Clinical trial NCT03529825: Rifaximin for Infection Prophylaxis in Hematopoietic Stem Cell Transplantation
Official title: Rifaximin for Infection Prophylaxis in Hematopoietic Stem Cell Transplantation
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: EARLY_PHASE1
Conditions: Microbial Colonization
Keywords: Rifaximin
Microbiome
Allogeneic hematopoietic stem cell transplantation
Blood stream infections
Acute graft versus host disease
Lead sponsor: Emory University
Sponsor class: OTHER
First posted: 2018-05-18
Enrollment: 26
Sex: ALL
Minimum age (years): 2
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Intervention model description: Pilot, single arm prospective study with retrospective control arm
Primary purpose: TREATMENT

Brief 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).

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).

Eligibility criteria: Inclusion Criteria:

1. Allogeneic HSCT recipients between the ages of 2 and 21 years.
2. Underlying hematologic malignancy, regardless of donor type or graft source.
3. Myeloablative conditioning regimen.

Exclusion Criteria:

1. Known hypersensitivity to rifaximin, or other rifamycin antimicrobial agents.
2. 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.
3. 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.
4. The use of prophylactic antibiotics is not permitted.
5. Following the standard practice in blood and marrow transplantation, pregnant or breast feeding patients will be excluded

Primary outcomes:
measure: Alterations to microbiome diversity in children treated with rifaximin compared to the historical cohort.
timeFrame: Period between the start of the preparative regimen and day 28 post transplant
Secondary outcomes:
measure: Rates of BSI pathogen infection/colonization frequency during the treatment period compared to the historical cohort.
timeFrame: Period between the start of the preparative regimen and day 28 post transplant
measure: Transplant related mortality (TRM)
timeFrame: Period between the start of the preparative regimen and day 28 post transplant
measure: Number of patients with Acute GVHD
timeFrame: Period between the start of the preparative regimen and day 100 post transplant
measure: Number of patients with Chronic GVHD including overlap syndrome
timeFrame: Period between the start of the preparative regimen and year 5 post-transplant.
measure: Number of patients with other Infections
timeFrame: Period between the start of the preparative regimen and day 28 post transplant
measure: Number of patients with relapse free survival at 1 year
timeFrame: Period between the start of the preparative regimen and year 1 post-transplant.
measure: Overall number of patients survived at 1 year
timeFrame: Period between the start of the preparative regimen and year 1 post-transplant.
Other outcomes:
Locations:
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta
state: Georgia
Study officials:
affiliation: Emory University
name: Muna Qayed, MD MSc
role: PRINCIPAL_INVESTIGATOR
References: