Acute LeukemiaPhase 2NCT06058572
Effect of Rifaximin on Gut Bacterial Flora Post Stem Cell Transplant in Patients With Acute Leukemia
A Phase 2 study testing Rifaximin 200Mg Tab and allogenic hematopoietic stem cell transplantation in people with Acute Leukemia, run by Tata Memorial Centre at 1 site in India.
Trial summary
Goal: This study is a randomized phase II interventional study. The purpose of this study is to see if addition of oral rifaximin tablets during allogeneic stem cell transplant can improve the quality of gut microbiome and reduce chances of death, infections and graft versus host disease (GVHD) post-transplant. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Acute Leukemia
- What is being tested?
- Rifaximin 200Mg Tab and allogenic hematopoietic stem cell transplantation
- Type of treatment
- Pill or oral medicine, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 166 participants
- Where
- 1 siteIndia
- Study dates
- 2 Aug 2024to 2 Aug 2027
- Phase
- Phase 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 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adults with acute leukemia undergoing allogeneic stem cell transplant.
- ECOG performance status 0, 1 or 2.
- Adequate Liver function
Who cannot join
- Known hypersensitivity to rifaximin or other rifampicin antimicrobial agents
- Current or past history of inflammatory bowel disease
- History of major bowel resection or presence of colostomy.
- Ongoing Verapamil, ketoconazole or itraconazole.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
India1 site, 1 recruiting
- Recruiting
Actrec Tmc
Navi Mumbai, Maharashtra, 410210
Anant Gokarn, DM, anantgokarn@gmail.com
Sumeet Mirgh, DM, anantgokarn@gmail.com
About this study
Official title: Randomized Trial to Study the Effect of Rifaximin on Gut Microbiome Diversity Post Allogeneic Stem Cell Transplant in Acute Leukemia.
- Goal: This study is a randomized phase II interventional study. The purpose of this study is to see if addition of oral rifaximin tablets during allogeneic stem cell transplant can improve the quality of gut microbiome and reduce chances of death, infections and graft versus host disease (GVHD) post-transplant.
- The study objectives are as follows:
- Primary Objective: To determine the impact of rifaximin on gut microbial diversity and compare it with controls.
- Secondary Objectives: a. To determine non-relapse mortality at 1-year post transplant in patients who receive peri-transplant transplant rifaximin and compare it with controls.
- b. To compare the incidence of severe GVHD in patients who receive peri-transplant rifaximin with the controls.
- c. To determine impact of gut decontamination with rifaximin on incidence of MDR sepsis and usage of higher antibiotics (e.g. Carbapenems, colistin, tigecycline, ceftazidime avibactum and ceftriaxone-sulbactam EDTA) in first 6 months post BMT.
- d. To determine the impact of rifaximin induced gut manipulation on immune reconstitution, T cell repertoire post-transplant and cytokine profile.
- Exploratory objective: To use single cell transcriptomics (SCT) to identify immune cell profile in gut biopsies post allogeneic stem cell transplant whenever biopsy is done, to correlate the impact of microbiome on gut immunity.
- Intervention: Tab Rifaximin 200 mg will be given orally twice daily from day -8 to day +60 of allogeneic stem cell transplant in acute leukemia patients. This will be in addition to standard of care post-transplant treatment.
- Comparator Agent: Standard of care treatment including standard anti GVHD measures, antibiotic support and transfusions as needed.
Read the detailed description
The gut microbiome plays a significant role in modulating the immune re-constitution post allogeneic stem cell transplant (ASCT). Low gut microbial diversity has been consistently associated with poor outcomes of transplant including increased incidence of acute graft versus host disease (aGVHD), post-transplant bacterial sepsis and non-relapse mortality (NRM). However, the exact mechanism by which gut microbiome influences local as well as systemic immunity is not completely known, and is thought to be due to the impact of microbial metabolites on intestinal epithelial cells and host antigen-presenting cells. Understanding these mechanisms and modulating the microbiome may be crucial to improving transplant outcomes. Rifaximin is a locally acting antibiotic that has been approved for manipulating the gut microbiome in hepatic failure. It is unique because of its ability to clear pathogenic bacteria, while preserving the anaerobic commensals. It can potentially modify the gut microbiome to increase the alpha diversity and this may help reduce aGVHD, infectious complications, and mortality post-transplant. High incidence of multidrug resistant sepsis and frequent use of broad spectrum antibiotics in India, would result in higher rates of dysbiotic gut- making microbiome manipulation to improve transplant outcomes more relevant in our country. We are proposing a randomized controlled trial to understand the benefits of modulating the gut microbiome in patients of ASCT while investigating the local and global immune repertoire using single cell sequencing and multicolour flow cytometry.
Study design: Single center, open-labeled, phase II study, randomized controlled trial.
Primary Objective: To determine the impact of rifaximin on gut microbial alpha diversity and compare it with controls.
Secondary Objectives:
To determine impact of rifaximin on 1 year non relapse mortality post-transplant, incidence of grade III/IV aGVHD, incidence of MDR sepsis, patterns of immune cell reconstitution, and cytokine profile post-transplant.
Exploratory objective: To use single-cell transcriptomics (SCT) to identify immune cell profiles in gut biopsies post ASCT in order to get insights into the impact of the microbiome on local gut immunity.
Study population: Adult patients who undergo ASCT at the Tata Memorial Centre.
Study Methodology in brief: Patients would be randomized to receive either oral tablet rifaximin 200 mg twice daily along with standard posttransplant treatment or to receive standard of care treatment alone. Stool samples and blood samples will be collected at different time points for microbiome analysis and immune cell profiling respectively. We plan to perform 16s rRNA-based next-generation sequencing of all variable regions using a phased primer approach using stool DNA as a template. Gut microbiome diversity will be calculated using the inverse Simpson index. Immune cell profile would be analyzed using 16 color flow cytometry. In selected cases where patients undergo colonoscopic gut biopsy for aGVHD, we will also obtain samples for transcriptome sequencing. This will help us understand how immune cells interact with gut mucosa and microbiome in patients of aGVHD
- Conditions
- Acute Leukemia
- Being tested
- Rifaximin 200Mg Tab, allogenic hematopoietic stem cell transplantation
- Keywords
- Rifaximin, Gut Microbiome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Rifaximin 200Mg TabDrug
Drug rifaximin 200 mg tablet form orally twice daily (with or without food) from day-8 of transplant to day + 60 of transplant
allogenic hematopoietic stem cell transplantationProcedure or surgery
Control arm will undergo allogenic hematopoietic stem cell transplantation as standard of care
| Study group | Role | What they receive |
|---|---|---|
| Rifaxmin +aHSCT | Experimental | drug rifaximin 200 mg tablet form orally twice daily (with or without food) from day-8 of transplant to day + 60 of transplant Receives: Drug: Rifaximin 200Mg Tab |
| aHSCT alone | Active comparator | control arm will underwent allogenic hematopoietic stem cell transplantation procedure as per standard of care Receives: Procedure: allogenic hematopoietic stem cell transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Impact of rifaximin on gut microbial diversity.
Gut microbial diversity as measured by inverse Simpson index (ISI) on stool samples on day 14 post transplant in Rifaximin arm and in controls.
Measured: 14 days post transplant
Secondary outcomes (4)
Non relapse mortality
Non relapse mortality (NRM) at 1 year post transplant in patients who receive peri-transplant transplant rifaximin and in controls
Measured: 1 year post transplant
Incidence of severe (grade III/IV) acute graft versus host disease
Incidence of severe (grade III/IV) acute graft versus host disease (aGVHD) in patients who receive peri-transplant rifaximin and in control arm.
Measured: 1 year post transplant
Impact of gut decontamination with rifaximin on incidence of multidrug resistant sepsis post transplant.
Incidence of multidrug resistant (MDR) sepsis and usage of higher antibiotics (e.g. Carbapenems, colistin, tigecycline, ceftazidime avibactam and ceftriaxone-sulbactam EDTA) in first 6 months post BMT in both rifaximin arm and in controls.
Measured: 6 months post transplant
Impact of rifaximin induced gut manipulation on immune reconstitution
Immune-reconstitution, T cell repertoire post transplant as measured by multicolor flow-cytometry in patients who receive rifaximin and in controls.
Measured: 1 year post transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Sep 2023
- Study start2 Aug 2024
- Primary completion2 Aug 2027 (estimated)
- Study completion2 Aug 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute LeukemiaStudies of the same condition, open and under way.
- More trials run by Tata Memorial CentreThe 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Interventional Arm : Tab Rifaximin 200 mg along with allogeneic stem cell transplant Standard Arm : Allogeneic stem cell transplant alone (with no anti-microbial prophylaxis)
- Masking
- None (open label)
- Enrolment
- 166 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Tata Memorial Centre
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Dr. Anant Gokarn, Gokarn, Principal investigator, Advanced Centre for Treatment, Research and Education in Cancer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06058572
- Sponsor's protocol ID
- 900872
- Status last verified
- Jul 2025
- First submitted
- 21 Sep 2023
- Last updated
- 31 Jul 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: No not shared
Study at a glance
From the registry record, last updated by the sponsor on 31 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized Trial to Study the Effect of Rifaximin on Gut Microbiome Diversity Post Allogeneic Stem Cell Transplant in Acute Leukemia.
- ClinicalTrials.gov ID
- NCT06058572
- Acronym / sponsor ID
- 900872
- Conditions
- Acute Leukemia
- Keywords
- Rifaximin, Gut Microbiome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 166 participants (estimated)
- Lead sponsor
- Tata Memorial CentreUniversity, hospital or other
- Study start
- 2 Aug 2024 (actual)
- Primary completion
- 2 Aug 2027 (estimated)
- Study completion
- 2 Aug 2027 (estimated)
- First posted
- 28 Sep 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.
- Goal: This study is a randomized phase II interventional study. The purpose of this study is to see if addition of oral rifaximin tablets during allogeneic stem cell transplant can improve the quality of gut microbiome and reduce chances of death, infections and graft versus host disease (GVHD) post-transplant.
- The study objectives are as follows:
- Primary Objective: To determine the impact of rifaximin on gut microbial diversity and compare it with controls.
- Secondary Objectives: a. To determine non-relapse mortality at 1-year post transplant in patients who receive peri-transplant transplant rifaximin and compare it with controls.
- b. To compare the incidence of severe GVHD in patients who receive peri-transplant rifaximin with the controls.
- c. To determine impact of gut decontamination with rifaximin on incidence of MDR sepsis and usage of higher antibiotics (e.g. Carbapenems, colistin, tigecycline, ceftazidime avibactum and ceftriaxone-sulbactam EDTA) in first 6 months post BMT.
- d. To determine the impact of rifaximin induced gut manipulation on immune reconstitution, T cell repertoire post-transplant and cytokine profile.
- Exploratory objective: To use single cell transcriptomics (SCT) to identify immune cell profile in gut biopsies post allogeneic stem cell transplant whenever biopsy is done, to correlate the impact of microbiome on gut immunity.
- Intervention: Tab Rifaximin 200 mg will be given orally twice daily from day -8 to day +60 of allogeneic stem cell transplant in acute leukemia patients. This will be in addition to standard of care post-transplant treatment.
- Comparator Agent: Standard of care treatment including standard anti GVHD measures, antibiotic support and transfusions as needed.
Detailed description
The gut microbiome plays a significant role in modulating the immune re-constitution post allogeneic stem cell transplant (ASCT). Low gut microbial diversity has been consistently associated with poor outcomes of transplant including increased incidence of acute graft versus host disease (aGVHD), post-transplant bacterial sepsis and non-relapse mortality (NRM). However, the exact mechanism by which gut microbiome influences local as well as systemic immunity is not completely known, and is thought to be due to the impact of microbial metabolites on intestinal epithelial cells and host antigen-presenting cells. Understanding these mechanisms and modulating the microbiome may be crucial to improving transplant outcomes. Rifaximin is a locally acting antibiotic that has been approved for manipulating the gut microbiome in hepatic failure. It is unique because of its ability to clear pathogenic bacteria, while preserving the anaerobic commensals. It can potentially modify the gut microbiome to increase the alpha diversity and this may help reduce aGVHD, infectious complications, and mortality post-transplant. High incidence of multidrug resistant sepsis and frequent use of broad spectrum antibiotics in India, would result in higher rates of dysbiotic gut- making microbiome manipulation to improve transplant outcomes more relevant in our country. We are proposing a randomized controlled trial to understand the benefits of modulating the gut microbiome in patients of ASCT while investigating the local and global immune repertoire using single cell sequencing and multicolour flow cytometry.
Study design: Single center, open-labeled, phase II study, randomized controlled trial.
Primary Objective: To determine the impact of rifaximin on gut microbial alpha diversity and compare it with controls.
Secondary Objectives:
To determine impact of rifaximin on 1 year non relapse mortality post-transplant, incidence of grade III/IV aGVHD, incidence of MDR sepsis, patterns of immune cell reconstitution, and cytokine profile post-transplant.
Exploratory objective: To use single-cell transcriptomics (SCT) to identify immune cell profiles in gut biopsies post ASCT in order to get insights into the impact of the microbiome on local gut immunity.
Study population: Adult patients who undergo ASCT at the Tata Memorial Centre.
Study Methodology in brief: Patients would be randomized to receive either oral tablet rifaximin 200 mg twice daily along with standard posttransplant treatment or to receive standard of care treatment alone. Stool samples and blood samples will be collected at different time points for microbiome analysis and immune cell profiling respectively. We plan to perform 16s rRNA-based next-generation sequencing of all variable regions using a phased primer approach using stool DNA as a template. Gut microbiome diversity will be calculated using the inverse Simpson index. Immune cell profile would be analyzed using 16 color flow cytometry. In selected cases where patients undergo colonoscopic gut biopsy for aGVHD, we will also obtain samples for transcriptome sequencing. This will help us understand how immune cells interact with gut mucosa and microbiome in patients of aGVHD
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Interventional Arm : Tab Rifaximin 200 mg along with allogeneic stem cell transplant Standard Arm : Allogeneic stem cell transplant alone (with no anti-microbial prophylaxis)
| Arm | Type | Receives |
|---|---|---|
| Rifaxmin +aHSCT | Experimental | Drug: Rifaximin 200Mg Tabdrug rifaximin 200 mg tablet form orally twice daily (with or without food) from day-8 of transplant to day + 60 of transplant |
| aHSCT alone | Active comparator | Procedure: allogenic hematopoietic stem cell transplantationcontrol arm will underwent allogenic hematopoietic stem cell transplantation procedure as per standard of care |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Adults with acute leukemia undergoing allogeneic stem cell transplant.
- ECOG performance status 0, 1 or 2.
- Adequate Liver function
Key exclusion criteria (4 listed)
- Known hypersensitivity to rifaximin or other rifampicin antimicrobial agents
- Current or past history of inflammatory bowel disease
- History of major bowel resection or presence of colostomy.
- Ongoing Verapamil, ketoconazole or itraconazole.
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 |
|---|---|---|
| Rifaximin 200Mg TabDrug rifaximin 200 mg tablet form orally twice daily (with or without food) from day-8 of transplant to day + 60 of transplant | Drug | Rifaxmin +aHSCT |
| allogenic hematopoietic stem cell transplantationControl arm will undergo allogenic hematopoietic stem cell transplantation as standard of care | Procedure or surgery | aHSCT alone |
Primary outcome
Impact of rifaximin on gut microbial diversity.
Time frame: 14 days post transplant
Gut microbial diversity as measured by inverse Simpson index (ISI) on stool samples on day 14 post transplant in Rifaximin arm and in controls.
4 secondary outcomes
Non relapse mortality
Time frame: 1 year post transplant
Incidence of severe (grade III/IV) acute graft versus host disease
Time frame: 1 year post transplant
Impact of gut decontamination with rifaximin on incidence of multidrug resistant sepsis post transplant.
Time frame: 6 months post transplant
Impact of rifaximin induced gut manipulation on immune reconstitution
Time frame: 1 year post transplant
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 31 Jul 2025.
- Individual participant dataSharing planned: No. No not shared
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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.
Dr. Anant Gokarn, Gokarn principal investigator
Advanced Centre for Treatment, Research and Education in Cancer
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Rifaximin 200Mg Tab and allogenic hematopoietic stem cell transplantation in people with Acute Leukemia, run by Tata Memorial Centre at 1 site in India.
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 4 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 31 Jul 2025. 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 India. 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 phase mean?
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 Dr. Anant Gokarn, DM (+91-02268735000 ext. 8685, anantgokarn@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Impact of rifaximin on gut microbial diversity. (time frame: 14 days post transplant). 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 31 Jul 2025.