Allogeneic Hematopoietic Cell Transplantation (HCT)Phase 2NCT03720392
Fecal Microbiota Transplantation (FMT) in Recipients After Allogeneic Hematopoietic Cell Transplantation (HCT)
A Phase 2 study testing FMT in people with Allogeneic Hematopoietic Cell Transplantation (HCT), run by Zachariah Michael DeFilipp at 1 site in United States.
Trial summary
This research study is studying the role fecal microbiota transplantation may play in post-Hematopoietic Cell Transplantation (HCT) recipients Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Allogeneic Hematopoietic Cell Transplantation (HCT)
- What is being tested?
- FMT
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 8 participants
- Where
- 1 siteUnited States
- Study dates
- 15 Jan 2019to 28 May 2020
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Men or women ≥ 18 and ≤ 80 years old
- Patients designated to undergo myeloablative or intermediate intensity allogeneic peripheral blood or bone marrow hematopoietic cell transplantation. Consent will be obtained prior to admission for HSCT. Patients receiving any donor source of stem cells are eligible. Eligible conditioning regimens are those defined as myeloablative by the ASBMT Consensus Criteria (Bacigalupo 2009) as well as the combination of fludarabine with melphalan (100-140 mg/mg2)
- Any GVHD prophylaxis regimen is allowed.
- ECOG performance status ≤ 2 (Karnofsky ≥ 60%, see Appendix A)
- Patients with adequate physical function as measured by
- Cardiac: Left ventricular ejection fraction at rest must be ≥ 40%, or shortening fraction >25%
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- Hepatic:
- Bilirubin ≤ 2.5 mg/dL, except for patients with Gilbert's syndrome or hemolysis
- ALT, AST, and Alkaline Phosphatase < 5 x ULN
- Renal: Serum creatinine within normal range, or if serum creatinine is outside normal range, then renal function (measured or estimated creatinine clearance or GFR) ≥ 40mL/min/1.73m2
- Pulmonary: DLCO (corrected for hemoglobin), FEV1 and FVC ≥ 50% predicted
- Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Women of childbearing potential will have a urine pregnancy test, which must be negative, on Study Day 1, prior to receiving FMT. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study and for 3 months after FMT.
- Ability to understand and the willingness to sign a written informed consent document, including the willingness to accept risk of unrelated donor stool.
- Ability to swallow large capsules.
Who cannot join
- Prior allogeneic hematopoietic stem cell transplantation. (Patients may have received a prior autologous hematopoietic stem cell transplant.)
- Participants who are receiving any other investigational agents.
- Uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
- Patients with active or uncontrolled bacterial, viral, or fungal infection(s) requiring systemic therapy.
- Planned use of prophylactic donor lymphocyte infusion (DLI) therapy.
- Delayed gastric emptying syndrome or large hiatal hernia
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- Known chronic aspiration
- Participants with a history of significant allergy to foods not excluded from the donor diet (excluded foods are tree nuts, peanuts, shellfish, eggs)
- Pregnant and breast-feeding women are ineligible because they are not eligible for hematopoietic stem cell transplantation.
- HIV-positive participants are ineligible.
- Participants who are unable to swallow pills.
- Participants with end-stage liver disease (cirrhosis)
- Participants with acute, active gastrointestinal infection (e.g., typhlitis, diverticulitis, appendicitis)
- Participants with inflammatory bowel disease (e.g., ulcerative colitis, Crohn's)
- Prior total colectomy
Trial locations and contacts
1 site in 1 country.
United States1 site
Massachusetts General Hospital Cancer Center
Boston, Massachusetts, 02214
About this study
Official title: A Phase 2 Study of Fecal Microbiota Transplantation (FMT) in Recipients After Allogeneic Hematopoietic Cell Transplantation (HCT)
This research study is studying the role fecal microbiota transplantation may play in post-Hematopoietic Cell Transplantation (HCT) recipients
Read the detailed description
This research study is a Phase II clinical trial. Phase II clinical trials test the safety and effectiveness of an investigational intervention to learn whether the intervention works in treating a specific disease. "Investigational" means that the intervention is being studied.
The FDA (the U.S. Food and Drug Administration) has not approved FMT for this use.
After HCT, the body's microbiome (the natural existence of various bacteria and organisms) in the intestinal tract may be affected, in that the number and types of good bacteria is reduced (also called a reduction in microbial flora diversity). Studies have shown that the number and types of good bacteria in the gut can impact whether or not a person develop a disease called graft-versus-host disease (GVHD). GVHD occurs when donated bone marrow cells attack the body with an immune response. Researchers believe that more microbial flora diversity in the gut is linked to a lower risk of developing GVHD.
FMT is a process utilizing microbial components which are the good, healthy bacteria that would otherwise naturally occur in the body. Since the participant may have decreased microbial flora diversity after HCT, these microbial components are taken from a 3rd party donor. They are extracted from fecal matter (stool) and put into a capsule which the participant then ingest.
Researchers believe that FMT administration may play a role in restoring higher microbial flora diversity in the gut. Therefore, FMT administration may play a role in decreasing the likelihood of developing GVHD.
In this research study, the investigators are...
- Examining the microbial flora diversity of your gut after FMT administration
- Looking for incidence rate of GVHD and other post-HCT complications
- Being tested
- FMT, Placebo
- Keywords
- Allogeneic Hematopoietic Cell Transplantation (HCT)
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FMTDrug
FMT is a process utilizing microbial components which are the good, healthy bacteria that would otherwise naturally occur in the body.
PlaceboDrug
a harmless pill, medicine, or procedure prescribed more for the psychological benefit to the patient than for any physiological effect.
| Study group | Role | What they receive |
|---|---|---|
| FMT Capsules | Experimental | * Two doses of FMT: one standard dose starting within four (4) days from the start of the conditioning regimen prior to HCT and one non-standard dose starting within 4 weeks after engraftment after HCT.
* A standard dose of oral FMT is 15 capsules per day for two consecutive days, Receives: Drug: FMT |
| Placebo Capsules | Placebo comparator | * Two doses of placebo, instead of FMT: one starting within four (4) days from the start of the conditioning regimen prior to HCT and the second one starting within 4 weeks after engraftment after HCT.
* A standard dose of oral Placebo is 15 capsules per day for two consecutive days, Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Proportion of Patients Who Achieve Gut Microbiome Diversity at One Month Following the Final Post-HCT FMT
Gut microbiome diversity will be assessed using urinary 3-indoxyl sulfate (3-IS) levels, with 35 umol/mmol creatinine as the cutoff (≥ 35: diverse; < 35: not diverse).
Measured: 1 Month
Secondary outcomes (6)
Number of Participants With Acute Graft-Versus-Host-Disease (GvHD)
The cumulative incidence of overall grades II-IV and grades III-IV acute GVHD will be assessed through six months after last dose of FMT or placebo. Acute GVHD will be assessed using the Mount Sinai Acute GVHD International Consortium (MAGIC) criteria.
Measured: 6 months
Number of Participants With Non-relapse-mortality
Non-relapse mortality (NRM) is defined as the time from first dose of FMT or placebo to death without relapse or progression or underlying disease.
Measured: 6 Months and 12 months
Number of Participants With Infection at 100 Days
Measured: 100 Days
Number of Participants With Progression Free Survival at 12 Months Post-treatment
Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions. Progression-Free Survival (PFS) is defined as the time from first dose of FMT or placebo to the earlier of underlying disease progression or death due to any cause. PFS was assessed at 12 months for all participants.
Measured: 12 Months
Overall Survival
Overall survival is measured as the amount of time from the first dose of Fecal Microbiota Transplantation (FMT) or placebo, that participants in this study are still alive. Overall survival is reported as number of participants alive at 12 months.
Measured: 12 Months
Graft Versus Host Disease(GVHD)/ Relapse Free Survival (GRFS)
GVHD-Free/Relapse-Free Survival (GRFS) is measured as the amount of time from the first dose of Fecal Microbiota Transplantation (FMT) or placebo, that participants in this study are not diagnosed with GVHD, disease progression, or relapse. GRFS is reported as number of patients without GVHD, disease progression, or relapse at 12 months.
Measured: 12 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Oct 2018
- Study start15 Jan 2019
- Primary completion28 May 2020
- Study completion28 May 2020
Why it stopped: Terminated due to sponsor decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Allogeneic Hematopoietic Cell Transplantation (HCT)Studies 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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Zachariah Michael DeFilipp
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Zachariah Michael DeFilipp, Massachusetts General Hospital
- Investigators
- Zachariah DeFilipp, MD, Principal investigator, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03720392
- Sponsor's protocol ID
- 18-293
- Status last verified
- Nov 2021
- First submitted
- 24 Oct 2018
- Last updated
- 1 Dec 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Dec 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Study of Fecal Microbiota Transplantation (FMT) in Recipients After Allogeneic Hematopoietic Cell Transplantation (HCT)
- ClinicalTrials.gov ID
- NCT03720392
- Acronym / sponsor ID
- 18-293
- Keywords
- Allogeneic Hematopoietic Cell Transplantation (HCT)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Terminated due to sponsor decision
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Zachariah Michael DeFilippUniversity, hospital or other
- Study start
- 15 Jan 2019 (actual)
- Primary completion
- 28 May 2020 (actual)
- Study completion
- 28 May 2020 (actual)
- First posted
- 25 Oct 2018 (actual)
- Results
- Posted 1 Dec 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This research study is studying the role fecal microbiota transplantation may play in post-Hematopoietic Cell Transplantation (HCT) recipients
Detailed description
This research study is a Phase II clinical trial. Phase II clinical trials test the safety and effectiveness of an investigational intervention to learn whether the intervention works in treating a specific disease. "Investigational" means that the intervention is being studied.
The FDA (the U.S. Food and Drug Administration) has not approved FMT for this use.
After HCT, the body's microbiome (the natural existence of various bacteria and organisms) in the intestinal tract may be affected, in that the number and types of good bacteria is reduced (also called a reduction in microbial flora diversity). Studies have shown that the number and types of good bacteria in the gut can impact whether or not a person develop a disease called graft-versus-host disease (GVHD). GVHD occurs when donated bone marrow cells attack the body with an immune response. Researchers believe that more microbial flora diversity in the gut is linked to a lower risk of developing GVHD.
FMT is a process utilizing microbial components which are the good, healthy bacteria that would otherwise naturally occur in the body. Since the participant may have decreased microbial flora diversity after HCT, these microbial components are taken from a 3rd party donor. They are extracted from fecal matter (stool) and put into a capsule which the participant then ingest.
Researchers believe that FMT administration may play a role in restoring higher microbial flora diversity in the gut. Therefore, FMT administration may play a role in decreasing the likelihood of developing GVHD.
In this research study, the investigators are...
- Examining the microbial flora diversity of your gut after FMT administration
- Looking for incidence rate of GVHD and other post-HCT complications
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| FMT Capsules | Experimental | Drug: FMT* Two doses of FMT: one standard dose starting within four (4) days from the start of the conditioning regimen prior to HCT and one non-standard dose starting within 4 weeks after engraftment after HCT. * A standard dose of oral FMT is 15 capsules per day for two consecutive days, |
| Placebo Capsules | Placebo comparator | Drug: Placebo* Two doses of placebo, instead of FMT: one starting within four (4) days from the start of the conditioning regimen prior to HCT and the second one starting within 4 weeks after engraftment after HCT. * A standard dose of oral Placebo is 15 capsules per day for two consecutive days, |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- Men or women ≥ 18 and ≤ 80 years old
- Patients designated to undergo myeloablative or intermediate intensity allogeneic peripheral blood or bone marrow hematopoietic cell transplantation. Consent will be obtained prior to admission for HSCT. Patients receiving any donor source of stem cells are eligible. Eligible conditioning regimens are those defined as myeloablative by the ASBMT Consensus Criteria (Bacigalupo 2009) as well as the combination of fludarabine with melphalan (100-140 mg/mg2)
- Any GVHD prophylaxis regimen is allowed.
- ECOG performance status ≤ 2 (Karnofsky ≥ 60%, see Appendix A)
- Patients with adequate physical function as measured by
Key exclusion criteria (15 listed)
- Prior allogeneic hematopoietic stem cell transplantation. (Patients may have received a prior autologous hematopoietic stem cell transplant.)
- Participants who are receiving any other investigational agents.
- Uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
- Patients with active or uncontrolled bacterial, viral, or fungal infection(s) requiring systemic therapy.
- Planned use of prophylactic donor lymphocyte infusion (DLI) therapy.
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 |
|---|---|---|
| FMTFMT is a process utilizing microbial components which are the good, healthy bacteria that would otherwise naturally occur in the body. | Drug | FMT Capsules |
| Placeboa harmless pill, medicine, or procedure prescribed more for the psychological benefit to the patient than for any physiological effect. | Drug | Placebo Capsules |
Primary outcome
The Proportion of Patients Who Achieve Gut Microbiome Diversity at One Month Following the Final Post-HCT FMT
Time frame: 1 Month
Gut microbiome diversity will be assessed using urinary 3-indoxyl sulfate (3-IS) levels, with 35 umol/mmol creatinine as the cutoff (≥ 35: diverse; < 35: not diverse).
6 secondary outcomes
Number of Participants With Acute Graft-Versus-Host-Disease (GvHD)
Time frame: 6 months
Number of Participants With Non-relapse-mortality
Time frame: 6 Months and 12 months
Number of Participants With Infection at 100 Days
Time frame: 100 Days
Number of Participants With Progression Free Survival at 12 Months Post-treatment
Time frame: 12 Months
Overall Survival
Time frame: 12 Months
Graft Versus Host Disease(GVHD)/ Relapse Free Survival (GRFS)
Time frame: 12 Months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Dec 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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.
Zachariah DeFilipp, MD principal investigator
Massachusetts General Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing FMT in people with Allogeneic Hematopoietic Cell Transplantation (HCT), run by Zachariah Michael DeFilipp at 1 site in United States.
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 14 inclusion and 15 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: stopped early, as of the sponsor's last update on 1 Dec 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 1 Dec 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
The Proportion of Patients Who Achieve Gut Microbiome Diversity at One Month Following the Final Post-HCT FMT (time frame: 1 Month). 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 1 Dec 2021.