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

Study Overview
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Brief Summary
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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
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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

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

Conditions
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Gastro-Intestinal Disorder

Intervention / Treatment
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* Drug: Fecal microbiota transplant (FMT)


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Maximum Age: 22 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

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


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Stratum A<br>Diagnosed with GvHD | Drug: Fecal microbiota transplant (FMT)<br>* 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<br>* Other names: FMT;|
| Experimental: Stratum B<br>GI Dysfunction | Drug: Fecal microbiota transplant (FMT)<br>* 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<br>* Other names: FMT;|

What is the study measuring?
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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 |