NCT04263597

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

Completed Phase 1, PHASE2 Last updated 6 November 2025

What this trial tests

Phase 1, PHASE2 trial testing 2'-fucosyllactose in Hematopoietic Stem Cell Transplant in 70 participants. Completed in 23 May 2025.

Timeline

26 August 2020

Primary endpoint
23 May 2025

23 May 2025

Quick facts

Lead sponsor	Children's Hospital Medical Center, Cincinnati
Phase	Phase 1, PHASE2
Status	Completed
Study type	INTERVENTIONAL
Allocation	non randomized
Design	parallel
Masking	none
Primary purpose	prevention
Enrollment	70
Start date	26 August 2020
Primary completion	23 May 2025
Estimated completion	23 May 2025
Sites	1 location across United States

Drugs / interventions tested

• 2'-fucosyllactose — full drug profile →

Conditions studied

• Hematopoietic Stem Cell Transplant — all drugs for Hematopoietic Stem Cell Transplant →

Sponsor

Children's Hospital Medical Center, Cincinnati

Who can join

0 and older, any sex, with Hematopoietic Stem Cell Transplant. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

6 peer-reviewed publications reference this trial (live from Europe PMC):

1. Roles of the intestinal microbiota and microbial metabolites in acute GVHD.
   Lin D, Hu B, Li P, Zhao Y, et al · · 2021 · cited 48× · PMID 34706782 · DOI 10.1186/s40164-021-00240-3
2. Nutritional modulation of the gut microbiome in allogeneic hematopoietic stem cell transplantation recipients.
   Muratore E, Leardini D, Baccelli F, Venturelli F, et al · · 2022 · cited 27× · PMID 36337625 · DOI 10.3389/fnut.2022.993668
3. Gut microbiota, microbiota-derived metabolites, and graft-versus-host disease.
   Yue X, Zhou H, Wang S, Chen X, et al · · 2024 · cited 26× · PMID 38239049 · DOI 10.1002/cam4.6799
4. The role of the intestinal microbiota in allogeneic HCT: clinical associations and preclinical mechanisms.
   Nguyen CL, Docampo MD, van den Brink MR, Markey KA. · · 2021 · cited 14× · PMID 33388483 · DOI 10.1016/j.gde.2020.11.007
5. Update in clinical and mouse microbiota research in allogeneic haematopoietic cell transplantation.
   Lindner S, Peled JU. · · 2020 · cited 5× · PMID 33003084 · DOI 10.1097/moh.0000000000000616
6. Personalizing Nutritional Therapy in Pediatric Oncology: The Role of Gut Microbiome Profiling and Metabolomics in Mitigating Mucositis and Enhancing Immune Response to Chemotherapy
   Pawłowski P, Zaj N, Iwaniszczuk K, Grzelka I, et al · · 2026