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

Gastro-Intestinal Disorder Clinical Trial

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT05664113
• Other study ID #: NEWGUT
• Secondary ID: NCI-2024-02553
• Status: Not yet recruiting
• Phase: Phase 1
• Start date: April 2024
• Est. completion date: December 31, 2027

Study information

• Verified date: March 2024
• Source: St. Jude Children's Research Hospital
• Contact: Gabriela Maron, MD
• Phone: 866-278-5833
• Email: referrainfo[@]stjude.org
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 20
• Est. completion date: December 31, 2027
• Est. primary completion date: December 31, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 22 Years

Eligibility

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.

Related Conditions & MeSH terms

• Digestive System Diseases
• Gastro-Intestinal Disorder
• Gastrointestinal Diseases
• Intestinal Diseases

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

Locations

Country	Name	City	State
United States	St. Jude Children's Research Hospital	Memphis	Tennessee

Sponsors (1)

Lead Sponsor	Collaborator
St. Jude Children's Research Hospital

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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	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
Secondary	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

External Links

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