Bone Marrow TransplantationEarly Phase 1NCT02733744
Fecal Microbiota Transplantation After HSCT
A Early Phase 1 study testing Fecal Microbiota Transplantation (FMT) in people with Bone Marrow Transplantation, run by Massachusetts General Hospital at 1 site in United States.
Trial summary
This research study is studying an intervention called fecal microbiota transplantation (FMT). Patients who are scheduled to undergo Hematopoietic Stem Cell Transplantation are invited to take part in this clinical trial to undergo empiric FMT soon after hematopoietic engraftment. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Bone Marrow Transplantation
- What is being tested?
- Fecal Microbiota Transplantation (FMT)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteUnited States
- Study dates
- May 2016to Dec 2017
- Phase
- Early Phase 1
- 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 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Men or women ≥ 18 and ≤ 65 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 Consensus Criteria (Bacigalupo 2009) as well as the combination of fludarabine with melphalan (100-140 mg/mg2)
- Any Graft-vs-Host disease (GVHD) prophylaxis regimen is allowed.
- Eastern Cooperative Oncology Group (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
- Alanine aminotransferase (ALT), aspartate aminotransferase (AST), and Alkaline Phosphatase < 5 x Upper Limit of Institutional Normal Range (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) ≥ 40millileters/min/1.73m2.
- Pulmonary: Diffusing lung capacity for carbon monoxide (DLCO) (corrected for hemoglobin), Forced expiratory volume in 1 second (FEV1) and Forced vital capacity (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. 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 oral medications.
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.
- Patients with history of primary idiopathic myelofibrosis or any severe marrow fibrosis.
- Planned use of prophylactic donor lymphocyte infusion (DLI) therapy.
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- Delayed gastric emptying syndrome
- Known chronic aspiration
- Patients 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)
Trial locations and contacts
1 site in 1 country.
United States1 site
Massachusetts general Hospital
Boston, Massachusetts, 02114
About this study
Official title: A Pilot Study to Determine the Feasibility of Fecal MicrobiotaTransplantation (FMT) in Hematopoietic Stem Cell Transplantation (HSCT) Recipients
This research study is studying an intervention called fecal microbiota transplantation (FMT). Patients who are scheduled to undergo Hematopoietic Stem Cell Transplantation are invited to take part in this clinical trial to undergo empiric FMT soon after hematopoietic engraftment. The primary endpoint will be to assess the feasibility of FMT in this population and to assess safety.
Read the detailed description
The microbiome (spectrum of bacteria in a patient's gut) is thought to play a role in helping to shape one's immune system given its direct contact with normal cells in our intestine. Recent studies have suggested that a microbiome with very few bacteria (low diversity) seems to be bad for health and a high-diversity microbiome (many different species) appears to be good. This appears to be true even for patients after HSCT where low diversity microbiome status has correlated with infections, GVHD and overall survival.
Fecal microbiota transplantation (FMT) is routinely performed for an infection caused by Clostridium difficile but is not yet approved by the FDA as a treatment for any disease. FMT restores a high diversity microbiome. It is hoped that through FMT, overall outcomes after HSCT can potentially be improved.
FMT will be performed in the first 3 weeks after recovery of white blood cells after HSCT. The source of FMT will be from healthy 3rd party donors. FMT will be performed through the ingestion of 30 capsules over 2 consecutive days.
- Conditions
- Bone Marrow Transplantation
- Being tested
- Fecal Microbiota Transplantation (FMT)
- Keywords
- Stem cell transplant, fecal microbiota transplantation
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Fecal Microbiota Transplantation (FMT)Biological
Also called FMT
FMT will be given through the ingestion of 30 capsules (15 capsules daily x 2 consecutive days)
| Study group | Role | What they receive |
|---|---|---|
| Fecal Microbiota Transplant | Experimental | Each participant will undergo allogeneic hematopoietic stem cell transplantation, according to institutional standards.
Participants will receive a single standard dose of oral Fecal Microbiota Transplantation (FMT), which is 15 capsules per day for two consecutive days, for a total of 30 capsules. Participants will be asked to fast for 4 hours prior to and 1 hour following capsule intake. Capsules will be individually handed to participants by a research nurse or physician. Each capsule will be taken with a sip of water. Receives: Biological: Fecal Microbiota Transplantation (FMT) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Feasibility measured by number of participants able to ingest 15 FMT capsules over a 2-day period
Number of participants able to ingest 15 FMT capsules over a 2 day period.
Measured: 2 days
Secondary outcomes (5)
Progression Free Survival
Measured: 2 years
Overall Survival
Measured: 2 years
Cumulative Incidence Of aGVHD
Measured: 2 years
Non-Relapse Mortality Rate
Measured: 2 years
Number of participants with treatment-related adverse events
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Apr 2016 (estimated)
- Study startMay 2016
- Primary completionDec 2016
- Study completionDec 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Bone Marrow TransplantationStudies of the same condition, open and under way.
- More trials run by Massachusetts General HospitalThe sponsor's other registered studies.
- 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Massachusetts General Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Yi-Bin A. Chen, MD, Massachusetts General Hospital
- Investigators
- Yi-Bin Chen, MD, Principal investigator, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02733744
- Sponsor's protocol ID
- 16-023
- Status last verified
- Dec 2017
- First submitted
- 30 Mar 2016
- Last updated
- 22 Dec 2017
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
DeFilipp Z, Peled JU, Li S, Mahabamunuge J, Dagher Z, Slingerland AE, Del Rio C, Valles B, Kempner ME, Smith M, Brown J, Dey BR, El-Jawahri A, McAfee SL, Spitzer TR, Ballen KK, Sung AD, Dalton TE, Messina JA, Dettmer K, Liebisch G, Oefner P, Taur Y, Pamer EG, Holler E, Mansour MK, van den Brink MRM, Hohmann E, Jenq RR, Chen YB. Third-party fecal microbiota transplantation following allo-HCT reconstitutes microbiome diversity. Blood Adv. 2018 Apr 10;2(7):745-753. doi: 10.1182/bloodadvances.2018017731.
PubMed 29592876
Study at a glance
From the registry record, last updated by the sponsor on 22 Dec 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study to Determine the Feasibility of Fecal MicrobiotaTransplantation (FMT) in Hematopoietic Stem Cell Transplantation (HSCT) Recipients
- ClinicalTrials.gov ID
- NCT02733744
- Acronym / sponsor ID
- 16-023
- Conditions
- Bone Marrow Transplantation
- Keywords
- Stem cell transplant, fecal microbiota transplantation
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Massachusetts General HospitalUniversity, hospital or other
- Study start
- May 2016
- Primary completion
- Dec 2016 (actual)
- Study completion
- Dec 2017 (actual)
- First posted
- 11 Apr 2016 (estimated)
- 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.
This research study is studying an intervention called fecal microbiota transplantation (FMT). Patients who are scheduled to undergo Hematopoietic Stem Cell Transplantation are invited to take part in this clinical trial to undergo empiric FMT soon after hematopoietic engraftment. The primary endpoint will be to assess the feasibility of FMT in this population and to assess safety.
Detailed description
The microbiome (spectrum of bacteria in a patient's gut) is thought to play a role in helping to shape one's immune system given its direct contact with normal cells in our intestine. Recent studies have suggested that a microbiome with very few bacteria (low diversity) seems to be bad for health and a high-diversity microbiome (many different species) appears to be good. This appears to be true even for patients after HSCT where low diversity microbiome status has correlated with infections, GVHD and overall survival.
Fecal microbiota transplantation (FMT) is routinely performed for an infection caused by Clostridium difficile but is not yet approved by the FDA as a treatment for any disease. FMT restores a high diversity microbiome. It is hoped that through FMT, overall outcomes after HSCT can potentially be improved.
FMT will be performed in the first 3 weeks after recovery of white blood cells after HSCT. The source of FMT will be from healthy 3rd party donors. FMT will be performed through the ingestion of 30 capsules over 2 consecutive days.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Fecal Microbiota Transplant | Experimental | Biological: Fecal Microbiota Transplantation (FMT)Each participant will undergo allogeneic hematopoietic stem cell transplantation, according to institutional standards. Participants will receive a single standard dose of oral Fecal Microbiota Transplantation (FMT), which is 15 capsules per day for two consecutive days, for a total of 30 capsules. Participants will be asked to fast for 4 hours prior to and 1 hour following capsule intake. Capsules will be individually handed to participants by a research nurse or physician. Each capsule will be taken with a sip of water. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- Men or women ≥ 18 and ≤ 65 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 Consensus Criteria (Bacigalupo 2009) as well as the combination of fludarabine with melphalan (100-140 mg/mg2)
- Any Graft-vs-Host disease (GVHD) prophylaxis regimen is allowed.
- Eastern Cooperative Oncology Group (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.
- Patients with history of primary idiopathic myelofibrosis or any severe marrow fibrosis.
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)Also: FMTFMT will be given through the ingestion of 30 capsules (15 capsules daily x 2 consecutive days) | Biological | Fecal Microbiota Transplant |
Primary outcome
Feasibility measured by number of participants able to ingest 15 FMT capsules over a 2-day period
Time frame: 2 days
Number of participants able to ingest 15 FMT capsules over a 2 day period.
5 secondary outcomes
Progression Free Survival
Time frame: 2 years
Overall Survival
Time frame: 2 years
Cumulative Incidence Of aGVHD
Time frame: 2 years
Non-Relapse Mortality Rate
Time frame: 2 years
Number of participants with treatment-related adverse events
Time frame: 1 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Dec 2017.
- Derived from this study: PubMed 29592876DeFilipp Z, Peled JU, Li S, Mahabamunuge J, Dagher Z, Slingerland AE, Del Rio C, Valles B, Kempner ME, Smith M, Brown J, Dey BR, El-Jawahri A, McAfee SL, Spitzer TR, Ballen KK, Sung AD, Dalton TE, Messina JA, Dettmer K, Liebisch G, Oefner P, Taur Y, Pamer EG, Holler E, Mansour MK, van den Brink MRM, Hohmann E, Jenq RR, Chen YB. Third-party fecal microbiota transplantation following allo-HCT reconstitutes microbiome diversity. Blood Adv. 2018 Apr 10;2(7):745-753. doi: 10.1182/bloodadvances.2018017731.
- 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.
Yi-Bin Chen, 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 Early Phase 1 study testing Fecal Microbiota Transplantation (FMT) in people with Bone Marrow Transplantation, run by Massachusetts General Hospital 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: finished, as of the sponsor's last update on 22 Dec 2017. 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. 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Feasibility measured by number of participants able to ingest 15 FMT capsules over a 2-day period (time frame: 2 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02733744, which also lists 1 linked publication.
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 22 Dec 2017.