Hematopoietic Stem Cell TransplantPhase 1/2NCT04263597
Oral Supplementation of 2'-Fucosyllactose in Allogeneic Bone Marrow Transplant Recipients
A Phase 1/2 study testing 2'-fucosyllactose in people with Hematopoietic Stem Cell Transplant, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 0 years and older
- Conditions the study is for
- Hematopoietic Stem Cell Transplant
- What is being tested?
- 2'-fucosyllactose
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 70 participants
- Where
- 1 siteUnited States
- Study dates
- 26 Aug 2020to 23 May 2025
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 0 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Be scheduled for allogeneic stem cell transplant
- All ages and underlying diagnoses, preparative regimens, stem cell sources and acute GVHD prophylaxes
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229
About this study
Official title: Oral Supplementation of 2'-Fucosyllactose in Allogeneic Bone Marrow Transplant Recipients to Maintain Intestinal Homeostasis
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.
Read the 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.
Phase II:
Initial 15 patients to establish safety as per the FDA have been enrolled. An additional 10 patients were enrolled and interim analyses demonstrating safety, lack of any dose limiting toxicities and a positive signal of increase in fecal acetate and propionate at day+7 compared to baseline values) were performed. The investigators will enroll approximately 65 additional patients to test efficacy of 2FL supplementation in children and young adult allogeneic HSCT patients with a goal to reduce intestinal inflammation and improve post HSCT outcomes such as acute GVHD and bloodstream infections.
- Conditions
- Hematopoietic Stem Cell Transplant
- Being tested
- 2'-fucosyllactose
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
2'-fucosyllactoseDrug
Also called 2FL supplementation
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.
| Study group | Role | What they receive |
|---|---|---|
| 2'-fucosyllactose for ages 0-5 years | Experimental | Dose for ages 0-5 years: 2.5 g/day; Receives: Drug: 2'-fucosyllactose |
| 2'-fucosyllactose for ages 5.1-10 years | Experimental | Dose for ages 5.1-10 years: 5 g/day; Receives: Drug: 2'-fucosyllactose |
| 2'-fucosyllactose for ages >10 years | Experimental | Dose for ages >10 years: 10 g/day; Receives: Drug: 2'-fucosyllactose |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Bloodstream Infections
Patients with a bloodstream infection occurring anytime between first day of 2-FL dosing until day+100 after transplant were collected. Typically 2FL was started with start of chemotherapy which was usually up to 22 days before transplant
Measured: Day+100 after transplant
Number of Patients Able to Take 2FL
Daily drug doses were documented by patients or nursing staff as applicable. Patients who took at least 60% of their total planned doses were deemed to meet protocol defined adherence to 2-FL
Measured: 1 week prior to start of chemotherapy until day+30 after transplant (transplant occurred up to 22 days from start of chemotherapy)
Change in Fecal Acetate Levels
Change in fecal acetate levels from baseline at day+ 7
Measured: Day+ 7 after transplant
Change in Fecal Propionate Levels
Change in fecal propionate levels from baseline at day+ 7
Measured: Day+ 7 after transplant
Change in Fecal Butyrate Levels
Change in fecal butyrate levels from baseline at day+ 7
Measured: Day+ 7 after transplant
Secondary outcomes (9)
Relative Abundance of Fecal Bifidobacteria at Day+30 Compared to Baseline
Stool underwent shotgun metagenomic sequencing and Kraken2 taxonomic classification; read counts were rarefied to a common depth. For each participant, the relative abundance (percentage of total classified reads) of the genus Bifidobacterium was calculated at baseline and at Day +30; phylum values were obtained by summing all constituent genera. The change from baseline (Day +30 minus baseline) was computed for participants with paired samples. Values are summarized as median [interquartile range].
Measured: Day+30 after transplant
Relative Abundance of Fecal Firmicutes at Day+30 Compared to Baseline for Patients on 2FL
Stool underwent shotgun metagenomic sequencing and Kraken2 taxonomic classification; read counts were rarefied to a common depth. For each participant, the relative abundance (percentage of total classified reads) of the phyla Firmicutes was calculated at baseline and at Day +30; phylum values were obtained by summing all constituent genera. The change from baseline (Day +30 minus baseline) was computed for participants with paired samples. Values are summarized as median [interquartile range].
Measured: Day+30 after transplant
Incidence of Acute GVHD
Incidence of acute GVHD in patients on 2FL
Measured: Day+100 after transplant
Incidence of Mucosal Barrier Injury Laboratory-confirmed Bloodstream Infection (MBI-LCBI)
Incidence of mucosal barrier injury laboratory confirmed bloodstream infections were collected from first dose of 2-FL until day+100 after transplant in evaluable patients
Measured: Day+ 100 after transplant
Urine 3-indoxyl Sulfate (3-IS) Levels
Unit of measure is urine 3-indole sulfate levels in mM normalized to urine creatinine
Measured: Baseline
Urine 3-indoxyl Sulfate (3-IS) Levels
Unit of measure is urine 3-indole sulfate levels in mM normalized to urine creatinine
Measured: day+7
Urine 3-indoxyl Sulfate (3-IS) Levels
Unit of measure is urine 3-indole sulfate levels in mM normalized to urine creatinine
Measured: day+14
Urine 3-indoxyl Sulfate (3-IS) Levels
Unit of measure is urine 3-indole sulfate levels in mM normalized to urine creatinine
Measured: Day+30
Relative Abundance of Fecal Proteobacteria at Day+30 Compared to Baseline for Patients on 2FL
Stool underwent shotgun metagenomic sequencing and Kraken2 taxonomic classification; read counts were rarefied to a common depth. For each participant, the relative abundance (percentage of total classified reads) of the phyla Proteobacteria was calculated at baseline and at Day +30); phylum values were obtained by summing all constituent genera. The change from baseline (Day +30 minus baseline) was computed for participants with paired samples. Values are summarized as median [interquartile range].
Measured: Day+30 after transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Feb 2020
- Study start26 Aug 2020
- Primary completion23 May 2025
- Study completion23 May 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hematopoietic Stem Cell TransplantStudies of the same condition, open and under way.
- More trials run by Children's Hospital Medical Center, CincinnatiThe 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
- Non-randomised
- Intervention model
- Parallel groups
- 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)
- Enrolment
- 70 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Pooja Khandelwal, MD, Principal investigator, Children's Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04263597
- Sponsor's protocol ID
- 2020-0008
- Status last verified
- Aug 2026
- First submitted
- 7 Feb 2020
- Last updated
- 2 Sep 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Ramos KN, Langenberg L, Lake KE, Hoerth CA, Luebbering N, Haslam DB, Newburg D, Morrow AL, Ollberding NJ, Teusink-Cross A, Taggart C, Bota K, Cook E, Khoury R, Denson LA, Watanabe-Chailland M, Romick L, Davies SM, Khandelwal P. Human Milk Oligosaccharide (2'-FL) Modifies the Gut Microbiome in Children after Bone Marrow Transplant. Blood. 2026 Aug 11:blood.2026034172. doi: 10.1182/blood.2026034172. Online ahead of print.
PubMed 42622257
Study at a glance
From the registry record, last updated by the sponsor on 2 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Oral Supplementation of 2'-Fucosyllactose in Allogeneic Bone Marrow Transplant Recipients to Maintain Intestinal Homeostasis
- ClinicalTrials.gov ID
- NCT04263597
- Acronym / sponsor ID
- 2020-0008
- Conditions
- Hematopoietic Stem Cell Transplant
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 70 participants (actual)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Study start
- 26 Aug 2020 (actual)
- Primary completion
- 23 May 2025 (actual)
- Study completion
- 23 May 2025 (actual)
- First posted
- 11 Feb 2020 (actual)
- Results
- Posted 2 Sep 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Phase II:
Initial 15 patients to establish safety as per the FDA have been enrolled. An additional 10 patients were enrolled and interim analyses demonstrating safety, lack of any dose limiting toxicities and a positive signal of increase in fecal acetate and propionate at day+7 compared to baseline values) were performed. The investigators will enroll approximately 65 additional patients to test efficacy of 2FL supplementation in children and young adult allogeneic HSCT patients with a goal to reduce intestinal inflammation and improve post HSCT outcomes such as acute GVHD and bloodstream infections.
Study design
- Primary purpose
- Prevention
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
- 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
| Arm | Type | Receives |
|---|---|---|
| 2'-fucosyllactose for ages 0-5 years | Experimental | Drug: 2'-fucosyllactoseDose for ages 0-5 years: 2.5 g/day; |
| 2'-fucosyllactose for ages 5.1-10 years | Experimental | Drug: 2'-fucosyllactoseDose for ages 5.1-10 years: 5 g/day; |
| 2'-fucosyllactose for ages >10 years | Experimental | Drug: 2'-fucosyllactoseDose for ages >10 years: 10 g/day; |
Who may qualify
- Ages
- 0 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Be scheduled for allogeneic stem cell transplant
- All ages and underlying diagnoses, preparative regimens, stem cell sources and acute GVHD prophylaxes
Key exclusion criteria (7 listed)
- 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
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 |
|---|---|---|
| 2'-fucosyllactoseAlso: 2FL supplementation2FL 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. | Drug | 2'-fucosyllactose for ages 0-5 years, 2'-fucosyllactose for ages 5.1-10 years, 2'-fucosyllactose for ages >10 years |
Primary outcome
Number of Bloodstream Infections
Time frame: Day+100 after transplant
Patients with a bloodstream infection occurring anytime between first day of 2-FL dosing until day+100 after transplant were collected. Typically 2FL was started with start of chemotherapy which was usually up to 22 days before transplant
Number of Patients Able to Take 2FL
Time frame: 1 week prior to start of chemotherapy until day+30 after transplant (transplant occurred up to 22 days from start of chemotherapy)
Daily drug doses were documented by patients or nursing staff as applicable. Patients who took at least 60% of their total planned doses were deemed to meet protocol defined adherence to 2-FL
Change in Fecal Acetate Levels
Time frame: Day+ 7 after transplant
Change in fecal acetate levels from baseline at day+ 7
Change in Fecal Propionate Levels
Time frame: Day+ 7 after transplant
Change in fecal propionate levels from baseline at day+ 7
Change in Fecal Butyrate Levels
Time frame: Day+ 7 after transplant
Change in fecal butyrate levels from baseline at day+ 7
9 secondary outcomes
Relative Abundance of Fecal Bifidobacteria at Day+30 Compared to Baseline
Time frame: Day+30 after transplant
Relative Abundance of Fecal Firmicutes at Day+30 Compared to Baseline for Patients on 2FL
Time frame: Day+30 after transplant
Incidence of Acute GVHD
Time frame: Day+100 after transplant
Incidence of Mucosal Barrier Injury Laboratory-confirmed Bloodstream Infection (MBI-LCBI)
Time frame: Day+ 100 after transplant
Urine 3-indoxyl Sulfate (3-IS) Levels
Time frame: Baseline
Urine 3-indoxyl Sulfate (3-IS) Levels
Time frame: day+7
Urine 3-indoxyl Sulfate (3-IS) Levels
Time frame: day+14
Urine 3-indoxyl Sulfate (3-IS) Levels
Time frame: Day+30
Relative Abundance of Fecal Proteobacteria at Day+30 Compared to Baseline for Patients on 2FL
Time frame: Day+30 after transplant
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Sep 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 42622257Ramos KN, Langenberg L, Lake KE, Hoerth CA, Luebbering N, Haslam DB, Newburg D, Morrow AL, Ollberding NJ, Teusink-Cross A, Taggart C, Bota K, Cook E, Khoury R, Denson LA, Watanabe-Chailland M, Romick L, Davies SM, Khandelwal P. Human Milk Oligosaccharide (2'-FL) Modifies the Gut Microbiome in Children after Bone Marrow Transplant. Blood. 2026 Aug 11:blood.2026034172. doi: 10.1182/blood.2026034172. Online ahead of print.
- 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. This study is not recruiting at the moment.
Pooja Khandelwal, MD principal investigator
Children's Hospital Medical Center, Cincinnati
1 site 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 2'-fucosyllactose in people with Hematopoietic Stem Cell Transplant, run by Children's Hospital Medical Center, Cincinnati at 1 site 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 2 inclusion and 7 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: finished, as of the sponsor's last update on 2 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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. 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?
Yes. Results are posted in the registry (first posted 2 Sep 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Bloodstream Infections (time frame: Day+100 after transplant). The study lists 5 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 NCT04263597, 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 2 Sep 2026.