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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04263597
Recruitment Status : Recruiting
First Posted : February 11, 2020
Last Update Posted : June 9, 2022
Sponsor:
Information provided by (Responsible Party):
Children's Hospital Medical Center, Cincinnati

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.

Condition or disease Intervention/treatment Phase
Hematopoietic Stem Cell Transplant Drug: 2'-fucosyllactose Phase 1 Phase 2

Detailed 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 first 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 for the ≥10 years age group. Once safety is determined the investigators will then enroll an additional 5 patients of ages 5-10 years and 5 patients of ages 0-5 years and administer 2'FL at starting doses according to their age group to children from day-7 to day+30 after HSCT. Enrollment in the 2 defined age groups (5-10 years and 0-5 years) will occur independent of each other/in parallel to establish safety. Once safety is established in these patients the investigators will proceed with the 3x3 study design dose finding portion of our study

Three patients will be enrolled in each arm at the starting dose level. Investigators will perform a dose escalation or de-escalation based on rates of dose limiting toxicities.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 70 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:

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

Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Oral Supplementation of 2'-Fucosyllactose in Allogeneic Bone Marrow Transplant Recipients to Maintain Intestinal Homeostasis
Actual Study Start Date : August 26, 2020
Estimated Primary Completion Date : September 2025
Estimated Study Completion Date : September 2025

Arm Intervention/treatment
Experimental: 2'-fucosyllactose for ages 0-5 years

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

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

Dose de-escalation for ages 0-5 years: 1.25 g/day

Drug: 2'-fucosyllactose
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.
Other Name: 2FL supplementation

Experimental: 2'-fucosyllactose for ages 5.1-10 years

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

Dose escalation for ages 5.1-10 years: 7.5 g/day;

Dose de-escalation for ages 5.1-10 years: 2.5 g/day

Drug: 2'-fucosyllactose
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.
Other Name: 2FL supplementation

Experimental: 2'-fucosyllactose for ages >10 years

Starting dose for ages >10 years: 10 g/day;

Dose escalation for ages >10 years: 15 g/day;

Dose de-escalation for ages >10 years: 5 g/day

Drug: 2'-fucosyllactose
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.
Other Name: 2FL supplementation




Primary Outcome Measures :
  1. Number of bloodstream infections [ Time Frame: Day+100 after transplant ]
    Number of bloodstream infections in patients on 2FL

  2. Number of patients able to take 2FL [ Time Frame: 1 week prior to start of chemotherapy until day+30 after transplant ]
    6 of 10 patients receiving 2FL able to take 80% of their planned doses


Secondary Outcome Measures :
  1. Relative abundance of fecal Bifidobacteria at day+30 compared to baseline by >/=10% [ Time Frame: Day+30 after transplant ]
    Change in relative abundance of fecal Bifidobacteria at day+30 compared to baseline by >/=10%

  2. Relative abundance of fecal Firmicutes and Proteobacteria at day+30 compared to baseline for patients on 2FL [ Time Frame: Day+30 after transplant ]
    Change in relative abundance of fecal Firmicutes and Proteobacteria at day+30 compared to baseline for patients on 2FL

  3. Incidence of acute GVHD [ Time Frame: Day+100 after transplant ]
    Incidence of acute GVHD in patients on 2FL

  4. Incidence of bloodstream infections [ Time Frame: Day+100 after transplant ]
    Incidence of bloodstream infections in patients on 2FL



Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04263597


Contacts
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Contact: Celeste Dourson (513) 636-7679 Celeste.Dourson@cchmc.org

Locations
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United States, Ohio
Cincinnati Children's Hospital Recruiting
Cincinnati, Ohio, United States, 45229
Contact: Celeste Dourson       Celeste.Dourson@cchmc.org   
Sponsors and Collaborators
Children's Hospital Medical Center, Cincinnati
Investigators
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Principal Investigator: Pooja Khandelwal, MD Children's Hospital Medical Center, Cincinnati
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Responsible Party: Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier: NCT04263597    
Other Study ID Numbers: 2020-0008
First Posted: February 11, 2020    Key Record Dates
Last Update Posted: June 9, 2022
Last Verified: June 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No