Acute GVHDPhase 1/2NCT04373057
Prebiotic Galacto-oligosaccharide and Acute GVHD
A Phase 1/2 study testing Galacto-oligosaccharide and Maltodextrin in people with Acute GVHD, run by University of Kansas Medical Center at 2 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Acute GVHD
- What is being tested?
- Galacto-oligosaccharide and Maltodextrin
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 128 participants
- Where
- 2 sitesUnited States
- Study dates
- 22 Jan 2021to 1 Feb 2029
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Plan to undergo allogeneic HCT for any cancer or non-cancer illness
- Age 18-80 years
- Karnofsky Performance Status >70
Who cannot join
- 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
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States2 sites, 1 recruiting
- Active, not recruiting
Duke
Durham, North Carolina, 27710
- Recruiting
Kansas University Medical Center
Kansas City, Kansas, 66160
Anthony Sung, MD
About this study
Official title: A Phase I/II Trial of the Prebiotic Galacto-oligosaccharide to Prevent Acute GVHD
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.
Read the detailed description
Humans carry on the order of 100 trillion microbial cells (the microbiota),which play a major role in normal health as well as disease pathogenesis. In the gut, the microbiota interact with the intestinal epithelium and immune system to regulate inflammation and metabolism. The impact of the microbiota on GVHD and other clinical outcomes may be mediated by bacterial metabolites such as short chain fatty acid (SCFAs). In particular, the SCFA butyrate has been shown to be a preferred fuel source of human colonic epithelial cells and essential for normal differentiation of regulatory immune cells in the intestine, which in turn may decrease GVHD. This is supported by murine studies, in which manipulating the gut microbiota to increase microbial SCFA production or by direct administration (oral gavage) of butyrate has been shown to improve HCT outcomes, including protection from GVHD
Manipulating the gut microbiota in HCT to decrease the risk of GVHD could potentially be done using several methods, namely prebiotics (dietary carbohydrates or fibers), probiotics (live bacteria), and fecal transplantation. In contrast to live bacteria, prebiotics are dietary carbohydrates classified as nutritional supplements that can sustain gut bacteria, regulate gastrointestinal transit time, and foster cooperative metabolic networks between enteric microbes. Because prebiotics encourage the growth of existing bacteria rather than introducing new organisms, they may be safer. Moreover, bacterial fermentation of prebiotics may yield SCFA.
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, three dose levels 0.75g/day (25% maximum dose, D1), 1.5g/day (50% maximum dose D2), and 2.9g/day (maximum dose, D3) will be evaluated to find out the provisional maximum tolerated dose (pMTD) of GOS to be used in phase 2. In phase 2, using pMTD 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. Note that if the pMTD is D3, there will be a D1 and D2 lead-in period similar to the Phase 1 study however if the pMTD is D2, there will only be a lead-in with D1 and if pMTD is D1, there will only be one dose with no lead-in. The study will generate important data on gut microbiota responses to help tailor or personalize future prebiotic therapies to patients and their microbiota.
- Conditions
- Acute GVHD
- Being tested
- Galacto-oligosaccharide, Maltodextrin
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Galacto-oligosaccharideDietary supplement
Also called Bimuno®, B-GOS
GOS will be administered at determined dose levels per protocol once daily from about 30 days before transplant to about 4 weeks after transplant.
MaltodextrinDietary 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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Dietary Supplement: Maltodextrin |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 30 days
Incidence of Grade II-IV acute GVHD at Day 100
Acute GVHD Scoring will be done following BMT CTN, 2013 criteria.
Measured: 100 days
Secondary outcomes (7)
Incidence of acute GI toxicities through Day 30
Acute GI toxicities include mucositis, nausea, vomiting, diarrhea, constipation, abdominal pain, and bloating through D30 as assessed by CTCAE v5.
Measured: 30 days
Incidences of Acute GI toxicities as measured by self-reported evaluation through Day 30
Acute GI toxicities as measured by self-reported (PRO-CTCAE) evaluation of decreased appetite, anxiety, sadness, fatigue, insomnia, general pain, shortness of breath, numbness and tingling, mouth sores, nausea, vomiting, diarrhea, constipation, abdominal pain, and bloating through D 30 (5 point ordinal scale per PRO-CTCAE)
Measured: 30 days
Incidence of chronic GVHD at Day 365 and Day 730
Chronic GVHD Scoring will be done following NIH Consensus Criteria
Measured: Day 365 and day 730
Health care utilization through Day 100, through Day 365, and through Day 730
Data will be obtained through chart review at or after Day 100, Day 365, and Day730 (respectively), and will be reported as means/medians depending on the distribution of data (for example, mean/median health care utilization through Day 100)
Measured: Day 100, Day 365, and Day 730
Overall survival (OS) at Day 365 and Day 730
Data will be obtained by chart review at or after D365 and D730 Data will be reported as a percentage (ie. 1-year and 2-year overall survival) for each arm
Measured: Day 365 and Day 730
Relapse-free survival (RFS) at Day 365 and Day 730
Data will be obtained by chart review at or after Day 365 and Day 730 Data will be reported as a percentage (ie. 1-year and 2-year overall survival) for each arm
Measured: Day 365 and Day 730
Total parenteral nutrition (TPN) use through Day 30 and Day 100
Data will be obtained as part of the GVHD Prophylaxis/medication assessments. Data will be reported as a percentage for each arm (at each of the time points)
Measured: Day 30 and Day 100
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 May 2020
- Study start22 Jan 2021
- Primary completion1 Jun 2027 (estimated)
- Study completion1 Feb 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Acute GVHDStudies of the same condition, open and under way.
- More trials run by University of Kansas Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 128 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Kansas Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Sponsor
- Investigators
- Anthony Sung, MD, Principal investigator, University of KansasMitchell Horwitz, MD, Principal investigator, Duke Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04373057
- Sponsor's protocol ID
- STUDY00161331
- Other IDs
- R01HL151365
- Status last verified
- Apr 2026
- First submitted
- 29 Apr 2020
- Last updated
- 22 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 37189240
Study at a glance
From the registry record, last updated by the sponsor on 22 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Trial of the Prebiotic Galacto-oligosaccharide to Prevent Acute GVHD
- ClinicalTrials.gov ID
- NCT04373057
- Acronym / sponsor ID
- STUDY00161331
- Conditions
- Acute GVHD
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 128 participants (estimated)
- Lead sponsor
- University of Kansas Medical CenterUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- 22 Jan 2021 (actual)
- Primary completion
- 1 Jun 2027 (estimated)
- Study completion
- 1 Feb 2029 (estimated)
- First posted
- 4 May 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Humans carry on the order of 100 trillion microbial cells (the microbiota),which play a major role in normal health as well as disease pathogenesis. In the gut, the microbiota interact with the intestinal epithelium and immune system to regulate inflammation and metabolism. The impact of the microbiota on GVHD and other clinical outcomes may be mediated by bacterial metabolites such as short chain fatty acid (SCFAs). In particular, the SCFA butyrate has been shown to be a preferred fuel source of human colonic epithelial cells and essential for normal differentiation of regulatory immune cells in the intestine, which in turn may decrease GVHD. This is supported by murine studies, in which manipulating the gut microbiota to increase microbial SCFA production or by direct administration (oral gavage) of butyrate has been shown to improve HCT outcomes, including protection from GVHD
Manipulating the gut microbiota in HCT to decrease the risk of GVHD could potentially be done using several methods, namely prebiotics (dietary carbohydrates or fibers), probiotics (live bacteria), and fecal transplantation. In contrast to live bacteria, prebiotics are dietary carbohydrates classified as nutritional supplements that can sustain gut bacteria, regulate gastrointestinal transit time, and foster cooperative metabolic networks between enteric microbes. Because prebiotics encourage the growth of existing bacteria rather than introducing new organisms, they may be safer. Moreover, bacterial fermentation of prebiotics may yield SCFA.
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, three dose levels 0.75g/day (25% maximum dose, D1), 1.5g/day (50% maximum dose D2), and 2.9g/day (maximum dose, D3) will be evaluated to find out the provisional maximum tolerated dose (pMTD) of GOS to be used in phase 2. In phase 2, using pMTD 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. Note that if the pMTD is D3, there will be a D1 and D2 lead-in period similar to the Phase 1 study however if the pMTD is D2, there will only be a lead-in with D1 and if pMTD is D1, there will only be one dose with no lead-in. The study will generate important data on gut microbiota responses to help tailor or personalize future prebiotic therapies to patients and their microbiota.
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Galacto-oligosaccharide | Experimental | Dietary Supplement: Galacto-oligosaccharidePhase 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. |
| Maltodextrin | Placebo comparator | Dietary Supplement: MaltodextrinPhase 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. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Plan to undergo allogeneic HCT for any cancer or non-cancer illness
- Age 18-80 years
- Karnofsky Performance Status >70
Key exclusion criteria (6 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Galacto-oligosaccharideAlso: Bimuno®, B-GOSGOS will be administered at determined dose levels per protocol once daily from about 30 days before transplant to about 4 weeks after transplant. | Dietary supplement | Galacto-oligosaccharide |
| MaltodextrinMaltodextrin 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. | Dietary supplement | Maltodextrin |
Primary outcome
provisional Maximum Tolerated Dose (pMTD) of GOS
Time frame: 30 days
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
Incidence of Grade II-IV acute GVHD at Day 100
Time frame: 100 days
Acute GVHD Scoring will be done following BMT CTN, 2013 criteria.
7 secondary outcomes
Incidence of acute GI toxicities through Day 30
Time frame: 30 days
Incidences of Acute GI toxicities as measured by self-reported evaluation through Day 30
Time frame: 30 days
Incidence of chronic GVHD at Day 365 and Day 730
Time frame: Day 365 and day 730
Health care utilization through Day 100, through Day 365, and through Day 730
Time frame: Day 100, Day 365, and Day 730
Overall survival (OS) at Day 365 and Day 730
Time frame: Day 365 and Day 730
Relapse-free survival (RFS) at Day 365 and Day 730
Time frame: Day 365 and Day 730
Total parenteral nutrition (TPN) use through Day 30 and Day 100
Time frame: Day 30 and Day 100
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Apr 2026.
- Derived from this study: PubMed 37189240Lakshmanan 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Anthony Sung, MD principal investigator
University of Kansas
Mitchell Horwitz, MD principal investigator
Duke Health
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Galacto-oligosaccharide and Maltodextrin in people with Acute GVHD, run by University of Kansas Medical Center at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 22 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Beth Turk, MPH, CCRC ((913) 574-0154, bturk2@kumc.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
provisional Maximum Tolerated Dose (pMTD) of GOS (time frame: 30 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04373057, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Apr 2026.