Fecal Microbiota Transplantation in GVHDPhase 1NCT03549676
Fecal Microbiota Transplantation for Treatment of Refractory Graft Versus Host Disease-a Pilot Study
A Phase 1 study testing Fecal Microbiota Transplantation in people with Fecal Microbiota Transplantation in GVHD, run by Shanghai Children's Medical Center.
Trial summary
The study evaluates safety and efficacy of fecal microbiota transplantation (FMT) for the treatment of refractory graft-versus-host-disease (GVHD) of the gut. FMT might be a beneficial treatment in this clinical situation with a poor prognosis and limited therapeutic options. Read more about this study
- Who is it for?
- Ages 3 to 18 years
- Conditions the study is for
- Fecal Microbiota Transplantation in GVHD
- What is being tested?
- Fecal Microbiota Transplantation
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- No sites listed yet
- Study dates
- 1 Jul 2019to 31 Dec 2020
- 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 3 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 3-18 years of age
- Allo-SCT patients with acute steroid-resistant GI-related GVHD grade III-IV. Steroid-resistant GI-related GVHD will be defined as lack of improvement (same stage) or worsening of GI symptoms after 7 days of steroid therapy (≥ 2 ml/kg of IV methylprednisolone)
- No definite contraindication for gastrointestinal endoscopy
- Signature of informed consent by the legal guardians of patients
Who cannot join
- Prior inclusion to an interventional study
- Previous Allo-SCT
- Known multi-drug resistance carriage prior to stool collection
- Severe colitis of any etiology or a history of inflammatory bowel disease (IBD)
- Uncontrolled infection (hemodynamic instability, ongoing high fever or bacteremia within 3 days after antibiotics administration)
- Active GI bleeding
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- Absolute neutrophil count < 500 cells/microL
- Absolute platelet count< 10 x 109 /L
- Patients who cannot give informed consent
Trial locations and contacts
No sites are listed yet.
About this study
The study evaluates safety and efficacy of fecal microbiota transplantation (FMT) for the treatment of refractory graft-versus-host-disease (GVHD) of the gut. FMT might be a beneficial treatment in this clinical situation with a poor prognosis and limited therapeutic options.
Read the detailed description
Graft-versus-host-disease (GVHD) is a major complication after hematopoietic stem cell transplantation (HSCT). Gut is the most vulnerable target organ of acute GVHD. Patients who have a gastrointestinal acute GVHD received a first-line standard treatment of corticosteroids. For patients who do not respond or progress after an initial response have a high mortality. Therefore, the investigation of effective second line therapy for these patients are in need. The study evaluates safety and efficacy of fecal microbiota transplantation (FMT) for the treatment of refractory GVHD of the gut. FMT might be a beneficial treatment in this clinical situation with a poor prognosis and limited therapeutic options.
- Conditions
- Fecal Microbiota Transplantation in GVHD
- Being tested
- Fecal Microbiota Transplantation
- Keywords
- FMT, GVHD, HSCT
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Fecal Microbiota TransplantationBiological
For patients who do not respond or partial respond for first time FMT treatment, a second time FMT treatment using different donor should be considered.
| Study group | Role | What they receive |
|---|---|---|
| HSCT patients with refractory GVHD | Experimental | Patients will accept FilmArray Gastrointestinal (GI) panel test before pre-treatment of HSCT and 28±3 days post-HSCT. Patients will receive 50ml fecal microbiota from unrelated healthy donors through nasojejunal tube and monitored under gastroscopy. Patients receiving FMT treatment will be followed for at least 6 months. The ideal follow up time is 2 year. Stool and blood samples will be serially collected and tested (before pre-treatment, 1/3/6/12 months after FMT). Receives: Biological: Fecal Microbiota Transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficacy of FMT in the treatment of refractory GVHD on day 7
Participants will be evaluated on days 7 following FMT. The volume and frequency of daily diarrhoea will be continuous observed and recorded in 7 days.
Measured: 7 days following FMT
Efficacy of FMT in the treatment of refractory GVHD on day 28
Participants will be evaluated on days 28 following FMT. The volume and frequency of daily diarrhoea will be continuous observed and recorded in 28 days.
Measured: 28 days following FMT
Secondary outcomes (7)
Treatment-related AEs and SAEs
The overall safety of the study will be evaluated with the incidence of all Adverse Events (AEs) and Serious Adverse Events (SAEs) within 28 days following FMT. The relationship of any kind AEs and FMT will be seriously evaluated. Non-serious AEs include: dyspepsia, abdominal pain, nausea, vomiting, diarrhea, constipation, fever, inhalation without mechanical ventilation, etc. SAEs include: death, sepsis, aspiration pneumonia, gastrointestinal hemorrhage, septic shock, etc.
Measured: up to 28 days following FMT
GVHD severity
Patients will be evaluated on days 7 and 28 following transplantation for severity of GVHD. GVHD severity is graded by the International Bone Marrow Transplant Registry Severity Index grading system.
Measured: up to 28 days following FMT
Implantation rate
The data will be compared with patients who had not participated in clinical trials.
Measured: through study completion, an average of 6 months
Survival rate
The data will be compared with patients who had not participated in clinical trials.
Measured: through study completion, an average of 6 months
Change in biomarkers
Change in levels of albumin and C-reactive protein between days 0 and days 28 will serve as a secondary endpoint.
Measured: up to 28 days following FMT
Number of patients with infectious disorders
Evaluation of FMT activity on infectious disorder.
Measured: through study completion, an average of 6 months
Quality of Life
The quality of life of patients will be measured using a standardized quality of life questionnaire (EORTC QLQ-C30) prior to transplantation and on days 7, 14, 21, 28 and 180 following transplantation.
Measured: up to 6 months following FMT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jun 2018
- Study start1 Jul 2019 (estimated)
- Primary completion31 May 2020 (estimated)
- Study completion31 Dec 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fecal Microbiota Transplantation in GVHDStudies 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shanghai Children's Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Cao Qing, Shanghai Children's Medical Center
- Investigators
- Qing Cao, MD, Study director, Shanghai Children's Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03549676
- Sponsor's protocol ID
- FMT-GVHD
- Status last verified
- Mar 2019
- First submitted
- 15 May 2018
- Last updated
- 26 Mar 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 26 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Fecal Microbiota Transplantation for Treatment of Refractory Graft Versus Host Disease-a Pilot Study
- ClinicalTrials.gov ID
- NCT03549676
- Acronym / sponsor ID
- FMT-GVHD
- Conditions
- Fecal Microbiota Transplantation in GVHD
- Keywords
- FMT, GVHD, HSCT
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Shanghai Children's Medical CenterUniversity, hospital or other
- Study start
- 1 Jul 2019 (estimated)
- Primary completion
- 31 May 2020 (estimated)
- Study completion
- 31 Dec 2020 (estimated)
- First posted
- 8 Jun 2018 (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 study evaluates safety and efficacy of fecal microbiota transplantation (FMT) for the treatment of refractory graft-versus-host-disease (GVHD) of the gut. FMT might be a beneficial treatment in this clinical situation with a poor prognosis and limited therapeutic options.
Detailed description
Graft-versus-host-disease (GVHD) is a major complication after hematopoietic stem cell transplantation (HSCT). Gut is the most vulnerable target organ of acute GVHD. Patients who have a gastrointestinal acute GVHD received a first-line standard treatment of corticosteroids. For patients who do not respond or progress after an initial response have a high mortality. Therefore, the investigation of effective second line therapy for these patients are in need. The study evaluates safety and efficacy of fecal microbiota transplantation (FMT) for the treatment of refractory GVHD of the gut. FMT might be a beneficial treatment in this clinical situation with a poor prognosis and limited therapeutic options.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| HSCT patients with refractory GVHD | Experimental | Biological: Fecal Microbiota TransplantationPatients will accept FilmArray Gastrointestinal (GI) panel test before pre-treatment of HSCT and 28±3 days post-HSCT. Patients will receive 50ml fecal microbiota from unrelated healthy donors through nasojejunal tube and monitored under gastroscopy. Patients receiving FMT treatment will be followed for at least 6 months. The ideal follow up time is 2 year. Stool and blood samples will be serially collected and tested (before pre-treatment, 1/3/6/12 months after FMT). |
Who may qualify
- Ages
- 3 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- 3-18 years of age
- Allo-SCT patients with acute steroid-resistant GI-related GVHD grade III-IV. Steroid-resistant GI-related GVHD will be defined as lack of improvement (same stage) or worsening of GI symptoms after 7 days of steroid therapy (≥ 2 ml/kg of IV methylprednisolone)
- No definite contraindication for gastrointestinal endoscopy
- Signature of informed consent by the legal guardians of patients
Key exclusion criteria (9 listed)
- Prior inclusion to an interventional study
- Previous Allo-SCT
- Known multi-drug resistance carriage prior to stool collection
- Severe colitis of any etiology or a history of inflammatory bowel disease (IBD)
- Uncontrolled infection (hemodynamic instability, ongoing high fever or bacteremia within 3 days after antibiotics administration)
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 TransplantationFor patients who do not respond or partial respond for first time FMT treatment, a second time FMT treatment using different donor should be considered. | Biological | HSCT patients with refractory GVHD |
Primary outcome
Efficacy of FMT in the treatment of refractory GVHD on day 7
Time frame: 7 days following FMT
Participants will be evaluated on days 7 following FMT. The volume and frequency of daily diarrhoea will be continuous observed and recorded in 7 days.
Efficacy of FMT in the treatment of refractory GVHD on day 28
Time frame: 28 days following FMT
Participants will be evaluated on days 28 following FMT. The volume and frequency of daily diarrhoea will be continuous observed and recorded in 28 days.
7 secondary outcomes
Treatment-related AEs and SAEs
Time frame: up to 28 days following FMT
GVHD severity
Time frame: up to 28 days following FMT
Implantation rate
Time frame: through study completion, an average of 6 months
Survival rate
Time frame: through study completion, an average of 6 months
Change in biomarkers
Time frame: up to 28 days following FMT
Number of patients with infectious disorders
Time frame: through study completion, an average of 6 months
Quality of Life
Time frame: up to 6 months following FMT
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Mar 2019.
- Individual participant dataSharing planned: Undecided
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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.
Qing Cao, MD study director
Shanghai Children's Medical Center
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Fecal Microbiota Transplantation in people with Fecal Microbiota Transplantation in GVHD, run by Shanghai Children's Medical Center.
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 4 inclusion and 9 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 26 Mar 2019. Status can change between updates, so confirm with the study contact before making plans.
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 Qing Cao, MD (+86 18930830511, caoqing@scmc.com.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Efficacy of FMT in the treatment of refractory GVHD on day 7 (time frame: 7 days following FMT). The study lists 2 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 26 Mar 2019.