Stem Cell Transplant ComplicationsPhase 1NCT03214289
Fecal Microbiota Transplantation for Steroid Resistant and Steroid Dependent Gut Acute Graft Versus Host Disease
A Phase 1 study testing Fecal Microbiota Transplantation in people with Stem Cell Transplant Complications and Graft Versus Host Disease, Acute, run by Sheba Medical Center at 1 site in Israel.
Trial summary
The investigators hypothesize that perturbations in the intestinal microbiota following allogeneic hematopoietic stem cell transplantation (HSCT) are essential for the development and propagation of acute graft-versus-host disease. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Stem Cell Transplant Complications, Graft Versus Host Disease, Acute, Fecal Microbiota Transplantation
- What is being tested?
- Fecal Microbiota Transplantation
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteIsrael
- Study dates
- 12 Jul 2017to 20 Dec 2019
- Phase
- 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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adults (ages 18 to 75 years) who underwent allogeneic hematopoietic stem cell transplantation (HSCT) and developed gut acute Graft-versus-Host Disease (aGvHD).
- Participants have steroid-resistant or steroid-dependent gut aGvHD.
- Steroid resistant gut aGvHD is defined as cases in which gastrointestinal symptoms do not improve within 7 days after initial steroid therapy (≥1 mg/kg of methylprednisolone) or clear progression after 5 days.)
- Steroid-dependent gut aGVHD is defined as cases in which reduction of steroid dose was not possible due to exacerbation of gastrointestinal symptoms.
- Participants may have undergone allogeneic HSCT for any diagnosis at any time prior to developing aGvHD, and are not restricted to any specific conditioning regimen or by the subsequent administration of donor lymphocyte infusion.
- Participants should be able to give informed consent.
Who cannot join
- Participants may not have gut aGvHD which permits the tapering of steroid dose.
- Participants may not have ongoing, uncontrolled infection (i.e. unresolved bacteremia, uncontrolled CMV infection).
- Participants may not have ongoing enteritis primarily caused by enteropathy other than gut GvHD, excluding resistant clostridium difficile infection.
- Participants may not have acute neutrophil count < 500 cells/µL.
- Participants may not have toxic megacolon
- Participants may not have active gastrointestinal bleeding.
- Participants may not be pregnant or lactating.
- Participants may not be unable to swallow pills.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Fecal Microbiota Transplantation for Treatment of Steroid Resistant and Steroid Dependent Gut Acute Graft Versus Host Disease- a Pilot Study
The investigators hypothesize that perturbations in the intestinal microbiota following allogeneic hematopoietic stem cell transplantation (HSCT) are essential for the development and propagation of acute graft-versus-host disease. Therefore, modification of HSCT recipients' gut microbiota using fecal transplantation from a healthy donor could be used to treat gut acute GVHD.
The study evaluates safety and feasibility of fecal microbiota transplantation with frozen capsules from healthy donors for the treatment of steroid resistant or steroid dependent acute graft-versus-host disease of the gut.
- Conditions
- Stem Cell Transplant Complications, Graft Versus Host Disease, Acute, Fecal Microbiota Transplantation
- Being tested
- Fecal Microbiota Transplantation
- Keywords
- Fecal Microbiota Transplantation, Intestinal Acute Graft Versus Host Disease, Hematopoietic Stem Cell Transplantation
- Medical subject headings
- Graft vs Host Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Fecal Microbiota TransplantationBiological
Thirty fecal microbiota capsules produced from a single healthy donor, unrelated to the participant.
| Study group | Role | What they receive |
|---|---|---|
| Fecal Microbiota Transplantation (FMT) | Experimental | Participants will receive a single dose of oral FMT, which is 15 capsules per day for 2 consecutive days (total of 30 capsules). All capsules administered to a participant are from the same unrelated donor. Participants will be asked to fast for 4 hours prior to and 1 hour following capsule intake. Participants will be asked to drink at least 360cc of water during administration.
Treatment will be administered on an inpatient basis. In patients with no/partial response, the FMT may be repeated from the same or a different donor.
Subjects receiving any amount of the FMT capsules will be followed for at least 6 months.Stool and blood samples will be serially collected. Receives: Biological: Fecal Microbiota Transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Serious adverse events
Participants will be evaluated for serious adverse events (SAEs) relating to FMT occurring within 28 days following transplantation. SAEs are defined as any adverse experience occurring during or after FMT that results in any of the following outcomes: death, life-threatening experience, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity or an important medical event events
Measured: 28 days following FMT.
Secondary outcomes (4)
Non-serious adverse events
Participants will be evaluated for non-serious adverse events relating to FMT within 28 days following transplantation. Non-serious adverse events are defined as diarrhea, nausea and vomiting, fatigue and malaise, headache, and distension/bloating/abdominal discomfort/pain
Measured: 28 days following FMT.
Gut acute Graft-versus-Host Disease (aGvHD) response.
Participants will be evaluated on days 7 and 28 following transplantation for response to therapy.
Response criteria for steroid-resistant patients is as follows:
- Complete response: resolution of all gastrointestinal signs and symptoms
- Partial response: decrease in severity of gut GVHD by at least one stage
- Progression: Progressive worsening of gut GVHD
- No change: No significant change in gut GVHD
Response criteria for steroid-dependent patients is as follows:
- Complete response: reduction of the steroid dose to 5 mg or less of prednisone (or a steroid equivalent) per day.
- Partial response: a reduction of ≥40% in the dose of steroid
- Progression: an increase of >=10% in the steroid dose
- No change: any other option
Measured: up to 28 days following FMT.
aGvHD severity
Participants will be evaluated on days 7 and 28 following transplantation for severity of aGvHD.
aGVHD severity is graded by the International Bone Marrow Transplant Registry Severity Index grading system
Measured: up to 28 days following FMT.
Reduction in the dose of steroids.
The dose of methylprednisolone, or an equivalent GCS, will be evaluated on day 7, and 28. The change in the dose at day 28, adjusted to the type of GCS used.
Measured: up to 28 days following FMT.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jul 2017
- Study start12 Jul 2017
- Primary completion20 Jul 2019 (estimated)
- Study completion20 Dec 2019 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Stem Cell Transplant ComplicationsStudies of the same condition, open and under way.
- More trials run by Sheba Medical CenterThe 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
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sheba Medical Center
- Sponsor type
- Other government
- Responsible party
- Principal investigator, Dr. Shouval Roni, Sheba Medical Center
- Investigators
- Roni Shouval, MD, Principal investigator, Sheba Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03214289
- Sponsor's protocol ID
- 3822-16-SMC
- Other IDs
- 3822-16-SMC
- Status last verified
- Apr 2018
- First submitted
- 29 Jun 2017
- Last updated
- 9 Apr 2018
- Results posted
- No
- Data monitoring committee
- No
- 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 9 Apr 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Fecal Microbiota Transplantation for Treatment of Steroid Resistant and Steroid Dependent Gut Acute Graft Versus Host Disease- a Pilot Study
- ClinicalTrials.gov ID
- NCT03214289
- Acronym / sponsor ID
- 3822-16-SMC
- Conditions
- Stem Cell Transplant Complications, Graft Versus Host Disease, Acute, Fecal Microbiota Transplantation
- Keywords
- Fecal Microbiota Transplantation, Intestinal Acute Graft Versus Host Disease, Hematopoietic Stem Cell Transplantation
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 4 participants (estimated)
- Lead sponsor
- Sheba Medical CenterOther government
- Study start
- 12 Jul 2017 (actual)
- Primary completion
- 20 Jul 2019 (estimated)
- Study completion
- 20 Dec 2019 (estimated)
- First posted
- 11 Jul 2017 (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.
The investigators hypothesize that perturbations in the intestinal microbiota following allogeneic hematopoietic stem cell transplantation (HSCT) are essential for the development and propagation of acute graft-versus-host disease. Therefore, modification of HSCT recipients' gut microbiota using fecal transplantation from a healthy donor could be used to treat gut acute GVHD.
The study evaluates safety and feasibility of fecal microbiota transplantation with frozen capsules from healthy donors for the treatment of steroid resistant or steroid dependent acute graft-versus-host disease of the gut.
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 Transplantation (FMT) | Experimental | Biological: Fecal Microbiota TransplantationParticipants will receive a single dose of oral FMT, which is 15 capsules per day for 2 consecutive days (total of 30 capsules). All capsules administered to a participant are from the same unrelated donor. Participants will be asked to fast for 4 hours prior to and 1 hour following capsule intake. Participants will be asked to drink at least 360cc of water during administration. Treatment will be administered on an inpatient basis. In patients with no/partial response, the FMT may be repeated from the same or a different donor. Subjects receiving any amount of the FMT capsules will be followed for at least 6 months.Stool and blood samples will be serially collected. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Adults (ages 18 to 75 years) who underwent allogeneic hematopoietic stem cell transplantation (HSCT) and developed gut acute Graft-versus-Host Disease (aGvHD).
- Participants have steroid-resistant or steroid-dependent gut aGvHD.
- Steroid resistant gut aGvHD is defined as cases in which gastrointestinal symptoms do not improve within 7 days after initial steroid therapy (≥1 mg/kg of methylprednisolone) or clear progression after 5 days.)
- Steroid-dependent gut aGVHD is defined as cases in which reduction of steroid dose was not possible due to exacerbation of gastrointestinal symptoms.
- Participants may have undergone allogeneic HSCT for any diagnosis at any time prior to developing aGvHD, and are not restricted to any specific conditioning regimen or by the subsequent administration of donor lymphocyte infusion.
Key exclusion criteria (8 listed)
- Participants may not have gut aGvHD which permits the tapering of steroid dose.
- Participants may not have ongoing, uncontrolled infection (i.e. unresolved bacteremia, uncontrolled CMV infection).
- Participants may not have ongoing enteritis primarily caused by enteropathy other than gut GvHD, excluding resistant clostridium difficile infection.
- Participants may not have acute neutrophil count < 500 cells/µL.
- Participants may not have toxic megacolon
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 TransplantationThirty fecal microbiota capsules produced from a single healthy donor, unrelated to the participant. | Biological | Fecal Microbiota Transplantation (FMT) |
Primary outcome
Serious adverse events
Time frame: 28 days following FMT.
Participants will be evaluated for serious adverse events (SAEs) relating to FMT occurring within 28 days following transplantation. SAEs are defined as any adverse experience occurring during or after FMT that results in any of the following outcomes: death, life-threatening experience, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity or an important medical event events
4 secondary outcomes
Non-serious adverse events
Time frame: 28 days following FMT.
Gut acute Graft-versus-Host Disease (aGvHD) response.
Time frame: up to 28 days following FMT.
aGvHD severity
Time frame: up to 28 days following FMT.
Reduction in the dose of steroids.
Time frame: up to 28 days following FMT.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Apr 2018.
- Individual participant dataSharing planned: No
Related trials for Stem Cell Transplant Complications
Active, not recruitingPhase 2
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Mucopolysaccharidosis Disorders, Hurler Syndrome
Active, not recruitingObservational study
Vaginal Microbiome and the Development of Vulvovaginal Graft Versus Host Disease
Bone Marrow Transplant Complications
Not yet recruitingPhase 1
Expanded Donor Stem Cells for Poor Graft Function After Stem Cell Transplantation in Children
Poor Graft Function, Hematopoietic Stem Cell Transplantation Complications
Active, not recruiting
Impact of Exercise on the Complications of Corticosteroids in Patients With GVHD: the RESTART Trial
Graft Vs Host Disease, Acute-graft-versus-host Disease
Not yet recruitingObservational study
Assessing the Incidence of Transplant Associated Thrombotic Microangiopathy (TA-TMA) in Adult Patients Undergoing Allogeneic Stem Cell Transplant (SCT)
Transplant Associated Microangiopathy TAM, Transplant Complication
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.
Roni Shouval, MD principal investigator
Sheba Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Fecal Microbiota Transplantation in people with Stem Cell Transplant Complications and Graft Versus Host Disease, Acute, run by Sheba Medical Center at 1 site in Israel.
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 6 inclusion and 8 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: status not confirmed, as of the sponsor's last update on 9 Apr 2018. 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 Israel. 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?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Contact Roni Shouval, MD (972-3-5305830, roni.shoval@sheba.health.gov.il), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Serious adverse events (time frame: 28 days following FMT.). 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 9 Apr 2018.