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CTRI Number  CTRI/2023/07/054803 [Registered on: 05/07/2023] Trial Registered Prospectively
Last Modified On: 17/08/2024
Post Graduate Thesis  No 
Type of Trial  Interventional 
Type of Study   Drug 
Study Design  Other 
Public Title of Study   Effect of Rifaximin on gut bacterial flora post Stem Cell Transplant in patients with Acute Leukemia. 
Scientific Title of Study   Randomized Trial to Study the Effect of Rifaximin on Gut Microbiome Diversity post Allogeneic Stem Cell Transplant in Acute Leukemia. 
Trial Acronym   
Secondary IDs if Any  
Secondary ID  Identifier 
NIL  NIL 
 
Details of Principal Investigator or overall Trial Coordinator (multi-center study)  
Name  Dr Anant Gokarn 
Designation  Professor 
Affiliation  Tata Memorial Centre 
Address  211, Paymaster Shodhika, ACTREC, Tata Memorial Centre, Sector 22, Kharghar, Navi Mumbai.

Raigarh
MAHARASHTRA
410210
India 
Phone  02268735000   
Fax    
Email  anantgokarn@gmail.com  
 
Details of Contact Person
Scientific Query
 
Name  Dr Anant Gokarn 
Designation  Professor 
Affiliation  Tata Memorial Centre 
Address  211, Paymaster Shodhika, ACTREC, Tata Memorial Centre, Sector 22, Kharghar, Navi Mumbai.

Raigarh
MAHARASHTRA
410210
India 
Phone  02268735000   
Fax    
Email  anantgokarn@gmail.com  
 
Details of Contact Person
Public Query
 
Name  Dr Anant Gokarn 
Designation  Professor 
Affiliation  Tata Memorial Centre 
Address  211, Paymaster Shodhika, ACTREC, Tata Memorial Centre, Sector 22, Kharghar, Navi Mumbai.

Raigarh
MAHARASHTRA
410210
India 
Phone  02268735000   
Fax    
Email  anantgokarn@gmail.com  
 
Source of Monetary or Material Support  
Awaiting funding from intramural and extramural sources 
 
Primary Sponsor  
Name  Lady Tata Memorial Trust  
Address  Bombay House, 24, Homi Mody Street,Mumbai400001 
Type of Sponsor  Other [Charitable Trust ] 
 
Details of Secondary Sponsor  
Name  Address 
NIL  NIL 
 
Countries of Recruitment     India  
Sites of Study  
No of Sites = 1  
Name of Principal Investigator  Name of Site  Site Address  Phone/Fax/Email 
Dr Anant Gokarn  Advanced Centre for Training, Research and Education in Cancer (ACTREC), Tata Memorial Centre  Room 211, Bone Marrow Transplant Unit- Dept. of Medical Oncology, Paymaster Shodika, ACTREC, Tata Memorial Centre Sector 22, Utsav Chowk- CISF road, Kharghar, Navi Mumbai
Raigarh
MAHARASHTRA 
91-022-68735000

anantgokarn@gmail.com 
 
Details of Ethics Committee  
No of Ethics Committees= 1  
Name of Committee  Approval Status 
INSTITUTIONAL ETHICS COMMITTEE-III  Approved 
 
Regulatory Clearance Status from DCGI  
Status 
Not Applicable 
 
Health Condition / Problems Studied  
Health Type  Condition 
Patients  (1) ICD-10 Condition: C81-C96||Malignant neoplasms of lymphoid, hematopoietic and related tissue,  
 
Intervention / Comparator Agent  
Type  Name  Details 
Comparator Agent  Standard of care treatment following allogeneic stem cell transplant in acute leukemia  Standard of care treatment including standard anti GVHD measures, antibiotic support and transfusions as needed. 
Intervention  Tablet Rifaximin  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. 
 
Inclusion Criteria  
Age From  18.00 Year(s)
Age To  70.00 Year(s)
Gender  Both 
Details  a. Patients with acute leukemia and planned for aHSCT.
b. Patients with age more than 18 years (> 18 years).
c. Patients who give written informed consent.
d. Underlying hematologic malignancy in which aHSCT is indicated.
e. ECOG performance status 0, 1 or 2 (on day of enrolment).
f. Adequate Liver function test with serum SGOT/AST and SGPT/ALT < 3.0 times upper normal limit (ULN) and total bilirubin < 2.0 times ULN (on day of enrolment).
 
 
ExclusionCriteria 
Details  a. Known hypersensitivity to rifaximin or other rifampicin antimicrobial agents
b. Patients on therapy with antibiotics for bacterial or fungal infections on day of enrolment (Except for azole prophylaxis for fungal infections, acyclovir prophylaxis for herpes and cotrimoxazole prophylaxis for pneumocystis jerovecii infections, which are permissible).
c. Patients with current or past history of inflammatory bowel disease
d. Patients with history of major bowel resection or patients with colostomy.
e. Use of rifampicin or rifaximin in last 1 month before enrolment.
f. Any serious medical condition or psychiatric illness that would prevent the subject from signing the informed consent form or in opinion of the investigator make the patient unfit for enrolment in the trial.
g. Patients on the following P-glycoprotein inhibitors at time of enrolment: Verapamil, ketoconazole and itraconazole will be excluded from the study.
 
 
Method of Generating Random Sequence   Stratified randomization 
Method of Concealment   Centralized 
Blinding/Masking   Open Label 
Primary Outcome  
Outcome  TimePoints 
To determine the impact of rifaximin on gut microbial alpha
diversity as measured by inverse simpson index (ISI) and compare it with controls. 
15 days post transplant 
 
Secondary Outcome  
Outcome  TimePoints 
To determine non relpase mortality (NRM) at 1 year post transplant in patients who receive peritransplant
transplant rifaximin and compare it with controls.  
1 year post transplant 
To compare the incidence of severe (grade III/IV) acute graft versus host disease (aGVHD) in patients who receive
peri-transplant rifaximin with that in control arm. 
1 year post transplant 
To determine impact of gut decontamination with rifaximin on incidence of
multidrug resistant (MDR) sepsis and usage of higher antibiotics (e.g. Carbapenems, colistin,
tigecycline, ceftazidime avibactum and ceftriaxone-sulbactam EDTA) in first 6
months post BMT 
6 months post transplant 
To determine the impact of rifaximin induced gut manipulation on immune
reconstitution, T cell repertoire post transplant and cytokine profile. 
1 year post transplant 
To use single cell transcriptomics (SCT) to identify immune cell
profile in gut biopsies post allogeneic stem cell transplant in order to get insights into
the impact of microbiome on gut immunity which may be responsible for aGVHD. 
1 year 
 
Target Sample Size   Total Sample Size="166"
Sample Size from India="166" 
Final Enrollment numbers achieved (Total)= "Applicable only for Completed/Terminated trials"
Final Enrollment numbers achieved (India)="Applicable only for Completed/Terminated trials" 
Phase of Trial   Phase 2 
Date of First Enrollment (India)   01/08/2023 
Date of Study Completion (India) Applicable only for Completed/Terminated trials 
Date of First Enrollment (Global)  Date Missing 
Date of Study Completion (Global) Applicable only for Completed/Terminated trials 
Estimated Duration of Trial   Years="3"
Months="0"
Days="0" 
Recruitment Status of Trial (Global)
Modification(s)  
Not Applicable 
Recruitment Status of Trial (India)  Open to Recruitment 
Publication Details   Not yet 
Individual Participant Data (IPD) Sharing Statement

Will individual participant data (IPD) be shared publicly (including data dictionaries)?  

Response - NO
Brief Summary  
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, 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
 
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