Oral Supplementation of 2'-Fucosyllactose in Allogeneic Bone Marrow Transplant Recipients

  • STATUS
    Recruiting
  • End date
    Sep 25, 2025
  • participants needed
    285
  • sponsor
    Children's Hospital Medical Center, Cincinnati
Updated on 25 January 2021
graft versus host disease
bone marrow transplant
acute graft-versus-host disease
allogeneic hematopoietic stem cell transplant

Summary

High dose chemotherapy and radiation used as preparative regimens in patients undergoing an allogeneic hematopoietic stem cell transplant (HSCT) disrupts intestinal homeostasis by damaging the intestinal epithelium and altering the intestinal microbiome. The investigators hypothesize that 2'-fucosyllactose (2FL) supplementation will be safe and tolerable and result in an increase in the relative abundance of intestinal Bifidobacteria. The investigators also hypothesize that 2FL supplementation will lead to reduction of Firmicutes and/or Proteobacteria, and improved intestinal homeostasis at day+30 as measured by lower pro-inflammatory cytokines, reduced levels of T-cell activation, lower markers of intestinal injury (fecal human DNA and plasma reg-3-alpha), increased fecal butyrate levels and ultimately lower incidence of acute GVHD and BSI at day+100.

Description

This phase I/IIa study is a single center prospective study at Cincinnati Children's Hospital Medical Center (CCHMC).

This study will assess the safety and tolerability of various doses of 2FL. Eligible patients will be allocated to the following arms as determined by age at enrollment:

Arm 1: 0-5 years; Arm 2: 5.1-10 years; Arm 3: >10 years

The investigators will enroll 5 patients of ages 10 years undergoing allogeneic HSCT. 2'-FL will be administered to these patients from day-7 until day+30 after HSCT at the starting dose. Once safety is determined the investigators will then enroll an additional 5 patients of ages 5-10 years and administer 2'FL to these patients at the proposed dose for this age group from day-7 to day+30 after HSCT. Once safety is determined, the investigators will enroll 5 patients of ages 0-5 years and administer 2'FL at starting doses to children from day-7 to day+30 after HSCT. Once safety is established in these patients we will proceed with the randomized portion of the study.

Patients will be enrolled in each arm and randomized 1:1 to either receive 2FL or placebo (2 g oral glucose). After 20 patients are enrolled at the first dose level for an arm, enrollment of that arm will be paused and all patients will be evaluated for safety and tolerability. The randomization for the 20 patients will be unblinded and the safety and tolerability of 2FL doses will be compared to placebo for that arm. If 6/10 patients can take 80% of their planned doses, that dose level will be determined as tolerable and a dose escalation will be performed. If 5/10 patients are unable to take at least 80% of planned doses the dose will be determined to not be well tolerated and a dose de-escalation will be done.

Details
Condition Stem Cell Transplantation, Hematopoietic Stem Cell Transplantation, HSCT, Hematopoietic Stem Cell Transplant, Stem Cell Transplant
Treatment 2'-fucosyllactose, Placebo (2g oral glucose)
Clinical Study IdentifierNCT04263597
SponsorChildren's Hospital Medical Center, Cincinnati
Last Modified on25 January 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Gender: Male or Female
Do you have Hematopoietic Stem Cell Transplant?
Do you have any of these conditions: HSCT or Stem Cell Transplantation or Hematopoietic Stem Cell Transplantation or Stem Cell Transplant or Hematopoietic Stem Cell Transplant?
Be scheduled for allogeneic stem cell transplant
All ages and underlying diagnoses, preparative regimens, stem cell sources and acute GVHD prophylaxes

Exclusion Criteria

Unable to take anything orally or enterally (i.e. intestinal failure)
Actively breastfeeding infants
Recent (within the week prior to enrollment) GI infection
Patients receiving anti-diarrheal medications such as loperamide
Patients who have received probiotics or prebiotics during the previous month
Patients who have had any type of gut damage within the past 3 months such as previous bowel perforations, previous episode of Grade 4 neutropenic colitis or typhlitis
Patients with inflammatory bowel disease, short bowel syndrome, and patients with a history of bowel resections
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