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

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

Tracking Information

• First Submitted Date: December 15, 2022
• First Posted Date: December 23, 2022
• Last Update Posted Date: March 21, 2024
• Estimated Study Start Date: April 2024
• Estimated Primary Completion Date: December 31, 2024 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: December 15, 2022)

• 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
• 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
• 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
• 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
• 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
• 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

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: December 15, 2022)

• 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
• 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

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

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

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.

• 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 Type: Interventional
• Study Phase: Phase 1
• Study Design: Allocation: Non-Randomized; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Gastro-Intestinal Disorder

Intervention

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

Study Arms

• Experimental: Stratum A
  Diagnosed with GvHD
  Intervention: Drug: Fecal microbiota transplant (FMT)
• Experimental: Stratum B
  GI Dysfunction
  Intervention: Drug: Fecal microbiota transplant (FMT)

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Not yet recruiting
• Estimated Enrollment (submitted: December 15, 2022): 20
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: December 31, 2027
• Estimated Primary Completion Date: December 31, 2024 (Final data collection date for primary outcome measure)

Eligibility 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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: up to 22 Years (Child, Adult)
• Accepts Healthy Volunteers: No

Contacts

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

• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT05664113
• Other Study ID Numbers: NEWGUT; NCI-2024-02553 ( Other Identifier: NCI Clinical Trial Registration Program )
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

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
• Supporting Materials: Statistical Analysis Plan (SAP)
• Supporting Materials: 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.

• Current Responsible Party: St. Jude Children's Research Hospital
• Original Responsible Party: Same as current
• Current Study Sponsor: St. Jude Children's Research Hospital
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Principal Investigator: Gabriela Maron, MD; St. Jude Children's Research Hospital

• PRS Account: St. Jude Children's Research Hospital
• Verification Date: March 2024