search
Back to results

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

Primary Purpose

Gastro-Intestinal Disorder

Status
Not yet recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Fecal microbiota transplant (FMT)
Sponsored by
St. Jude Children's Research Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Gastro-Intestinal Disorder

Eligibility Criteria

undefined - 22 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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: 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 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 Current or prolonged GI dysfunction following HCT, defined as having diarrhea or loose stools >/= 4 weeks with at least one of the following: Requiring NG or G-tube feeds Requiring TPN or IVF for more than 4 weeks 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.

Sites / Locations

  • St. Jude Children's Research Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Stratum A

Stratum B

Arm Description

Diagnosed with GvHD

GI Dysfunction

Outcomes

Primary Outcome Measures

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
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
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
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
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
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

Secondary Outcome Measures

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
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

Full Information

First Posted
December 15, 2022
Last Updated
October 17, 2023
Sponsor
St. Jude Children's Research Hospital
search

1. Study Identification

Unique Protocol Identification Number
NCT05664113
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).
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Not yet recruiting
Study Start Date
November 2023 (Anticipated)
Primary Completion Date
December 31, 2024 (Anticipated)
Study Completion Date
December 31, 2027 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
St. Jude Children's Research Hospital

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Gastro-Intestinal Disorder

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
20 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Stratum A
Arm Type
Experimental
Arm Description
Diagnosed with GvHD
Arm Title
Stratum B
Arm Type
Experimental
Arm Description
GI Dysfunction
Intervention Type
Drug
Intervention Name(s)
Fecal microbiota transplant (FMT)
Other Intervention Name(s)
FMT
Intervention Description
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
Primary Outcome Measure Information:
Title
Proportion of participants with a serious adverse event occurring within 30 days following FMT
Description
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
Time Frame
30 Days
Title
Proportion of participants with a non-serious adverse event occurring within 30 days following FMT
Description
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
Time Frame
30 Days
Title
Proportion of patients expressing interest who meet eligibility
Description
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
Time Frame
2 years
Title
Proportion of patients recruited in the eligible population
Description
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
Time Frame
2 years
Title
Proportion of participants that drop up post-enrollment
Description
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
Time Frame
3 years
Title
Proportion of participants providing all protocol required stool samples
Description
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
Time Frame
3 years
Secondary Outcome Measure Information:
Title
Proportion of participants with a complete response or a partial response
Description
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
Time Frame
180 days
Title
Percentage of participants who reduce or discontinue steroids at the end of the study
Description
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
Time Frame
1 year

10. Eligibility

Sex
All
Maximum Age & Unit of Time
22 Years
Accepts Healthy Volunteers
No
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: 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 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 Current or prolonged GI dysfunction following HCT, defined as having diarrhea or loose stools >/= 4 weeks with at least one of the following: Requiring NG or G-tube feeds Requiring TPN or IVF for more than 4 weeks 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Gabriela Maron, MD
Phone
866-278-5833
Email
referrainfo@stjude.org
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Gabriela Maron, MD
Organizational Affiliation
St. Jude Children's Research Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
St. Jude Children's Research Hospital
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38105
Country
United States
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Gabriela Maron, MD
Phone
866-278-5833
Email
referralinfo@stjude.org

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing 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.
Links:
URL
http://www.stjude.org
Description
St. Jude Children's Research Hospital
URL
http://www.stjude.org/protocols
Description
Clinical Trials Open at St. Jude

Learn more about this trial

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

We'll reach out to this number within 24 hrs