A Phase 2 interventional study of Best Practice and Fecal Microbiota Transplantation in Graft-versus-host Disease Prevention, sponsored by M.D. Anderson Cancer Center. Withdrawn at 1 site in United States. Open to participants aged 16 Years to 75 Years. Per ClinicalTrials.gov, last updated 2020-03-04.
Sponsored by M.D. Anderson Cancer Center · Phase 2, Interventional, and Prevention
This phase II trial studies how well a fecal microbiota transplant with or without total gut decontamination works in preventing graft versus host disease in patients exposed to broad-spectrum antibiotics. Fecal microbiota transplantation is the administration by enema of fecal matter (stool) that includes helpful bacteria from a normal, healthy donor. Total gut decontamination uses antibiotics to remove/reduce the amount of bacteria in the digestive system. It is not yet known if a fecal microbiota transplant with or without total gut decontamination works better in preventing graft versus host disease compared to standard immunosuppressive therapies (therapies that lower the normal function of the immune system).
PRIMARY OBJECTIVES:
I. To estimate the proportion of patients who develop acute graft-versus-host disease (GVHD) of the gastrointestinal (GI) tract by day 100 post-transplant for patients randomized to the standard of care, total gut decontamination (TGD) followed by fecal microbiota transplant (fecal microbiota transplantation [FMT]) and FMT alone arms.
SECONDARY OBJECTIVES:
I. Overall maximum stage of lower GI tract GVHD by day 100 post-transplant. II. Cumulative incidence of acute GVHD grade II-IV and maximum grade through 6 months.
III. Time to onset of acute GVHD and acute GI GVHD. IV. Incidence of adverse events and serious adverse events. V. Incidence of bacterial blood stream infections through 6 months. VI. Hematologic recovery (neutrophils and platelets). VII. Characterization of the intestinal microbiota at enrollment, pre-FMT / time of engraftment, 2 month post-FMT/ engraftment, onset of gastrointestinal tract (GIT) GVHD, and at study completion (6 months).
VIII. Relapse-free survival at 6 months post-randomization. IX. Non-relapse mortality at 6 months post-randomization. X. Overall survival (OS) at 6 months post-randomization.
OUTLINE: Patients are randomized to 1 of 3 arms.
ARM A (TGD + FMT): Patients receive piperacillin-tazobactam orally (PO) three times daily (TID) and nystatin PO four times daily (QID) until FMT. Patients undergo stem cell transplantation on day 0, then undergo FMT via enema over 5-10 minutes within 3 weeks after transplantation.
ARM B (FMT): Patients undergo stem cell transplantation on day 0, then undergo FMT via enema over 5-10 minutes within 3 weeks after transplantation.
ARM C (STANDARD THERAPY): Patients receive standard of care.
After completion of study treatment, patients are followed up at 100 days and 6 months.
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Exclusion Criteria:
Patients receive piperacillin-tazobactam PO TID and nystatin QID. Patients undergo stem cell transplantation on day 0, then undergo FMT via enema over 5-10 minutes within 3 weeks after transplantation.
Procedure: Fecal Microbiota Transplantation · Drug: Nystatin · Drug: Piperacillin-Tazobactam
Patients undergo stem cell transplantation on day 0, then undergo FMT via enema over 5-10 minutes within 3 weeks after transplantation.
Procedure: Fecal Microbiota Transplantation
Patients receive standard of care.
Other: Best Practice
Given standard of care
Also known as: standard of care, standard therapy
Undergo FMT
Also known as: Fecal Material Transplantation, Fecal Transplantation, FMT, Poo Transplant, Poop Transplant, Stool Transplant
Given PO
Also known as: Mycostatin, Nystex
Given PO
Also known as: PIPER/TAZO, Piperacillin/Tazobactam, Zosyn
Development of acute gastrointestinal (GI) graft versus host disease (GVHD)
Summary statistics, including mean, median, range, and standard deviation, will be provided for continuous variables. Frequency tables will be used to summarize categorical variables.
Time frame: Within 100 days from time of transplant
Relapse-free survival
Relapse is defined by either morphological or cytogenetic evidence of acute leukemia or myelodysplastic syndrome (MDS) inconstant with pre-transplant features, or radiologic evidence of lymphoma, documented or not by biopsy. The distribution of time-to-event endpoints, as well as the median and 90% confidence interval, will be estimated using the Kaplan-Meier method.
Time frame: At 6 months post-randomization
Microbiome diversity
Will be measured using the inverse Simpson index. Summary statistics, including mean, median, range, and standard deviation, will be provided for continuous variables. Frequency tables will be used to summarize categorical variables.
Time frame: At 2 weeks post fecal microbiota transplantation (FMT) (or engraftment for control arm)
Overall maximum stage of lower GI tract GVHD
Will be defined as the proportion of patients who do not develop GI GVHD within 100 days post-transplant and will be monitored simultaneously in cohorts of 5 patients separately in each arm using the approach of Thall, Simon, and Estey.
Time frame: Within 100 days post-transplant
Cumulative incidence of acute GVHD grade II-IV and maximum grade
Summary statistics, including mean, median, range, and standard deviation, will be provided for continuous variables. Frequency tables will be used to summarize categorical variables.
Time frame: Up to 6 months
Incidence of adverse and serious adverse events
Defined as the proportion of patients who develop blood stream infections caused by enteric bacteria within 60 days of FMT. Will be monitored simultaneously in cohorts of 5 patients separately in each arm using the approach of Thall, Simon, and Estey.
Time frame: Within 60 days of FMT
Incidence of bacterial blood stream infections
Will identify those caused by a potential enteric pathogen. Summary statistics, including mean, median, range, and standard deviation, will be provided for continuous variables. Frequency tables will be used to summarize categorical variables.
Time frame: Up to 6 months
Hematologic recovery (neutrophils and platelets)
Summary statistics, including mean, median, range, and standard deviation, will be provided for continuous variables. Frequency tables will be used to summarize categorical variables.
Time frame: Up to 6 months
Characterization of the intestinal microbiota
Summary statistics, including mean, median, range, and standard deviation, will be provided for continuous variables. Frequency tables will be used to summarize categorical variables.
Time frame: Baseline up to 6 months
Non-relapse mortality
Relapse is defined by either morphological or cytogenetic evidence of acute leukemia or MDS inconstant with pre-transplant features, or radiologic evidence of lymphoma, documented or not by biopsy. The distribution of time-to-event endpoints, as well as the median and 90% confidence interval, will be estimated using the Kaplan-Meier method.
Time frame: At 6 months post-randomization
Overall survival
The distribution of time-to-event endpoints, as well as the median and 90% confidence interval, will be estimated using the Kaplan-Meier method.
Time frame: At 6 months post-randomization
Analysis of T-cell subsets
Analysis of T-cell subsets (including specifically regulatory T-cells) will be performed by characterization of peripheral blood flow cytometry.
Time frame: Up to 6 months post-enrollment
Analysis of serum/stool butyrate levels
Time frame: Up to 6 months post-enrollment
Assessment of gut permeability
Will be performed via lactulose/ mannitol assay.
Time frame: At time of discontinuation of antibiotics (engraftment)
This study is withdrawn, as verified in Mar 2020. You cannot join it, but the record below documents what was studied.
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M.D. Anderson Cancer Center