InfectionPhase 2/3EFFECT-CPENCT03802461
Effectiveness of Fecal Flora Alteration for Eradication of Carbapenemase-producing Enterobacteriaceae Colonization
A Phase 2/3 study testing Fecal Microbiota Transplantation (FMT) in people with Infection, run by Susy Hota at 13 sites in Canada.
Trial summary
Carbapenemase-producing Enterobacteriaceae (CPE) are bacteria carried in the gastrointestinal tract that are resistant to carbapenems, antibiotics of last resort. CPE infections result in death in 25-50% of cases. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Infection
- What is being tested?
- Fecal Microbiota Transplantation (FMT)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 13 sitesCanada
- Study dates
- 22 Mar 2019to 20 Mar 2024
- Phase
- Phase 2/3
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age ≥ 18 years
- ≥ 1 rectal swab, groin, stool, or urine specimen positive for a CPE within the past 1 month.
- Presence of CPE will be confirmed at baseline through collection of pooled groin/rectal swab and urine specimen.
- Women of childbearing age must be using at least one reliable form of birth control.
- Must be able to provide informed consent.
Who cannot join
- Active infection with CPE at the time of assessment.
- Pregnancy, planned pregnancy or breastfeeding.
- Current admission to intensive care unit.
- Significantly immunocompromised patients .
- neutropenia (ANC < 1)
- ongoing use of systemic corticosteroids > 30 mg/day
Show all 23Show fewer
- ongoing use of biologic therapy
- undergoing chemotherapy, received chemotherapy ≤ 30 days from baseline visit, or expected to undergo chemotherapy in the upcoming 12 months
- active hematologic malignancy
- solid organ transplant recipient
- hematopoetic stem cell transplant recipient
- HIV positive patients with cluster differentiation 4 (CD4) cell count < 350
- Patients with ascites or receiving peritoneal dialysis.
- History of inflammatory bowel disease (Crohn's or Ulcerative colitis).
- Chronic diarrhea or active colitis for any reason.
- Ileus or active gastrointestinal motility disorder at baseline.
- History of total colectomy.
- Severe, irreversible bleeding disorder.
- History of anaphylactic or anaphylactoid allergic reaction to any foods.
- Anticipated life expectancy less than 6 months.
- Unable to tolerate enema.
- Participant is not a Canadian citizen or permanent resident, and not expected to remain in Toronto region for 12 months.
- Any reason in the view of the investigator.
Trial locations and contacts
13 sites in 1 country.
13 sites in 1 country
Canada13 sites
William Osler Health System
Brampton, Ontario, L6R 3J7
Joseph Brant Hospital
Burlington, Ontario, L7S 1W7
Lakeridge Health
Oshawa, Ontario, L1G 2B9
MacKenzie Health
Richmond Hill, Ontario, L4C 4Z3
The Scarborough Hospital
Scarborough Village, Ontario, M1P 2T7
North York General Hospital
Toronto, Ontario, M2K 1E1
Michael Garron Hospital
Toronto, Ontario, M4C 3E7
Sunnybrook Health Sciences Centre
Toronto, Ontario, M4N 3M5
St Michael's Hospital
Toronto, Ontario, M5B 1W8
University Health Network
Toronto, Ontario, M5B 1W8
Public Health Ontario Laboratories
Toronto, Ontario, M5G 1M1
Sinai Health System
Toronto, Ontario, M5G 1X5
St. Joseph Health Centre
Toronto, Ontario, M6R 1B5
About this study
Official title: Effectiveness of Fecal Flora Alteration for Eradication of Carbapenemase-producing Enterobacteriaceae Colonization Trial (EFFECT-CPE): a Multisite, Open-label, Randomized Controlled Feasibility Pilot Trial
Carbapenemase-producing Enterobacteriaceae (CPE) are bacteria carried in the gastrointestinal tract that are resistant to carbapenems, antibiotics of last resort. CPE infections result in death in 25-50% of cases. Fecal microbiota transplantation (FMT) is the transfer of stool from a healthy donor to a recipient to alter the composition of gut microbes. Early studies support its use for eliminating CPE carriage but definitive studies are lacking. The investigators propose a feasibility pilot for a multicenter, non-blinded randomized trial comparing the effectiveness of FMT with no intervention (standard of care) in eliminating intestinal carriage of CPE. Forty patients with CPE will be randomly assigned to receive FMT by enema or no intervention. Feasibility will be demonstrated by the ability to recruit and retain 40 patients over 12 months, and to provide FMT made at a central site to at least one off-site hospital. The primary clinical endpoint for the full trial is CPE intestinal carriage 3 months after the intervention. Secondary endpoints include: CPE carriage at 1, 6 and 12 months; time to decolonization of CPE; safety; CPE infections over 12 months; and, intestinal carriage of other antibiotic-resistant organisms. Data on the clinical outcomes will be collected but not analyzed in this feasibility study.
Read the detailed description
This is a multisite, open-label randomized controlled internal pilot trial designed to assess the feasibility of a larger trial aimed at determining the effectiveness of fecal microbiota transplantation (FMT) by enema in short and long term intestinal decolonization of carbapenemase-producing Enterobacteriaceae (CPE). Forty (40) asymptomatic adult patients intestinally colonized with CPE will be allocated in a 1:1 ratio to receive a bowel preparation followed by FMT by enema route, versus standard of care (no intervention). FMT will be provided by the University of Toronto Microbiota Therapeutics Outcomes Program (MTOP), using standardized operating procedures for recruiting and screening FMT donors, manufacturing FMT and administering FMT by enema. The feasibility outcomes are: successful randomization of 40 patients within 12 months, retention of >90% (36/40) of patients up to 6 months, and provision of FMT at a non-primary study site in at least one patient. Data on the clinical and exploratory outcomes will be collected but not analyzed in this pilot study. The primary clinical outcome is incidence of intestinal decolonization of CPE at 3 months. Secondary clinical outcomes include: time to decolonization of CPE; incidence of CPE clinical infections up to 12 months post-intervention; incidence of intestinal decolonization of CPE and other antibiotic-resistant organisms (extended spectrum beta-lactamase Enterobacteriaceae - ESBLs and vancomycin-resistant Enterococci - VRE) at 1, 3, 6 and 12 months post-intervention; and, safety profile. As an exploratory outcome, changes in fecal microbiome composition will be examined before and after intervention. This study leverages existing support, research infrastructures and expertise - including the Toronto Invasive Bacterial Diseases Network (TIBDN), Toronto Antimicrobial Resistance Research Network (TARRN), and the University of Toronto Microbiota Therapeutics Outcomes Program (MTOP) - to optimize feasibility regarding patient recruitment and FMT administration.
- Conditions
- Infection
- Being tested
- Fecal Microbiota Transplantation (FMT)
- Keywords
- Fecal Microbiota Transplantation, Gut Microbiome, Carbapenemase-producing Enterobacteriaceae (CPE)
- Medical subject headings
- Infections
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Fecal Microbiota Transplantation (FMT)Biological
Feces from healthy donor
| Study group | Role | What they receive |
|---|---|---|
| Fecal Microbiota Transplantation (FMT) | Active comparator | Bowel lavage preparation followed by FMT administered by enema, given on 3 occasions. Fecal filtrate for FMT will be prepared from 50 g of healthy donor stool, homogenized, and diluted in 300 mL sterile normal saline. Receives: Biological: Fecal Microbiota Transplantation (FMT) |
| Standard of Care | No intervention | Patients in this arm will not receive intervention and will be on standard of care . |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of intestinal colonization of patients with CPE 3 months after intervention.
Incidence of CPE colonization in FMT arm vs control arm at 3 months
Measured: 3 months
Randomization rate in study
Completion of randomization of 40 study participants during the study period will be used to indicate feasibility of the study.
Measured: 12 months
Proportion of patients retained in study for up to 6 months
A retention of 90% of patients up to 6 months in the study will be used to indicate feasibility.
Measured: 6 months
Secondary outcomes (6)
Incidence of CPE decolonization in FMT-treatment and non-treatment groups at 1, 6 and 12 months.
As above
Measured: 1, 6, and 12 months
Time to CPE decolonization in FMT-treatment and non-treatment groups.
As above
Measured: 1, 3, 6, and 12 months
Incidence of CPE clinical infection in FMT treatment and non-treatment groups over 12 months.
As above
Measured: 1, 3, 6, and 12 months
Incidence of extended spectrum beta-lactamase organisms (ESBL) and vancomycin-resistant Enterococci (VRE) intestinal colonization at 0, 1, 3, 6 and 12 months in FMT treatment and non-treatment groups.
Changes in colonization status of other antimicrobial resistant organisms over the study period
Measured: 1, 3, 6, and 12 months
Incidence of solicited and unsolicited adverse and serious adverse events in both groups
Participants will be asked to report adverse and serious adverse events will be throughout the study period and will be asked specifically about adverse events during study visits
Measured: 3 months
Number of patients with all-cause mortality at 30 days post-randomization
As above
Measured: 1 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jan 2019
- Study start22 Mar 2019
- Primary completion15 Dec 2023 (estimated)
- Study completion20 Mar 2024 (estimated)
Why it stopped: Temporarily on hold due to COVID-19 pandemic
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for InfectionStudies of the same condition, open and under way.
- 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
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Asymptomatic adult patients intestinally colonized with CPE will either randomized to receive fecal microbiota transplantation (intervention) or standard of care (no intervention).
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Susy Hota
- Sponsor type
- University, hospital or other
- Collaborators
- Sinai Health System, Ontario, Canada, University of Toronto, The Toronto Invasive Bacterial Diseases Network, Ontario, Canada, Public Health Ontario Laboratories, Ontario, Canada
- Responsible party
- Sponsor-investigator, Susy Hota, University Health Network, Toronto
- Investigators
- Susy S. Hota, MD MSc FRCPC, Principal investigator, Infectious Diseases Physician, University Health NetworkSusan M. Poutanen, MD MPH FRCPC, Principal investigator, Microbiologist & Infectious Disease Physician, Sinai Health System
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03802461
- Sponsor's protocol ID
- 18-5177
- Acronym
- EFFECT-CPE
- Status last verified
- Nov 2022
- First submitted
- 8 Nov 2018
- Last updated
- 3 Nov 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effectiveness of Fecal Flora Alteration for Eradication of Carbapenemase-producing Enterobacteriaceae Colonization Trial (EFFECT-CPE): a Multisite, Open-label, Randomized Controlled Feasibility Pilot Trial
- ClinicalTrials.gov ID
- NCT03802461
- Acronym / sponsor ID
- EFFECT-CPE / 18-5177
- Conditions
- Infection
- Keywords
- Fecal Microbiota Transplantation, Gut Microbiome, Carbapenemase-producing Enterobacteriaceae (CPE)
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- SuspendedReason stopped: Temporarily on hold due to COVID-19 pandemic
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Susy HotaUniversity, hospital or other
- Collaborators
- Sinai Health System, Ontario, Canada, University of Toronto, The Toronto Invasive Bacterial Diseases Network, Ontario, Canada, Public Health Ontario Laboratories, Ontario, Canada
- Study start
- 22 Mar 2019 (actual)
- Primary completion
- 15 Dec 2023 (estimated)
- Study completion
- 20 Mar 2024 (estimated)
- First posted
- 14 Jan 2019 (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.
Carbapenemase-producing Enterobacteriaceae (CPE) are bacteria carried in the gastrointestinal tract that are resistant to carbapenems, antibiotics of last resort. CPE infections result in death in 25-50% of cases. Fecal microbiota transplantation (FMT) is the transfer of stool from a healthy donor to a recipient to alter the composition of gut microbes. Early studies support its use for eliminating CPE carriage but definitive studies are lacking. The investigators propose a feasibility pilot for a multicenter, non-blinded randomized trial comparing the effectiveness of FMT with no intervention (standard of care) in eliminating intestinal carriage of CPE. Forty patients with CPE will be randomly assigned to receive FMT by enema or no intervention. Feasibility will be demonstrated by the ability to recruit and retain 40 patients over 12 months, and to provide FMT made at a central site to at least one off-site hospital. The primary clinical endpoint for the full trial is CPE intestinal carriage 3 months after the intervention. Secondary endpoints include: CPE carriage at 1, 6 and 12 months; time to decolonization of CPE; safety; CPE infections over 12 months; and, intestinal carriage of other antibiotic-resistant organisms. Data on the clinical outcomes will be collected but not analyzed in this feasibility study.
Detailed description
This is a multisite, open-label randomized controlled internal pilot trial designed to assess the feasibility of a larger trial aimed at determining the effectiveness of fecal microbiota transplantation (FMT) by enema in short and long term intestinal decolonization of carbapenemase-producing Enterobacteriaceae (CPE). Forty (40) asymptomatic adult patients intestinally colonized with CPE will be allocated in a 1:1 ratio to receive a bowel preparation followed by FMT by enema route, versus standard of care (no intervention). FMT will be provided by the University of Toronto Microbiota Therapeutics Outcomes Program (MTOP), using standardized operating procedures for recruiting and screening FMT donors, manufacturing FMT and administering FMT by enema. The feasibility outcomes are: successful randomization of 40 patients within 12 months, retention of >90% (36/40) of patients up to 6 months, and provision of FMT at a non-primary study site in at least one patient. Data on the clinical and exploratory outcomes will be collected but not analyzed in this pilot study. The primary clinical outcome is incidence of intestinal decolonization of CPE at 3 months. Secondary clinical outcomes include: time to decolonization of CPE; incidence of CPE clinical infections up to 12 months post-intervention; incidence of intestinal decolonization of CPE and other antibiotic-resistant organisms (extended spectrum beta-lactamase Enterobacteriaceae - ESBLs and vancomycin-resistant Enterococci - VRE) at 1, 3, 6 and 12 months post-intervention; and, safety profile. As an exploratory outcome, changes in fecal microbiome composition will be examined before and after intervention. This study leverages existing support, research infrastructures and expertise - including the Toronto Invasive Bacterial Diseases Network (TIBDN), Toronto Antimicrobial Resistance Research Network (TARRN), and the University of Toronto Microbiota Therapeutics Outcomes Program (MTOP) - to optimize feasibility regarding patient recruitment and FMT administration.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Asymptomatic adult patients intestinally colonized with CPE will either randomized to receive fecal microbiota transplantation (intervention) or standard of care (no intervention).
| Arm | Type | Receives |
|---|---|---|
| Fecal Microbiota Transplantation (FMT) | Active comparator | Biological: Fecal Microbiota Transplantation (FMT)Bowel lavage preparation followed by FMT administered by enema, given on 3 occasions. Fecal filtrate for FMT will be prepared from 50 g of healthy donor stool, homogenized, and diluted in 300 mL sterile normal saline. |
| Standard of Care | No intervention | –Patients in this arm will not receive intervention and will be on standard of care . |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Age ≥ 18 years
- ≥ 1 rectal swab, groin, stool, or urine specimen positive for a CPE within the past 1 month.
- Women of childbearing age must be using at least one reliable form of birth control.
- Must be able to provide informed consent.
Key exclusion criteria (23 listed)
- Active infection with CPE at the time of assessment.
- Pregnancy, planned pregnancy or breastfeeding.
- Current admission to intensive care unit.
- Significantly immunocompromised patients .
- Patients with ascites or receiving peritoneal dialysis.
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 |
|---|---|---|
| Fecal Microbiota Transplantation (FMT)Feces from healthy donor | Biological | Fecal Microbiota Transplantation (FMT) |
Primary outcome
Incidence of intestinal colonization of patients with CPE 3 months after intervention.
Time frame: 3 months
Incidence of CPE colonization in FMT arm vs control arm at 3 months
Randomization rate in study
Time frame: 12 months
Completion of randomization of 40 study participants during the study period will be used to indicate feasibility of the study.
Proportion of patients retained in study for up to 6 months
Time frame: 6 months
A retention of 90% of patients up to 6 months in the study will be used to indicate feasibility.
6 secondary outcomes
Incidence of CPE decolonization in FMT-treatment and non-treatment groups at 1, 6 and 12 months.
Time frame: 1, 6, and 12 months
Time to CPE decolonization in FMT-treatment and non-treatment groups.
Time frame: 1, 3, 6, and 12 months
Incidence of CPE clinical infection in FMT treatment and non-treatment groups over 12 months.
Time frame: 1, 3, 6, and 12 months
Incidence of extended spectrum beta-lactamase organisms (ESBL) and vancomycin-resistant Enterococci (VRE) intestinal colonization at 0, 1, 3, 6 and 12 months in FMT treatment and non-treatment groups.
Time frame: 1, 3, 6, and 12 months
Incidence of solicited and unsolicited adverse and serious adverse events in both groups
Time frame: 3 months
Number of patients with all-cause mortality at 30 days post-randomization
Time frame: 1 month
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Nov 2022.
- Individual participant dataSharing planned: No
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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.
Susy S. Hota, MD MSc FRCPC principal investigator
Infectious Diseases Physician, University Health Network
Susan M. Poutanen, MD MPH FRCPC principal investigator
Microbiologist & Infectious Disease Physician, Sinai Health System
13 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Fecal Microbiota Transplantation (FMT) in people with Infection, run by Susy Hota at 13 sites in Canada.
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 5 inclusion and 23 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: paused, as of the sponsor's last update on 3 Nov 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in Canada. 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 combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
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?
Incidence of intestinal colonization of patients with CPE 3 months after intervention. (time frame: 3 months). The study lists 3 primary outcomes in all. 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 3 Nov 2022; AzTrials last refreshed its copy on 8 Nov 2026.