What side effects are associated with this type of intervention?

Common treatments for bone marrow transplant include high-dose chemotherapy and radiation, which can cause side effects such as nausea, vomiting, diarrhea, mucositis, and increased risk of infections due to immunosuppression. Treatments similar to 2'-Fucosyllactose (2FL), such as other prebiotics and probiotics, are generally well-tolerated but can cause mild gastrointestinal symptoms like bloating, gas, and diarrhea. These treatments aim to promote the growth of beneficial gut bacteria and improve intestinal homeostasis, potentially reducing inflammation and the risk of graft-versus-host disease (GVHD).

What prior FDA approvals exist?

The FDA has approved several treatments for bone marrow transplant patients, primarily focusing on managing complications and improving outcomes. While specific prebiotics like 2'-Fucosyllactose (2FL) are still under investigation, probiotics such as Lactobacillus and Bifidobacterium species have been studied for their potential benefits in maintaining gut health and reducing infections. These probiotics aim to restore the balance of gut microbiota disrupted by high-dose chemotherapy and radiation, similar to the hypothesized benefits of 2FL. However, comprehensive FDA approvals specifically targeting gut microbiome modulation in bone marrow transplant patients are still emerging.

What other types of research exist?

Promising alternatives to 2'-Fucosyllactose (2FL) for bone marrow transplant patients include lactulose, which has been shown to increase fecal bifidobacterial counts, and synbiotics such as combinations of Bifidobacterium breve with galacto-oligosaccharides (GOS) and fructooligosaccharides (FOS). These alternatives have demonstrated efficacy in promoting the growth of beneficial gut bacteria and improving intestinal homeostasis.

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Oligosaccharide

Oral 2'-Fucosyllactose Supplementation for Bone Marrow Transplant Recipients

Phase 1 & 2

Recruiting

Led By Pooja Khandelwal, MD

Research Sponsored by Children's Hospital Medical Center, Cincinnati

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be scheduled for allogeneic stem cell transplant

Must not have

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, day+7, day+14, day+30 after transplant

Awards & highlights

No Placebo-Only Group

Summary

This trial tests a sugar supplement called 2'-fucosyllactose (2FL) to help children and young adults who have had a stem cell transplant. These patients often suffer from gut problems due to their treatment. The supplement is expected to increase good gut bacteria, reduce bad bacteria, and lower inflammation, leading to better overall health outcomes. 2'-Fucosyllactose (2'-FL) is a natural prebiotic found in human milk and is known for promoting beneficial gut bacteria and improving immune function.

Who is the study for?

This trial is for patients of any age and diagnosis who are scheduled for an allogeneic stem cell transplant. It's not suitable for those unable to take oral supplements, actively breastfeeding infants, anyone with a recent GI infection or taking anti-diarrheal meds, and people who've used probiotics/prebiotics recently or have had significant gut damage in the past 3 months.

What is being tested?

The trial tests if adding a supplement called 2'-fucosyllactose (2FL) can help balance gut bacteria and reduce inflammation after bone marrow transplants. The goal is to see if it makes the intestines healthier by day+30 post-transplant and lowers complications like acute GVHD and bloodstream infections by day+100.

What are the potential side effects?

While specific side effects aren't listed, since this is a dietary supplement being tested in a vulnerable population, potential side effects could include digestive discomforts such as bloating, gas, nausea or changes in bowel habits.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am scheduled for a stem cell transplant from a donor.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have not had severe gut damage or infections in the last 3 months.

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I have a history of inflammatory bowel disease, short bowel syndrome, or bowel surgery.

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I had a stomach or intestinal infection in the last week.

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I cannot eat or receive nutrition by mouth or tube.

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I have taken probiotics or prebiotics in the last month.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, day+7, day+14, day+30 after transplant

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, day+7, day+14, day+30 after transplant

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, day+7, day+14, day+30 after transplant for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change in fecal butyrate/acetate/propionate levels

Number of bloodstream infections

Number of patients able to take 2FL

Secondary study objectives

Incidence of acute GVHD

Incidence of bloodstream infections

Incidence of graft versus host disease (GVHD)

+4 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

3Treatment groups

Experimental Treatment

Group I: 2'-fucosyllactose for ages >10 yearsExperimental Treatment1 Intervention

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

Group II: 2'-fucosyllactose for ages 5.1-10 yearsExperimental Treatment1 Intervention

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

Group III: 2'-fucosyllactose for ages 0-5 yearsExperimental Treatment1 Intervention

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

0 / 6Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

2'-Fucosyllactose (2FL) is a prebiotic that promotes the growth of beneficial gut bacteria such as Bifidobacteria, which play a crucial role in maintaining intestinal homeostasis. By enhancing the population of these beneficial bacteria, 2FL helps to stabilize the gut microbiome, reduce inflammation, and improve the integrity of the intestinal barrier. This is particularly important for Bone Marrow Transplant (BMT) patients, as the high-dose chemotherapy and radiation used in their preparative regimens can severely disrupt intestinal homeostasis, leading to complications such as graft-versus-host disease (GVHD) and bloodstream infections (BSI). By supporting a healthy gut microbiome, 2FL can potentially reduce these risks and improve overall outcomes for BMT patients.

Human Milk Oligosaccharides Modulate Fecal Microbiota and Are Safe for Use in Children With Overweight: A Randomized Controlled Trial.Combinational effects of prebiotic oligosaccharides on bifidobacterial growth and host gene expression in a simplified mixed culture model and neonatal mice.