtain informed consent (and assent where appropriate).&#xD;
&#xD;
          2. Clinically significant medical conditions within the six months before administration&#xD;
             of MSCs: e.g. sepsis, pneumonia active serious infection or other conditions that&#xD;
             would, in the opinion of the investigators, compromise the safety of the patient.&#xD;
&#xD;
          3. Specific exclusions;&#xD;
&#xD;
             a. Evidence of hepatitis B, C, or HIV&#xD;
&#xD;
          4. History of cancer including melanoma (with the exception of localized skin cancers)&#xD;
&#xD;
          5. Investigational drug within thirty (30) days of baseline&#xD;
&#xD;
          6. A resident outside the United States&#xD;
&#xD;
          7. Pregnant or trying to become pregnant, or breast feeding.&#xD;
&#xD;
          8. History of clinically significant auto-immunity (other than Crohn's disease) or any&#xD;
             previous example of fat-directed autoimmunity&#xD;
&#xD;
          9. Previous allergic reaction to a perianal fistula plug.&#xD;
&#xD;
         10. If adipose tissue is not technically feasible&#xD;
&#xD;
         11. Weight less than 35 kg&#xD;
&#xD;
         12. Allergic to local anesthetics&#xD;
&#xD;
         13. Non-enterocutaneous tracts (i.e. recto-vaginal, entero-vesicular)&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>12 Years</minimum_age>
    <maximum_age>17 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Michael C Stephens</last_name>
    <role>Principal Investigator</role>
    <affiliation>Mayo Clinic</affiliation>
  </overall_official>
  <overall_official>
    <last_name>William Faubion</last_name>
    <role>Principal Investigator</role>
    <affiliation>Mayo Clinic</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Mayo Clinic in Rochester</name>
      <address>
        <city>Rochester</city>
        <state>Minnesota</state>
        <zip>55905</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>July 2017</verification_date>
  <study_first_submitted>January 4, 2017</study_first_submitted>
  <study_first_submitted_qc>January 5, 2017</study_first_submitted_qc>
  <study_first_posted type="Estimate">January 9, 2017</study_first_posted>
  <last_update_submitted>July 3, 2017</last_update_submitted>
  <last_update_submitted_qc>July 3, 2017</last_update_submitted_qc>
  <last_update_posted type="Actual">July 6, 2017</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor-Investigator</responsible_party_type>
    <investigator_affiliation>Mayo Clinic</investigator_affiliation>
    <investigator_full_name>William A. Faubion, M.D.</investigator_full_name>
    <investigator_title>Sponsor Investigator</investigator_title>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Fistula</mesh_term>
  </condition_browse>
  <!-- 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/NCT03014219
org study id: 15-005574
nct id: NCT03014219
lead sponsor: 
has dmc: No
is fda regulated drug: Yes
is fda regulated device: No
The investigators propose to study the safety of autologous mesenchymal stromal cell transfer
      using a biomatrix (the Gore Bio-A Fistula Plug) in a Phase I study using a single dose of 20
      million cells. 20 patients (age 12 to 17 years) with Crohns perianal fistulas will be
      enrolled.

      Subjects will undergo standard adjuvant therapy including drainage of infection and placement
      of a draining seton. Six weeks post placement of the draining seton, the seton will be
      replaced with the MSC loaded Gore fistula plug as per current clinical practice. The subjects
      will be subsequently followed for fistula response and closure for 24 months. This is an
      autologous product derived from the patient and used only for the same patient.
Visit 1: Patients will be evaluated for eligibility (inclusion/exclusion checklist) and
      written, informed consent will be obtained. Patients will undergo general exam with vital
      signs. Patients will be scheduled for a fat biopsy to collect the tissue needed to grow MSC.
      In the event there is no cell growth from the tissue obtained from the first biopsy, one
      further attempt will made from a second tissue sample from this patient. However, if the
      second attempt fails to grow cells, no further attempts will be made, and the subject will
      not continue in the study.

      The subjects will be subsequently followed for fistula response and closure for 24 months.
      Study visits are Day 1, Week 2, Week 4, Week 8, Week 12, Week 24, Week 52, and Week 104. This
      is an autologous product derived from the patient and used only for the same patient.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Number of Subjects with any Adverse Events that are related to study drug
time frame: [Time Frame: 2-24 months
description: The primary endpoint of this study is to determine the safety of using adipose derived, autologous mesenchymal stromal cells (MSC) bound to the Gore Bio-A Fistula Plug for treatment of refractory CD perianal fistulae.
measure: Number of Subjects with healing in response to the study drug treatment
time frame: Time Frame: 2-24 months
description: The Outcome Measure will be the presence or absence of fistula drainage (Yes vs. No)
arm group label: Only 1 arm: treatment with MSC-AFP
arm group type: Experimental
description: Single Treatment Group: Eligible patients will be treated with a Gore Bio-A Fistula Plug that has been coated with autologous mesenchymal stromal cells. This is a drug study, specifically phase 1 study of autologous mesenchymal stromal cells. Single dose of 20 million cells.
intervention type: Drug
intervention name: Only 1 arm: treatment with MSC-AFP
description: Eligible patients will be treated with a fistula plug that has been coated with autologous mesenchymal stromal cell. Drug study. Single dose of 20 million cells.
arm group label: Only 1 arm: treatment with MSC-AFP
other name: mesenchymal stromal cells
criteria: 
gender: All
minimum age: 12 Years
maximum age: 17 Years
healthy volunteers: No
last name: Michael C Stephens
role: Principal Investigator
affiliation: Mayo Clinic
last name: William Faubion
role: Principal Investigator
affiliation: Mayo Clinic
facility: 
country: United States
responsible party type: Sponsor-Investigator
investigator affiliation: Mayo Clinic
investigator full name: William A. Faubion, M.D.
investigator title: Sponsor Investigator
mesh term: Fistula