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

NCT04263597 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: 2020-0008
• Study Type: interventional
• Target: 70
• Locations: 1 country
• Sites: 1

Brief 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. Phase II: The investigators hypothesize that 2FL supplementation will be safe and tolerable and result in an increase in the relative abundance of fecal short chain fatty acids such as butyrate, acetate and propionate at day+7 compared to baseline values.

Conditions

Hematopoietic Stem Cell Transplant

Outcome Measures

Primary Outcomes (3)

• Number of bloodstream infections

  Number of bloodstream infections in patients on 2FL

  Day+100 after transplant

• Number of patients able to take 2FL

  6 of 10 patients receiving 2FL able to take 80% of their planned doses

  1 week prior to start of chemotherapy until day+30 after transplant

• Change in fecal butyrate/acetate/propionate levels

  Change in fecal butyrate/acetate/propionate levels from baseline at day+ 7 in patients enrolled in phase II of the study

  Day+ 7 after transplant

Secondary Outcomes (7)

• Relative abundance of fecal Bifidobacteria at day+30 compared to baseline by >/=10%

  Day+30 after transplant

• Relative abundance of fecal Firmicutes and Proteobacteria at day+30 compared to baseline for patients on 2FL

  Day+30 after transplant

• Incidence of acute GVHD

  Day+100 after transplant

• Incidence of bloodstream infections

  Day+100 after transplant

• Incidence of mucosal barrier injury laboratory-confirmed bloodstream infection (MBI-LCBI)

  Day+ 100 after transplant

Study Arms (3)

2'-fucosyllactose for ages 0-5 years

EXPERIMENTAL

Dose for ages 0-5 years: 2.5 g/day;

Drug: 2'-fucosyllactose

2'-fucosyllactose for ages 5.1-10 years

EXPERIMENTAL

Dose for ages 5.1-10 years: 5 g/day;

Drug: 2'-fucosyllactose

2'-fucosyllactose for ages >10 years

EXPERIMENTAL

Dose for ages \>10 years: 10 g/day;

Drug: 2'-fucosyllactose

Interventions

2'-fucosyllactose DRUG

2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.

Also known as: 2FL supplementation

2'-fucosyllactose for ages 0-5 years; 2'-fucosyllactose for ages 5.1-10 years; 2'-fucosyllactose for ages >10 years

Eligibility Criteria

• Age: 0 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Be scheduled for allogeneic stem cell transplant
• All ages and underlying diagnoses, preparative regimens, stem cell sources and acute GVHD prophylaxes

You may not qualify if:

• 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

Sponsors & Collaborators

• Children's Hospital Medical Center, Cincinnati: lead

Study Sites (1)

Cincinnati Children's Hospital

Cincinnati, Ohio, 45229, United States

Location

MeSH Terms

Interventions

2'-fucosyllactose

Study Officials

• Pooja Khandelwal, MD

  Children's Hospital Medical Center, Cincinnati

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Eligible patients will be allocated into the following arms as determined by age at enrollment. Arm 1: 0-5 years; Arm 2: 5.1-10 years; Arm 3: \>10 years

Study Record Dates

• First Submitted: February 7, 2020
• First Posted: February 11, 2020
• Study Start: August 26, 2020
• Primary Completion: May 23, 2025
• Study Completion: May 23, 2025
• Last Updated: November 6, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)