<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/NCT03834038</url>
  </required_header>
  <id_info>
    <org_study_id>CDI.LYO.FMT (C2016-020)</org_study_id>
    <nct_id>NCT03834038</nct_id>
    <nct_alias>NCT02592343</nct_alias>
  </id_info>
  <brief_title>Lyophilized Fecal Microbiota Transplantation for Recurrent Clostridium Difficile Infection</brief_title>
  <official_title>Prospective, Open-label Trial to Evaluate Efficacy of Lyophilized Fecal Microbiota Transplantation for Treatment of Recurrent C. Difficile Infection</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Vancouver Island Health Authority</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Vancouver Island Health Authority</source>
  <oversight_info>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      The primary goal is to study participants with recurrent C. difficile infection (CDI) treated&#xD;
      with lyophilized fecal microbiota transplantation (FMT). The safety, clinical response and&#xD;
      relapse rate in patients will be assessed.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Recurrence of CDI following a course of standard antibiotic therapy is high, especially in&#xD;
      the elderly patients over 65 years of age, in hospitalized and in the immunocompromised&#xD;
      patients. As CDI is characterized by intestinal dysbiosis. Fecal Microbiology Transplantation&#xD;
      (FMT) has been investigated as alternative treatment for CDI and has been determined to be&#xD;
      effective and safe. One of the major challenges of offering FMT is the availability of&#xD;
      suitable donors. A donor may no longer be able to continue to donate for a number of reasons&#xD;
      and this may lead to temporary interruption of FMT in centers which offer the program. In&#xD;
      order to continue to offer FMT whenever needed, we will investigate the efficacy of&#xD;
      lyophilized FMT. The lyophilization (freeze-drying) process works by dehydrating a frozen&#xD;
      donor stool sample to complete dryness, using controlled temperature and pressure gradients.&#xD;
      This lyophilized process results in a powdered form of the sample. Studies have shown that&#xD;
      lyophilized donor stool samples have similar microbial compositions as the same fresh&#xD;
      sample.The technique of freeze drying has been used for decades for the industrial storage of&#xD;
      microbes and has been used. Preliminary study of lyophilized stool for FMT has been performed&#xD;
      in dogs. Preliminary efficacy data in dogs with inflammatory bowel disease suggest equal&#xD;
      efficacy as compared to fresh stool, although controlled study has yet to be performed.&#xD;
      Should the lyophilized FMT (L-FMT) demonstrate to be equally or more effective than frozen&#xD;
      FMT, there would be significant advantages. As with frozen FMT, lyophilized FMT will allow&#xD;
      patients to receive FMT immediately as it can take up to two weeks for a donor's screening&#xD;
      laboratory testing results to be available. Lyophilized FMT will also be more cost effective&#xD;
      as less number of donors will need to be screened given the prolonged shelf life of&#xD;
      lyophilized FMT which can be kept at above the freezing temperature. 9 This will also allow&#xD;
      wider distribution and accessibility especially to the regions with limited capacity to&#xD;
      manufacture FMT.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Active, not recruiting</last_known_status>
  <start_date type="Actual">February 18, 2016</start_date>
  <completion_date type="Anticipated">March 2020</completion_date>
  <primary_completion_date type="Anticipated">June 2019</primary_completion_date>
  <phase>N/A</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>Treatment of CDI</measure>
    <time_frame>13 weeks from last FMT</time_frame>
    <description>Evaluate treatment efficacy determined by no recurrence of CDI-related diarrhea</description>
  </primary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Anticipated">100</enrollment>
  <condition>Clostridium Difficile Infection</condition>
  <arm_group>
    <arm_group_label>Open Label Lyophilized Fecal Microbiota Transplantation</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Eligible participants with history of recurrent or refractory CDI</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Lyophilized Fecal Microbiota Transplantation</intervention_name>
    <description>Lyophilized FMT</description>
    <arm_group_label>Open Label Lyophilized Fecal Microbiota Transplantation</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Age ≥ 12 years or older.&#xD;
&#xD;
          -  Able to provide informed consent.&#xD;
&#xD;
          -  Willing and able to comply with all the required study procedures.&#xD;
&#xD;
          -  A positive stool test for C. difficile toxin/gene using either PCR or enzyme&#xD;
             immunoassay within 3 months of recruitment unless patient taking treatment&#xD;
             specifically for CDI for more than 3 months.&#xD;
&#xD;
          -  History of at least ≥ 2 recurrent CDI where recurrence is deﬁned as return of diarrhea&#xD;
             consistent with CDI within 8 weeks following CDI symptom resolution for at least 24&#xD;
             hours after a minimum of 10-day course of standard antibiotic therapy for each episode&#xD;
             and/or ongoing symptoms consistent with CDI* (defined below) despite at least 7 days&#xD;
             of treatment using oral vancomycin at a minimum dose of 250 mg four times daily.&#xD;
&#xD;
               -  Symptoms of CDI include: diarrhea deﬁned as: 3 or more unformed bowel movements&#xD;
                  in 24 hours for a minimum of 2 days with no other causes for diarrhea&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Planned or actively taking another investigational product&#xD;
&#xD;
          -  CDI symptom-free for 3 or more weeks following completion of CDI treatment&#xD;
&#xD;
          -  Patients with neutropenia with absolute neutrophil count &lt;0.5 x 109/L&#xD;
&#xD;
          -  Evidence of toxic megacolon or gastrointestinal perforation on abdominal x-ray&#xD;
&#xD;
          -  Active gastroenteritis due to Salmonella, Shigella, E. coli 0157H7, Yersinia or&#xD;
             Campylobacter.&#xD;
&#xD;
          -  Presence of colostomy&#xD;
&#xD;
          -  Unable to tolerate FMT or enema for any reason.&#xD;
&#xD;
          -  Requiring systemic antibiotic therapy for more than 7 days.&#xD;
&#xD;
          -  Actively taking Saccharomyces boulardii or other probiotic; yogurt is allowed&#xD;
&#xD;
          -  Severe underlying disease such that the patient is not expected to survive for at&#xD;
             least 30 days.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>12 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Christine Lee, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Vancouver Island Health Authority</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Vancouver Island Health Authority</name>
      <address>
        <city>Victoria</city>
        <state>British Columbia</state>
        <zip>V8R 1J8</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Canada</country>
  </location_countries>
  <verification_date>May 2019</verification_date>
  <study_first_submitted>February 6, 2019</study_first_submitted>
  <study_first_submitted_qc>February 6, 2019</study_first_submitted_qc>
  <study_first_posted type="Actual">February 7, 2019</study_first_posted>
  <last_update_submitted>May 15, 2019</last_update_submitted>
  <last_update_submitted_qc>May 15, 2019</last_update_submitted_qc>
  <last_update_posted type="Actual">May 17, 2019</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Infections</mesh_term>
    <mesh_term>Communicable Diseases</mesh_term>
    <mesh_term>Clostridium Infections</mesh_term>
  </condition_browse>
  <pending_results>
    <submitted>September 15, 2022</submitted>
    <returned>August 4, 2023</returned>
  </pending_results>
  <!-- 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/NCT03834038
org study id: CDI.LYO.FMT (C2016-020)
nct id: NCT03834038
nct alias: NCT02592343
lead sponsor: 
is fda regulated drug: No
is fda regulated device: No
The primary goal is to study participants with recurrent C. difficile infection (CDI) treated
      with lyophilized fecal microbiota transplantation (FMT). The safety, clinical response and
      relapse rate in patients will be assessed.
Recurrence of CDI following a course of standard antibiotic therapy is high, especially in
      the elderly patients over 65 years of age, in hospitalized and in the immunocompromised
      patients. As CDI is characterized by intestinal dysbiosis. Fecal Microbiology Transplantation
      (FMT) has been investigated as alternative treatment for CDI and has been determined to be
      effective and safe. One of the major challenges of offering FMT is the availability of
      suitable donors. A donor may no longer be able to continue to donate for a number of reasons
      and this may lead to temporary interruption of FMT in centers which offer the program. In
      order to continue to offer FMT whenever needed, we will investigate the efficacy of
      lyophilized FMT. The lyophilization (freeze-drying) process works by dehydrating a frozen
      donor stool sample to complete dryness, using controlled temperature and pressure gradients.
      This lyophilized process results in a powdered form of the sample. Studies have shown that
      lyophilized donor stool samples have similar microbial compositions as the same fresh
      sample.The technique of freeze drying has been used for decades for the industrial storage of
      microbes and has been used. Preliminary study of lyophilized stool for FMT has been performed
      in dogs. Preliminary efficacy data in dogs with inflammatory bowel disease suggest equal
      efficacy as compared to fresh stool, although controlled study has yet to be performed.
      Should the lyophilized FMT (L-FMT) demonstrate to be equally or more effective than frozen
      FMT, there would be significant advantages. As with frozen FMT, lyophilized FMT will allow
      patients to receive FMT immediately as it can take up to two weeks for a donor's screening
      laboratory testing results to be available. Lyophilized FMT will also be more cost effective
      as less number of donors will need to be screened given the prolonged shelf life of
      lyophilized FMT which can be kept at above the freezing temperature. 9 This will also allow
      wider distribution and accessibility especially to the regions with limited capacity to
      manufacture FMT.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Treatment of CDI
time frame: 13 weeks from last FMT
description: Evaluate treatment efficacy determined by no recurrence of CDI-related diarrhea
arm group label: Open Label Lyophilized Fecal Microbiota Transplantation
arm group type: Experimental
description: Eligible participants with history of recurrent or refractory CDI
intervention type: Drug
intervention name: Lyophilized Fecal Microbiota Transplantation
description: Lyophilized FMT
arm group label: Open Label Lyophilized Fecal Microbiota Transplantation
criteria: 
gender: All
minimum age: 12 Years
maximum age: N/A
healthy volunteers: No
last name: Christine Lee, MD
role: Principal Investigator
affiliation: Vancouver Island Health Authority
facility: 
country: Canada
responsible party type: Sponsor
mesh term: Infections
mesh term: Communicable Diseases
mesh term: Clostridium Infections
submitted: September 15, 2022
returned: August 4, 2023