Rifaximin for Infection Prophylaxis in Hematopoietic Stem Cell Transplantation

NCT03529825 | Completed Early Phase 1 DMC FDA Drug

• 1 other identifier: IRB00101158
• Study Type: interventional
• Target: 26
• Locations: 1 country
• Sites: 1

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).

Conditions

Microbial Colonization

Keywords

Rifaximin; Microbiome; Allogeneic hematopoietic stem cell transplantation; Blood stream infections; Acute graft versus host disease

Outcome Measures

Primary Outcomes (1)

• 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.

  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.

  Period between the start of the preparative regimen and day 28 post transplant

• Transplant related mortality (TRM)

  Period between the start of the preparative regimen and day 28 post transplant

• Number of patients with Acute GVHD

  Period between the start of the preparative regimen and day 100 post transplant

• Number of patients with Chronic GVHD including overlap syndrome

  Period between the start of the preparative regimen and year 5 post-transplant.

• Number of patients with other Infections

  Period between the start of the preparative regimen and day 28 post transplant

Study Arms (2)

Rifaximin

EXPERIMENTAL

Rifaximin will be administered twice a day orally or by nasogastric tube to patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT).

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.

Interventions

Rifaximin DRUG

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).

Rifaximin

Eligibility Criteria

• Age: 2 Years - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Emory University: lead

Study Sites (1)

Children's Healthcare of Atlanta

Atlanta, Georgia, 30322, United States

Location

MeSH Terms

Conditions

Communicable Diseases

Interventions

Rifaximin

Condition Hierarchy (Ancestors)

Infections; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Rifamycins; Heterocyclic Compounds, 4 or More Rings; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds; Lactams, Macrocyclic; Macrocyclic Compounds; Polycyclic Compounds

Study Officials

• Muna Qayed, MD MSc

  Emory University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor

Model Details: Pilot, single arm prospective study with retrospective control arm

Study Record Dates

• First Submitted: May 8, 2018
• First Posted: May 18, 2018
• Study Start: July 18, 2018
• Primary Completion: October 19, 2020
• Study Completion: September 10, 2021
• Last Updated: October 12, 2021

Record last verified: 2021-10

Locations

US (1)