Microbial ColonizationEarly Phase 1NCT03529825
Rifaximin for Infection Prophylaxis in Hematopoietic Stem Cell Transplantation
A Early Phase 1 study testing Rifaximin in people with Microbial Colonization, run by Emory University at 1 site in United States.
Trial summary
Primary purpose of the study is to see if rifaximin can improve the balance of bacteria within the gut, which has been shown to improve transplant outcomes. It will also assess whether rifaximin can reduce the risk of infection in blood/marrow transplant (BMT). Read more about this study
- Who is it for?
- Ages 2 to 21 years
- Conditions the study is for
- Microbial Colonization
- What is being tested?
- Rifaximin
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 26 participants
- Where
- 1 siteUnited States
- Study dates
- 18 Jul 2018to 10 Sep 2021
- 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 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Allogeneic HSCT recipients between the ages of 2 and 21 years.
- Underlying hematologic malignancy, regardless of donor type or graft source.
- Myeloablative conditioning regimen.
Who cannot join
- Known hypersensitivity to rifaximin, or other rifamycin antimicrobial agents.
- Minimally toxic conditioning regimen (e.g. low dose TBI based). Since these regimens induce minimal myelosuppression and gut injury, patients receiving them probably stand little to gain from antibiotic prophylaxis.
- Patients with ongoing bacterial, viral or fungal active infections are not eligible for this study. Patients who remain on broad spectrum antibiotics for the treatment of a previous infection are not eligible.
- The use of prophylactic antibiotics is not permitted.
- Following the standard practice in blood and marrow transplantation, pregnant or breast feeding patients will be excluded
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
About this study
Primary purpose of the study is to see if rifaximin can improve the balance of bacteria within the gut, which has been shown to improve transplant outcomes. It will also assess whether rifaximin can reduce the risk of infection in blood/marrow transplant (BMT).
Read the detailed description
This study is for patients who will be having a blood/marrow transplant (BMT) to treat leukemia, lymphoma or other cancer of the blood. The blood or marrow cells will come from another person (donor)-allogeneic BMT. Bacterial infections and acute graft versus host disease (AGVHD) are frequent complications of allogeneic BMT. Bacterial infections sometimes happen because injury to the gut during transplant allows gut bacteria to cross the injured gut barrier and get to the blood. AGVHD happens when certain white blood cells, called T-cells, in the donor cells (the graft) attack the patient's body.
Primary purpose of the study is to see if rifaximin can improve the balance of bacteria within the gut, which has been shown to improve transplant outcomes. It will also assess whether rifaximin can reduce the risk of infection in blood/marrow transplant (BMT).
- Conditions
- Microbial Colonization
- Being tested
- Rifaximin
- Keywords
- Rifaximin, Microbiome, Allogeneic hematopoietic stem cell transplantation, Blood stream infections, Acute graft versus host disease
- Medical subject headings
- Communicable Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RifaximinDrug
Rifaximin will be administered twice a day orally or by nasogastric tube, at a dose of 15 mg/kg divided BID with a maximum dose of 1,650 mg, day -7 to day +28 or discharge (maximum duration 36 days).
| Study group | Role | What they receive |
|---|---|---|
| Rifaximin | Experimental | Rifaximin will be administered twice a day orally or by nasogastric tube to patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT). Receives: Drug: Rifaximin |
| Retrospective comparison cohort | No intervention | Thirty six patients who underwent HSCT for hematologic malignancies, and received myeloablative conditioning, without prophylactic antibiotics, between 2013-2017 enrolled in the Aflac biorepository will comprise the comparison arm. Clinical data on transplant and infection characteristics is available and linked to stool microbiome samples already analyzed and described. Stored plasma and peripheral blood mononuclear cells are available for further analysis. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Alterations to microbiome diversity in children treated with rifaximin compared to the historical cohort.
Composition will be assessed using 16S RNA sequencing. The Shannon index will be calculated for quantification of bacterial diversity.
Measured: Period between the start of the preparative regimen and day 28 post transplant
Secondary outcomes (7)
Rates of BSI pathogen infection/colonization frequency during the treatment period compared to the historical cohort.
Results of the GI pathogen panel, a test incorporated into routine patient care detecting DNA or RNA of 22 common viral, bacterial, and parasitic organisms, will provide a second assessment through molecular detection of the presence of common organisms associated with intestinal dysbiosis.
Measured: Period between the start of the preparative regimen and day 28 post transplant
Transplant related mortality (TRM)
Number of deaths occurring in continuous complete remission.
Measured: Period between the start of the preparative regimen and day 28 post transplant
Number of patients with Acute GVHD
Early onset (before day 100) acute GVHD (including all grades, and stratified by grades) will be assessed according to the Blood and Marrow Transplant Clinical Trials Network Manual version 2, 2005, section 1 using the NIH consensus criteria
Measured: Period between the start of the preparative regimen and day 100 post transplant
Number of patients with Chronic GVHD including overlap syndrome
Chronic GVHD including overlap syndrome, will be assessed according to the 2014 NIH consensus criteria.
Measured: Period between the start of the preparative regimen and year 5 post-transplant.
Number of patients with other Infections
Other Infections will be defined in accordance with the Blood and Marrow Transplant Clinical Trials Network Manual of Procedures
Measured: Period between the start of the preparative regimen and day 28 post transplant
Number of patients with relapse free survival at 1 year
Survival without relapse of underlying malignancy.
Measured: Period between the start of the preparative regimen and year 1 post-transplant.
Overall number of patients survived at 1 year
Survival with or without relapse of underlying malignancy.
Measured: Period between the start of the preparative regimen and year 1 post-transplant.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 May 2018
- Study start18 Jul 2018
- Primary completion19 Oct 2020
- Study completion10 Sep 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Microbial ColonizationStudies of the same condition, open and under way.
- More trials run by Emory 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
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Model description
- Pilot, single arm prospective study with retrospective control arm
- Masking
- None (open label)
- Enrolment
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Muna Qayed, Emory University
- Investigators
- Muna Qayed, MD MSc, Principal investigator, Emory University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03529825
- Sponsor's protocol ID
- IRB00101158
- Status last verified
- Oct 2021
- First submitted
- 8 May 2018
- Last updated
- 12 Oct 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Oct 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Rifaximin for Infection Prophylaxis in Hematopoietic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT03529825
- Acronym / sponsor ID
- IRB00101158
- Conditions
- Microbial Colonization
- Keywords
- Rifaximin, Microbiome, Allogeneic hematopoietic stem cell transplantation, Blood stream infections, Acute graft versus host disease
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 26 participants (actual)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Study start
- 18 Jul 2018 (actual)
- Primary completion
- 19 Oct 2020 (actual)
- Study completion
- 10 Sep 2021 (actual)
- First posted
- 18 May 2018 (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.
Primary purpose of the study is to see if rifaximin can improve the balance of bacteria within the gut, which has been shown to improve transplant outcomes. It will also assess whether rifaximin can reduce the risk of infection in blood/marrow transplant (BMT).
Detailed description
This study is for patients who will be having a blood/marrow transplant (BMT) to treat leukemia, lymphoma or other cancer of the blood. The blood or marrow cells will come from another person (donor)-allogeneic BMT. Bacterial infections and acute graft versus host disease (AGVHD) are frequent complications of allogeneic BMT. Bacterial infections sometimes happen because injury to the gut during transplant allows gut bacteria to cross the injured gut barrier and get to the blood. AGVHD happens when certain white blood cells, called T-cells, in the donor cells (the graft) attack the patient's body.
Primary purpose of the study is to see if rifaximin can improve the balance of bacteria within the gut, which has been shown to improve transplant outcomes. It will also assess whether rifaximin can reduce the risk of infection in blood/marrow transplant (BMT).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Pilot, single arm prospective study with retrospective control arm
| Arm | Type | Receives |
|---|---|---|
| Rifaximin | Experimental | Drug: RifaximinRifaximin will be administered twice a day orally or by nasogastric tube to patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT). |
| Retrospective comparison cohort | No intervention | –Thirty six patients who underwent HSCT for hematologic malignancies, and received myeloablative conditioning, without prophylactic antibiotics, between 2013-2017 enrolled in the Aflac biorepository will comprise the comparison arm. Clinical data on transplant and infection characteristics is available and linked to stool microbiome samples already analyzed and described. Stored plasma and peripheral blood mononuclear cells are available for further analysis. |
Who may qualify
- Ages
- 2 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Allogeneic HSCT recipients between the ages of 2 and 21 years.
- Underlying hematologic malignancy, regardless of donor type or graft source.
- Myeloablative conditioning regimen.
Key exclusion criteria (5 listed)
- Known hypersensitivity to rifaximin, or other rifamycin antimicrobial agents.
- Minimally toxic conditioning regimen (e.g. low dose TBI based). Since these regimens induce minimal myelosuppression and gut injury, patients receiving them probably stand little to gain from antibiotic prophylaxis.
- Patients with ongoing bacterial, viral or fungal active infections are not eligible for this study. Patients who remain on broad spectrum antibiotics for the treatment of a previous infection are not eligible.
- The use of prophylactic antibiotics is not permitted.
- Following the standard practice in blood and marrow transplantation, pregnant or breast feeding patients will be excluded
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 |
|---|---|---|
| RifaximinRifaximin will be administered twice a day orally or by nasogastric tube, at a dose of 15 mg/kg divided BID with a maximum dose of 1,650 mg, day -7 to day +28 or discharge (maximum duration 36 days). | Drug | Rifaximin |
Primary outcome
Alterations to microbiome diversity in children treated with rifaximin compared to the historical cohort.
Time frame: Period between the start of the preparative regimen and day 28 post transplant
Composition will be assessed using 16S RNA sequencing. The Shannon index will be calculated for quantification of bacterial diversity.
7 secondary outcomes
Rates of BSI pathogen infection/colonization frequency during the treatment period compared to the historical cohort.
Time frame: Period between the start of the preparative regimen and day 28 post transplant
Transplant related mortality (TRM)
Time frame: Period between the start of the preparative regimen and day 28 post transplant
Number of patients with Acute GVHD
Time frame: Period between the start of the preparative regimen and day 100 post transplant
Number of patients with Chronic GVHD including overlap syndrome
Time frame: Period between the start of the preparative regimen and year 5 post-transplant.
Number of patients with other Infections
Time frame: Period between the start of the preparative regimen and day 28 post transplant
Number of patients with relapse free survival at 1 year
Time frame: Period between the start of the preparative regimen and year 1 post-transplant.
Overall number of patients survived at 1 year
Time frame: Period between the start of the preparative regimen and year 1 post-transplant.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Oct 2021.
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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.
Muna Qayed, MD MSc principal investigator
Emory University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Rifaximin in people with Microbial Colonization, run by Emory University 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 3 inclusion and 5 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 12 Oct 2021. 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 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Alterations to microbiome diversity in children treated with rifaximin compared to the historical cohort. (time frame: Period between the start of the preparative regimen and day 28 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 12 Oct 2021.