Hematopoietic Cell Transplantation (HCT)Early Phase 1NCT05951153
Tributyrin to Promote Gut Health Among Children Undergoing Hematopoietic Cell Transplantation
A Early Phase 1 study testing Tributyrin in people with Hematopoietic Cell Transplantation (HCT), run by Duke University.
Trial summary
To determine the safety and tolerability of tributyrin among children undergoing hematopoietic cell transplantation (HCT). Read more about this study
- Who is it for?
- Ages 2 to 17 years
- Conditions the study is for
- Hematopoietic Cell Transplantation (HCT)
- What is being tested?
- Tributyrin
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 1 Aug 2024to Nov 2026
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
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 2 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age 2-17 years with standard of care nasogastric tube placement or existing gastric tubes
- Planned allogeneic hematopoietic cell transplantation with myeloablative preparative regimen
- Planned graft-versus-host-disease prophylaxis with calcineurin inhibitor + mycophenoalte mofetil or calcineurin inhibitor + methotrexate
Who cannot join
- Previous history of hematopoietic cell transplantataion
About this study
Official title: Phase I, Single-arm, Dose-escalation Trial of Tributyrin to Promote Gut Health Among Children Undergoing Hematopoietic Cell Transplantation
To determine the safety and tolerability of tributyrin among children undergoing hematopoietic cell transplantation (HCT).
Read the detailed description
Primary Objectives:
- Evaluate the safety and tolerability of tributyrin and determine the maximum tolerated dose MTD among children undergoing hematopoietic cell transplantation (HCT).
Secondary Objectives:
- Determine the effect of tributyrin administration on fecal butyrate concentrations.
- Measure the effect of tributyrin on gut microbiome composition and measures of gut inflammation and barrier integrity.
Hypotheses
- Enteral administration of tributyrin will be safe and well-tolerated in children undergoing HCT at doses up to 100 mg/kg/day, with no identified dose-limiting toxicities. The most common adverse events will be gastrointestinal and will occur at frequencies and grades typically observed among children after HCT. No adverse effects will occur that are classified as probably or definitely related to the study product.
- Tributyrin will result in a dose-dependent increase in fecal butyrate concentrations, with maximal fecal butyrate concentrations exceeding concentrations in baseline fecal samples by at least two-fold in >50% of subjects at all evaluated tributyrin doses.
- Compared to historical controls, children receiving tributyrin will have greater preservation of gut microbial diversity and higher abundances of Clostridiales and other putatively beneficial gut anaerobes (e.g., Bifidobacterium, Lactobacillus) after HCT. Additionally, children receiving tributyrin will have lower fecal levels of calprotectin and lactoferrin and lower plasma levels of lipopolysaccharide-binding protein and intestinal fatty acid-binding protein, corresponding to lower levels of intestinal inflammation and improved gut barrier function.
- Conditions
- Hematopoietic Cell Transplantation (HCT)
- Being tested
- Tributyrin
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TributyrinCombination product
Also called Nutritional supplement
Tributyrin is a nutritional supplement prepared by esterification of glycerin with butyric acid that promotes delivery of free butyrate to distal portions of the gut.
| Study group | Role | What they receive |
|---|---|---|
| Tributyrin nutritional supplement | Experimental | Children between the ages of 2-17 with planned allogeneic hematopoietic cell transplantation with myeloablative preparative regimen, who also have standard of care nasogastric tube placement or existing gastric tubes will receive a daily dose of tributyrin. Receives: Combination Product: Tributyrin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum tolerated dose (MTD) of tributyrin among children undergoing hematopoietic cell transplantation (HCT)
An evaluation of the safety and tolerability of tributyrin.
Measured: 36 months
Secondary outcomes (4)
Change in fecal butyrate concentration
Measured: Baseline, 36 months
Change in gut microbiome composition
Measured: Baseline, 36 months
Change in gut inflammation
Measured: Baseline, 36 months
Change in gut barrier integrity
Measured: Baseline, 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jul 2023
- Study start1 Aug 2024 (estimated)
- Primary completionNov 2025 (estimated)
- Study completionNov 2026 (estimated)
Why it stopped: PI withdrew study
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematopoietic Cell Transplantation (HCT)Studies of the same condition, open and under way.
- More trials run by Duke UniversityThe 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
- Early Phase 1
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Duke University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05951153
- Sponsor's protocol ID
- Pro00112603
- Status last verified
- Aug 2024
- First submitted
- 26 Jun 2023
- Last updated
- 15 Aug 2024
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided: IPD sharing plan is undecided by study team at this time.
Study at a glance
From the registry record, last updated by the sponsor on 15 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I, Single-arm, Dose-escalation Trial of Tributyrin to Promote Gut Health Among Children Undergoing Hematopoietic Cell Transplantation
- ClinicalTrials.gov ID
- NCT05951153
- Acronym / sponsor ID
- Pro00112603
- Conditions
- Hematopoietic Cell Transplantation (HCT)
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: PI withdrew study
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Duke UniversityUniversity, hospital or other
- Study start
- 1 Aug 2024 (estimated)
- Primary completion
- Nov 2025 (estimated)
- Study completion
- Nov 2026 (estimated)
- First posted
- 18 Jul 2023 (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.
To determine the safety and tolerability of tributyrin among children undergoing hematopoietic cell transplantation (HCT).
Detailed description
Primary Objectives:
- Evaluate the safety and tolerability of tributyrin and determine the maximum tolerated dose MTD among children undergoing hematopoietic cell transplantation (HCT).
Secondary Objectives:
- Determine the effect of tributyrin administration on fecal butyrate concentrations.
- Measure the effect of tributyrin on gut microbiome composition and measures of gut inflammation and barrier integrity.
Hypotheses
- Enteral administration of tributyrin will be safe and well-tolerated in children undergoing HCT at doses up to 100 mg/kg/day, with no identified dose-limiting toxicities. The most common adverse events will be gastrointestinal and will occur at frequencies and grades typically observed among children after HCT. No adverse effects will occur that are classified as probably or definitely related to the study product.
- Tributyrin will result in a dose-dependent increase in fecal butyrate concentrations, with maximal fecal butyrate concentrations exceeding concentrations in baseline fecal samples by at least two-fold in >50% of subjects at all evaluated tributyrin doses.
- Compared to historical controls, children receiving tributyrin will have greater preservation of gut microbial diversity and higher abundances of Clostridiales and other putatively beneficial gut anaerobes (e.g., Bifidobacterium, Lactobacillus) after HCT. Additionally, children receiving tributyrin will have lower fecal levels of calprotectin and lactoferrin and lower plasma levels of lipopolysaccharide-binding protein and intestinal fatty acid-binding protein, corresponding to lower levels of intestinal inflammation and improved gut barrier function.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Tributyrin nutritional supplement | Experimental | Combination Product: TributyrinChildren between the ages of 2-17 with planned allogeneic hematopoietic cell transplantation with myeloablative preparative regimen, who also have standard of care nasogastric tube placement or existing gastric tubes will receive a daily dose of tributyrin. |
Who may qualify
- Ages
- 2 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Age 2-17 years with standard of care nasogastric tube placement or existing gastric tubes
- Planned allogeneic hematopoietic cell transplantation with myeloablative preparative regimen
- Planned graft-versus-host-disease prophylaxis with calcineurin inhibitor + mycophenoalte mofetil or calcineurin inhibitor + methotrexate
Key exclusion criteria (1 listed)
- Previous history of hematopoietic cell transplantataion
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 |
|---|---|---|
| TributyrinAlso: Nutritional supplementTributyrin is a nutritional supplement prepared by esterification of glycerin with butyric acid that promotes delivery of free butyrate to distal portions of the gut. | Combination product | Tributyrin nutritional supplement |
Primary outcome
Maximum tolerated dose (MTD) of tributyrin among children undergoing hematopoietic cell transplantation (HCT)
Time frame: 36 months
An evaluation of the safety and tolerability of tributyrin.
4 secondary outcomes
Change in fecal butyrate concentration
Time frame: Baseline, 36 months
Change in gut microbiome composition
Time frame: Baseline, 36 months
Change in gut inflammation
Time frame: Baseline, 36 months
Change in gut barrier integrity
Time frame: Baseline, 36 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Aug 2024.
- Individual participant dataSharing planned: Undecided. IPD sharing plan is undecided by study team at this time.
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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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Tributyrin in people with Hematopoietic Cell Transplantation (HCT), run by Duke University.
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 1 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: withdrawn, as of the sponsor's last update on 15 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
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 phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
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?
Maximum tolerated dose (MTD) of tributyrin among children undergoing hematopoietic cell transplantation (HCT) (time frame: 36 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 15 Aug 2024.