Prebiotic Galacto-oligosaccharide and Acute GVHD

NCT04373057 | Recruiting Phase 1 FDA Drug

• 2 other identifiers: STUDY00161331R01HL151365
• Study Type: interventional
• Target: 128
• Locations: 1 country
• Sites: 2

Brief Summary

The purpose of this study is to determine whether the carbohydrate prebiotic (dietary supplement) known as galacto-oligosaccharide (GOS) can modulate the microbiome (the bacteria in the gut) and help prevent graft-versus host disease (GVHD) after allogeneic stem cell transplant. The study has two two parts. In phase 1, the best dose of GOS will be evaluated. In phase 2, using the best dose of GOS, participants will be randomized to receive GOS or a placebo (maltodextrin, a common food additive that is not known to affect the microbiome) so that the effect of GOS can be determined.

Conditions

Acute GVHD

Outcome Measures

Primary Outcomes (2)

• provisional Maximum Tolerated Dose (pMTD) of GOS

  GOS will be dosed as outlined using a modified 3+3 design: 0.75g x 4 days, followed by 1.5g x 4 days, followed by 2.9g for the duration of the study. If two or more subjects experience new grade 2 or 3 pre-HCT toxicities at a given dose level, that dose will be considered not tolerable and the previous dose pMTD. If two or more subjects experience new grade 2 or higher pre-HCT toxicities at the 0.75g/day dose, study may be paused to revisit the design. If one subject experiences new grade 2 or 3 pre-HCT toxicities at a given dose level, additional 3 subjects will be enrolled at this same dose schedule. If one or more of these additional subjects experience new grade 2 or 3 pre-HCT toxicities at that same or lower dose level, this dose level will be considered not tolerable and the previous dose the pMTD. If no subjects experience new grade 2 or 3 pre-HCT toxicities or only one of six subjects experience new grade 2 or 3 pre-HCT toxicities, 2.9g/day dose will be assumed to be the pMTD

  30 days

• Incidence of Grade II-IV acute GVHD at Day 100

  Acute GVHD Scoring will be done following BMT CTN, 2013 criteria.

  100 days

Secondary Outcomes (7)

• Incidence of acute GI toxicities through Day 30

  30 days

• Incidences of Acute GI toxicities as measured by self-reported evaluation through Day 30

  30 days

• Incidence of chronic GVHD at Day 365 and Day 730

  Day 365 and day 730

• Health care utilization through Day 100, through Day 365, and through Day 730

  Day 100, Day 365, and Day 730

• Overall survival (OS) at Day 365 and Day 730

  Day 365 and Day 730

Study Arms (2)

Galacto-oligosaccharide

EXPERIMENTAL

Phase I: Subjects will receive GOS, at dose levels 0.75g, 1.5g, and 2.9 g/day administered once daily. GOS will be dosed per the following schedule using a modified 3+3 design: 0.75g x 4 days, followed by 1.5g x 4 days, followed by 2.9g for the duration of the study starting from about 30 days before transplant to about 4 weeks after transplant. Phase II: Subjects will receive GOS, at dose levels 0.25\*MTD, 0.5\*MTD, and MTD with MTD determined by the phase 1 of the study, once daily from about 30 days before transplant to about 4 weeks after transplant.

Dietary Supplement: Galacto-oligosaccharide

Maltodextrin

PLACEBO COMPARATOR

Phase II: Subjects will receive maltodextrin at comparable dose level as GOS (in Phase II) once daily from about 30 days before transplant to about 4 weeks after transplant.

Dietary Supplement: Maltodextrin

Interventions

Galacto-oligosaccharide DIETARY_SUPPLEMENT

GOS will be administered at determined dose levels per protocol once daily from about 30 days before transplant to about 4 weeks after transplant.

Also known as: Bimuno®, B-GOS

Galacto-oligosaccharide

Maltodextrin DIETARY_SUPPLEMENT

Maltodextrin will be administered at comparable dose level as GOS (in Phase II) once daily from about 30 days before transplant to about 4 weeks after transplant.

Maltodextrin

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Plan to undergo allogeneic HCT for any cancer or non-cancer illness
• Age 18-80 years
• Karnofsky Performance Status \>70

You may not qualify if:

• Pregnant/lactating
• Malabsorption syndrome, short bowel or chologenic diarrhea
• At time of enrollment, Grade 2 or higher GI symptoms per NCI-CTCAE
• Active treatment with other prebiotics, probiotics, or herbal supplementation (ok if stops before enrollment)
• Active treatment with antibiotics (with the exception of prophylactic antibiotics)
• Concurrent enrollment on the Duke HCT Home Transplant study or another clinical trial targeting GVHD; patients who are enrolled in observational or non-pharmacologic intervention trials (for example, the Duke HCT Research-POP Pre and Peri-HCT Optimization Program aka "R-POP") or pharmacologic or cellular therapy trials with other targets (for example, NK DLI) are NOT excluded

Sponsors & Collaborators

• University of Kansas Medical Center: lead
• National Heart, Lung, and Blood Institute (NHLBI): collaborator

Study Sites (2)

Kansas University Medical Center

Kansas City, Kansas, 66160, United States

RECRUITING

Duke

Durham, North Carolina, 27710, United States

ACTIVE NOT RECRUITING

Related Publications (1)

• Lakshmanan AP, Deola S, Terranegra A. The Promise of Precision Nutrition for Modulation of the Gut Microbiota as a Novel Therapeutic Approach to Acute Graft-versus-host Disease. Transplantation. 2023 Dec 1;107(12):2497-2509. doi: 10.1097/TP.0000000000004629. Epub 2023 May 16.

  PMID: 37189240 DERIVED

MeSH Terms

Interventions

4'-galactooligosaccharide; maltodextrin

Study Officials

• Anthony Sung, MD

  University of Kansas

  PRINCIPAL INVESTIGATOR

• Mitchell Horwitz, MD

  Duke Health

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Beth Turk, MPH, CCRC

CONTACT

(913) 574-0154; bturk2@kumc.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Randomized, double blind study
• Purpose: PREVENTION
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 29, 2020
• First Posted: May 4, 2020
• Study Start: January 22, 2021
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): February 1, 2029
• Last Updated: April 22, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)