Clinical trial NCT05664113: 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
Overall status: RECRUITING
Phase: PHASE1
Conditions: Gastro-Intestinal Disorder
Lead sponsor: St. Jude Children's Research Hospital
Sponsor class: OTHER
First posted: 2022-12-23
Enrollment: 10
Sex: ALL
Maximum age (years): 22
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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

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.

Primary outcomes:
measure: Proportion of participants with a serious adverse event occurring within 30 days following FMT
timeFrame: 30 Days
measure: Proportion of participants with a non-serious adverse event occurring within 30 days following FMT
timeFrame: 30 Days
measure: Proportion of patients expressing interest who meet eligibility
timeFrame: 2 years
measure: Proportion of patients recruited in the eligible population
timeFrame: 2 years
measure: Proportion of participants that drop up post-enrollment
timeFrame: 3 years
measure: Proportion of participants providing all protocol required stool samples
timeFrame: 3 years
Secondary outcomes:
measure: Proportion of participants with a complete response or a partial response
timeFrame: 180 days
measure: Percentage of participants who reduce or discontinue steroids at the end of the study
timeFrame: 1 year
Other outcomes:
Locations:
city: Memphis
country: United States
facility: St. Jude Children's Research Hospital
state: Tennessee
Study officials:
affiliation: St. Jude Children's Research Hospital
name: Gabriela Maron, MD
role: PRINCIPAL_INVESTIGATOR
References: