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

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Gastro-Intestinal Disorder
• Intervention / Treatment: Drug: Fecal microbiota transplant (FMT)

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
• Enrollment (Estimated): 20
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Gabriela Maron, MD; Phone Number: 866-278-5833; Email: referrainfo@stjude.org

Study Locations

• United States
  • Tennessee
    • Memphis, Tennessee, United States, 38105
      • St. Jude Children's Research Hospital
      • Contact: Gabriela Maron, MD; Phone Number: 866-278-5833; Email: referralinfo@stjude.org

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 22 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / 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 Names: 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 Names: FMT

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of participants with a serious adverse event occurring within 30 days following FMT	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	30 Days
Proportion of participants with a non-serious adverse event occurring within 30 days following FMT	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	30 Days
Proportion of patients expressing interest who meet eligibility	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	2 years
Proportion of patients recruited in the eligible population	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	2 years
Proportion of participants that drop up post-enrollment	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	3 years
Proportion of participants providing all protocol required stool samples	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	3 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of participants with a complete response or a partial response	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	180 days
Percentage of participants who reduce or discontinue steroids at the end of the study	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	1 year

Collaborators and Investigators

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

Publications and helpful links

Helpful Links

• St. Jude Children's Research Hospital
• Clinical Trials Open at St. Jude

Study record dates

Study Major Dates

• Study Start (Estimated): April 1, 2024
• Primary Completion (Estimated): December 31, 2024
• Study Completion (Estimated): December 31, 2027

Study Registration Dates

• First Submitted: December 15, 2022
• First Submitted That Met QC Criteria: December 15, 2022
• First Posted (Actual): December 23, 2022

Study Record Updates

• Last Update Posted (Actual): March 21, 2024
• Last Update Submitted That Met QC Criteria: March 19, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Gastrointestinal Diseases; Digestive System Diseases; Intestinal Diseases
• Other Study ID Numbers: NEWGUT; NCI-2024-02553 (Other Identifier: NCI Clinical Trial Registration Program)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD 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.
• IPD Sharing Time Frame: Data will be made available at the time of article publication.
• IPD Sharing 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.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No