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Feasibility, Safety, and Potential Efficacy of Fecal Microbiota Transplantation (FMT) for Gastrointestinal Dysfunction in Children Following Hematopoietic Cell Transplant (HCT).

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ClinicalTrials.gov Identifier: NCT05664113
Recruitment Status : Not yet recruiting
First Posted : December 23, 2022
Last Update Posted : March 21, 2024
Sponsor:
Information provided by (Responsible Party):
St. Jude Children's Research Hospital

Study Description
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Brief Summary:

The study participant is being asked to take part in this clinical trial, a type of research study, because the participant has Gastrointestinal (GI) symptoms following a Hematopoietic Cell Transplant (HCT).

Primary Objective

  • To determine the safety and feasibility of FMT for treating a GvHD of the gut following HCT.
  • To determine the safety and feasibility of FMT for treating HCT induced gut dysfunction.

Secondary Objectives

  • To assess the potential efficacy of FMT for treating a GvHD of the gut following HCT.
  • To assess the potential efficacy of FMT for treating HCT induced gut dysfunction.

Condition or disease Intervention/treatment Phase
Gastro-Intestinal Disorder Drug: Fecal microbiota transplant (FMT) Phase 1

Detailed Description:
Participants will be eligible to receive an FMT on or after Day +30 post-HCT. FMT will be performed using FMP material obtained from OpenBiome. 60 mL of FMP will be administered via NJ tube and 250 mL via colonoscopy. A second FMT may be performed at least 14 days after the initial FMT in GI clinical symptoms have partially improved or have not changed. The second FMT will be administered using the same procedure as in the initial FMT
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Feasibility, Safety, and Potential Efficacy of Fecal Microbiota Transplantation (FMT) for Gastrointestinal Dysfunction in Children Following Hematopoietic Cell Transplant (HCT).
Estimated Study Start Date : April 2024
Estimated Primary Completion Date : December 31, 2024
Estimated Study Completion Date : December 31, 2027

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Bowel Movement

Arms and Interventions
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Arm Intervention/treatment
Experimental: Stratum A
Diagnosed with GvHD
 Drug: Fecal microbiota transplant (FMT)
FMT Lower Delivery Microbiota Preparation, Dose: 250 mL of Microbiota Preparation Material and Route of administration: colonoscopy FMT Upper Delivery Microbiota Preparation, Dose: 60 mL of Microbiota Preparation Material and Route of administration: Naso-enteral tube
Other Name: FMT
Experimental: Stratum B
GI Dysfunction
 Drug: Fecal microbiota transplant (FMT)
FMT Lower Delivery Microbiota Preparation, Dose: 250 mL of Microbiota Preparation Material and Route of administration: colonoscopy FMT Upper Delivery Microbiota Preparation, Dose: 60 mL of Microbiota Preparation Material and Route of administration: Naso-enteral tube
Other Name: FMT


Outcome Measures
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Primary Outcome Measures :
  1. Proportion of participants with a serious adverse event occurring within 30 days following FMT [ Time Frame: 30 Days ]

    Serious adverse events will be a primary outcome measure for the following groups:

    • FMT for treating a GvHD of the gut following HCT
    • FMT for treating HCT induced gut dysfunction

  2. Proportion of participants with a non-serious adverse event occurring within 30 days following FMT [ Time Frame: 30 Days ]

    Non-serious adverse events will be a primary outcome measure for the following groups:

    • FMT for treating a GvHD of the gut following HCT
    • FMT for treating HCT induced gut dysfunction

  3. Proportion of patients expressing interest who meet eligibility [ Time Frame: 2 years ]

    Participant eligibility will be a primary outcome measure for the following groups:

    • FMT for treating a GvHD of the gut following HCT
    • FMT for treating HCT induced gut dysfunction

  4. Proportion of patients recruited in the eligible population [ Time Frame: 2 years ]

    Participant recruitment will be a primary outcome measure for the following groups:

    • FMT for treating a GvHD of the gut following HCT
    • FMT for treating HCT induced gut dysfunction

  5. Proportion of participants that drop up post-enrollment [ Time Frame: 3 years ]

    Participant retention will be a primary outcome measure for the following groups:

    • FMT for treating a GvHD of the gut following HCT
    • FMT for treating HCT induced gut dysfunction

  6. Proportion of participants providing all protocol required stool samples [ Time Frame: 3 years ]

    Stool specimens will be a primary outcome measure for the following groups:

    • FMT for treating a GvHD of the gut following HCT
    • FMT for treating HCT induced gut dysfunction


Secondary Outcome Measures :
  1. Proportion of participants with a complete response or a partial response [ Time Frame: 180 days ]

    Complete response or partial response will be a secondary outcome measure for the following groups:

    • FMT for treating a GvHD of the gut following HCT
    • FMT for treating HCT induced gut dysfunction

  2. Percentage of participants who reduce or discontinue steroids at the end of the study [ Time Frame: 1 year ]

    Reduction in dose of steroids will be a secondary outcome measure for the following groups:

    • FMT for treating a GvHD of the gut following HCT
    • FMT for treating HCT induced gut dysfunction


Eligibility Criteria
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Ages Eligible for Study:   up to 22 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Age < 22 years old.
  • Received an allogeneic HCT greater than or equal to 30 days prior to enrollment

  • Diagnosed with one of the following conditions:

    1. Steroid-resistant gut a GvHD (defined as GI symptoms that do not improve within 5 days after initial steroid therapy, >/= 1mg/kg of prednisolone) OR

    2. Steroid-dependent gut a GvHD (defined as the presence of a response to methylprednisolone 2 mg/kg/day but relapsing when an attempt was made to taper steroid treatment).

      OR

    3. Current or prolonged GI dysfunction following HCT, defined as having diarrhea or loose stools >/= 4 weeks with at least one of the following:

      1. Requiring NG or G-tube feeds
      2. Requiring TPN or IVF for more than 4 weeks
      3. Diagnosis of gastroparesis by GI specialist documented in the medical record
  • Willing and able to provide informed assent/consent

Exclusion Criteria:

  • Cytomegalovirus (CMV) or Epstein Barr Virus (EBV) IgG negative at the time of consent
  • Female participant who is pregnant or nursing
  • History of previous FMT
  • Intra-abdominal surgery within 4 weeks of enrollment
  • At increased risk for peritonitis: presence of intra-abdominal devices (G-or GJ-tubes are acceptable), receiving peritoneal dialysis, or ascites
  • Concurrent abdominal radiation therapy
  • Any acute or chronic illness/condition as well as medication that in the opinion of the investigator puts the subject at greater risk from FMT or may confound the study results.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05664113


Contacts
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Contact: Gabriela Maron, MD 866-278-5833 referrainfo@stjude.org

Locations
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United States, Tennessee
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Contact: Gabriela Maron, MD    866-278-5833    referralinfo@stjude.org   
Sponsors and Collaborators
St. Jude Children's Research Hospital
Investigators
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Principal Investigator: Gabriela Maron, MD St. Jude Children's Research Hospital
More Information
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Additional Information:

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Responsible Party: St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier: NCT05664113    
Other Study ID Numbers: NEWGUT
NCI-2024-02553 ( Other Identifier: NCI Clinical Trial Registration Program )
First Posted: December 23, 2022    Key Record Dates
Last Update Posted: March 21, 2024
Last Verified: March 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Individual participant de-identified datasets containing the variables analyzed in the published article will be made available (related to the study primary or secondary objectives contained in the publication). Supporting documents such as the protocol, statistical analyses plan, and informed consent are available through the CTG website for the specific study. Data used to generate the published article will be made available at the time of article publication. Investigators who seek access to individual level de-identified data will contact the computing team in the Department of Biostatistics (ClinTrialDataRequest@stjude.org) who will respond to the data request.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Time Frame: Data will be made available at the time of article publication.
Access Criteria: Data will be provided to researchers following a formal request with the following information: full name of requestor, affiliation, data set requested, and timing of when data is needed. As an informational point, the lead statistician and study principal investigator will be informed that primary results datasets have been requested.

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Intestinal Diseases
Gastrointestinal Diseases
Digestive System Diseases