Gastro-Intestinal Acute Graft Versus Host Disease (GI-aGVHD)Phase 2NCT04059757
Fecal Microbiota Transplantation For The Treatment Of Gastro-Intestinal Acute GVHD
A Phase 2 study testing Fecal Microbiota Transplantation (FMT) in people with Gastro-Intestinal Acute Graft Versus Host Disease (GI-aGVHD), run by Leland Metheny at 1 site in United States.
Trial summary
Gastro-Intestinal Acute Graft Versus Host Disease (GI-aGVHD) is a complication of allogeneic stem cell transplant which is usually treated with steroids. You are being asked to take part in this study because you have recently been diagnosed with GI-GVHD. The standard of care for GI-aGVHD is steroids. Read more about this study
- Who is it for?
- Ages 19 to 74 years
- Conditions the study is for
- Gastro-Intestinal Acute Graft Versus Host Disease (GI-aGVHD)
- 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
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- May 2022to Sep 2023
- Phase
- Phase 2
- 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 19 to 74 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- One of the following diagnosis:
- High risk aGVHD (either biopsy proven or clinical diagnosed) (see Appendix B \& C) as defined by either:
- Lower gastrointestinal (GI) stage 3+
- Hyper-acute GVHD as defined by aGVHD of the GI tract within the first 14 days of transplant AND
- Subjects with treatment-naive acute GVHD as defined as those who have not received previous systemic treatment for acute GVHD, except for a maximum of 7 days of no less than 1 mg/kg/day of methyl-prednisolone (or equivalent dose of prednisone).
- OR:
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- Steroid refractory aGVHD of the GI tract (either biopsy proven or clinical diagnosed) as defined by:
- no response to steroid treatment (minimum daily dose: 2 mg/kg methyl-prednisolone or equivalent) lasting at least 7 days, or
- progression of at least one grade within the first 72 h of treatment
- ECOG Performance status < 3
- Patients who underwent an allogeneic hematopoietic stem cell transplantation from any donor source.
- Patients who are able stop prophylactic antibiotics during the treatment period
- Subjects must have the ability to understand and the willingness to sign a written informed consent document.
Who cannot join
- Active malignancy
- Patients with any concurrent uncontrolled clinically significant medical condition including active infection, laboratory abnormality, or psychiatric illness which could place the patient at unacceptable risk of study treatment.
- Pregnant or breastfeeding women
- Any condition that would, in the investigator's judgment, interfere with full participation in the study, including administration of study drug and attending required study visits; pose a significant risk to the subject; or interfere with interpretation of study data.
- Known allergies, hypersensitivity, or intolerance to any of the study medications, excipients, or similar compounds
- Patients with any severe gastrointestinal condition other than GI-GVHD.
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- Inability (e.g. dysphagia) to or unwilling to swallow capsules
- Active gastrointestinal infection at time of enrollment
- Known or suspected toxic megacolon and/or known small bowel ileus
- Major gastrointestinal surgery (e.g. significant bowel resection) within 3 months before enrollment. This does not include appendectomy or cholecystectomy
- History of total colectomy or bariatric surgery
- Concurrent intensive induction chemotherapy, radiation therapy or biological treatment for active malignancy
- Unable or unwilling to comply with protocol requirements
- Expected life expectancy < 6 months
- Patients who have CMV >2,000 copies/mL of whole blood or EBV >2,000 copies/mL of whole blood.
Trial locations and contacts
1 site in 1 country.
United States1 site
Case Medical Center, University Hospitals Seidman Cancer Center, Case Comprehensive Cancer Center
Cleveland, Ohio, 44106-5065
About this study
Official title: Fecal Microbiota Transplantation For The Treatment Of Gastro-Intestinal Acute Graft Versus Host Disease
Gastro-Intestinal Acute Graft Versus Host Disease (GI-aGVHD) is a complication of allogeneic stem cell transplant which is usually treated with steroids. You are being asked to take part in this study because you have recently been diagnosed with GI-GVHD. The standard of care for GI-aGVHD is steroids. When aGVHD does not respond to steroids it is described as steroid-refractory aGVHD. There is no standard therapy for steroid-refractory GI-aGVHD.
This study is a Phase II study. The main goal of a Phase II study is to see the efficacy and what side effects are seen with FMT as a treatment for GVHD.
Read more
Fecal Microbiota Transplantation (FMT) is the transfer of fecal material from a healthy donor to a patient in order to restore the diversity of the intestinal microbiota. FMT is currently indicated for the treatment of recurrent Clostridium Difficile infection.
FMT is considered experimental in this study, meaning it is not approved by the FDA for the treatment of GVHD.
- Being tested
- Fecal Microbiota Transplantation (FMT)
- Keywords
- Graft Versus Host Disease (GVHD)
- 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 Transplantation (FMT)Biological
1 dose = 30 capsules on day 1 of Fecal Microbiota Transplantation
| Study group | Role | What they receive |
|---|---|---|
| Fecal Microbiota Transplantation (FMT) | Experimental | One dose of FMT equal to 30 capsules will be administered on day 1 of a 28 day cycle. Steroids and routine GVHD prophylaxis medications and antibiotics may be administered concurrently with FMT therapy.
Participants will be followed for 28 days following completion of the FMT dose or protocol defined outcome.
aGVHD will be treated as per standard of care. Receives: Biological: Fecal Microbiota Transplantation (FMT) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants experiencing toxicity
Toxicity is defined as:
- Any bacterial or fungal infection that can be definitely attributed to FMT.
- Any grade 3 or more adverse event that occurs during or immediately after receiving the treatment and is definitely attributed to FMT.
Measured: up to 6 months from start of treatment
Efficacy of FMT therapy in high risk and in steroid refractory GI-aGVHD as defined as number of responses at day 28 (+/- 3 days) post FMT treatment
Response will be determined from the maximum GI-aGVHD stage and grade at day 28 (+/- 3 days) post FMT treatment. Response will be determined by P.I and a second physician.
- Complete response (CR) is defined as the complete resolution of GI aGVHD symptoms, without secondary GVHD therapy.
- Partial response (PR) is defined as improvement without complete resolution and without worsening of GI aGVHD, without secondary aGVHD therapy.
- No response (NR) is defined as the same grade of GVHD, progression, death, or the addition of secondary GVHD therapy.
- Progression is defined as worsening GI aGVHD.
Measured: 28 days (+/- 3 days) post FMT treatment
Secondary outcomes (6)
Non-relapse mortality (NRM) as measured by percentage of participants who die not related to relapse
Non-relapse mortality (NRM) as measured by percentage of participants who die not related to relapse
Measured: up to 6 months from start of treatment
Relapse as measured by percentage of participants who relapse
Relapse as measured by percentage of participants who relapse
Measured: up to 6 months from start of treatment
Relapse-related mortality as measured by percentage of participants with death related to relapse
Relapse-related mortality as measured by percentage of participants with death related to relapse.
Measured: up to 6 months from start of treatment
Percentage of participants who develop cGVHD by the end of trial
Percentage of participants who develop cGVHD by the end of trial
Measured: up to 6 months from start of treatment
Overall survival (OS) as measured by percentage of participants who are alive at end of trial
Overall survival (OS) as measured by percentage of participants who are alive at end of trial
Measured: up to 6 months from start of treatment
Percentage of patients who discontinue steroids at the end of the study
Percentage of patients who discontinue steroids at the end of the study.
Measured: up to 6 months from start of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Aug 2019
- Study startMay 2022 (estimated)
- Primary completionJun 2023 (estimated)
- Study completionSep 2023 (estimated)
Why it stopped: Drugs unavailable
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gastro-Intestinal Acute Graft Versus Host Disease (GI-aGVHD)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
- Not applicable
- Intervention model
- Single group
- Model description
- The study follows R. Simon's minimax two-stage phase II design with type I error of 0.05 and power of 80%. Ten participants will be enrolled on stage one. If there are 2 or less responses, the trial will be stopped and the treatment will be considered less than 60% effective for this patient population. If there are 3 or more responses, 7 additional patients will be enrolled.
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Leland Metheny
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Leland Metheny, Case Comprehensive Cancer Center
- Investigators
- Leland Metheny, MD, Principal investigator, Case Medical Center, University Hospitals Seidman Cancer Center, Case Comprehensive Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04059757
- Sponsor's protocol ID
- CASE4Z19
- Status last verified
- Jul 2022
- First submitted
- 7 Aug 2019
- Last updated
- 7 Jul 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Individual participant data that underline or influence the results observed from the study.
Study at a glance
From the registry record, last updated by the sponsor on 7 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Fecal Microbiota Transplantation For The Treatment Of Gastro-Intestinal Acute Graft Versus Host Disease
- ClinicalTrials.gov ID
- NCT04059757
- Acronym / sponsor ID
- CASE4Z19
- Keywords
- Graft Versus Host Disease (GVHD)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Drugs unavailable
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Leland MethenyUniversity, hospital or other
- Study start
- May 2022 (estimated)
- Primary completion
- Jun 2023 (estimated)
- Study completion
- Sep 2023 (estimated)
- First posted
- 16 Aug 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.
Gastro-Intestinal Acute Graft Versus Host Disease (GI-aGVHD) is a complication of allogeneic stem cell transplant which is usually treated with steroids. You are being asked to take part in this study because you have recently been diagnosed with GI-GVHD. The standard of care for GI-aGVHD is steroids. When aGVHD does not respond to steroids it is described as steroid-refractory aGVHD. There is no standard therapy for steroid-refractory GI-aGVHD.
This study is a Phase II study. The main goal of a Phase II study is to see the efficacy and what side effects are seen with FMT as a treatment for GVHD.
Fecal Microbiota Transplantation (FMT) is the transfer of fecal material from a healthy donor to a patient in order to restore the diversity of the intestinal microbiota. FMT is currently indicated for the treatment of recurrent Clostridium Difficile infection.
FMT is considered experimental in this study, meaning it is not approved by the FDA for the treatment of GVHD.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- The study follows R. Simon's minimax two-stage phase II design with type I error of 0.05 and power of 80%. Ten participants will be enrolled on stage one. If there are 2 or less responses, the trial will be stopped and the treatment will be considered less than 60% effective for this patient population. If there are 3 or more responses, 7 additional patients will be enrolled.
| Arm | Type | Receives |
|---|---|---|
| Fecal Microbiota Transplantation (FMT) | Experimental | Biological: Fecal Microbiota Transplantation (FMT)One dose of FMT equal to 30 capsules will be administered on day 1 of a 28 day cycle. Steroids and routine GVHD prophylaxis medications and antibiotics may be administered concurrently with FMT therapy. Participants will be followed for 28 days following completion of the FMT dose or protocol defined outcome. aGVHD will be treated as per standard of care. |
Who may qualify
- Ages
- 19 to 74 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- One of the following diagnosis:
- High risk aGVHD (either biopsy proven or clinical diagnosed) (see Appendix B \& C) as defined by either:
- Steroid refractory aGVHD of the GI tract (either biopsy proven or clinical diagnosed) as defined by:
- ECOG Performance status < 3
- Patients who underwent an allogeneic hematopoietic stem cell transplantation from any donor source.
Key exclusion criteria (15 listed)
- Active malignancy
- Patients with any concurrent uncontrolled clinically significant medical condition including active infection, laboratory abnormality, or psychiatric illness which could place the patient at unacceptable risk of study treatment.
- Pregnant or breastfeeding women
- Any condition that would, in the investigator's judgment, interfere with full participation in the study, including administration of study drug and attending required study visits; pose a significant risk to the subject; or interfere with interpretation of study data.
- Known allergies, hypersensitivity, or intolerance to any of the study medications, excipients, or similar compounds
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)1 dose = 30 capsules on day 1 of Fecal Microbiota Transplantation | Biological | Fecal Microbiota Transplantation (FMT) |
Primary outcome
Number of participants experiencing toxicity
Time frame: up to 6 months from start of treatment
Toxicity is defined as: * Any bacterial or fungal infection that can be definitely attributed to FMT. * Any grade 3 or more adverse event that occurs during or immediately after receiving the treatment and is definitely attributed to FMT.
Efficacy of FMT therapy in high risk and in steroid refractory GI-aGVHD as defined as number of responses at day 28 (+/- 3 days) post FMT treatment
Time frame: 28 days (+/- 3 days) post FMT treatment
Response will be determined from the maximum GI-aGVHD stage and grade at day 28 (+/- 3 days) post FMT treatment. Response will be determined by P.I and a second physician. * Complete response (CR) is defined as the complete resolution of GI aGVHD symptoms, without secondary GVHD therapy. * Partial response (PR) is defined as improvement without complete resolution and without worsening of GI aGVHD, without secondary aGVHD therapy. * No response (NR) is defined as the same grade of GVHD, progression, death, or the addition of secondary GVHD therapy. * Progression is defined as worsening GI aGVHD.
6 secondary outcomes
Non-relapse mortality (NRM) as measured by percentage of participants who die not related to relapse
Time frame: up to 6 months from start of treatment
Relapse as measured by percentage of participants who relapse
Time frame: up to 6 months from start of treatment
Relapse-related mortality as measured by percentage of participants with death related to relapse
Time frame: up to 6 months from start of treatment
Percentage of participants who develop cGVHD by the end of trial
Time frame: up to 6 months from start of treatment
Overall survival (OS) as measured by percentage of participants who are alive at end of trial
Time frame: up to 6 months from start of treatment
Percentage of patients who discontinue steroids at the end of the study
Time frame: up to 6 months from start of treatment
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Jul 2022.
- Individual participant dataSharing planned: Yes. Individual participant data that underline or influence the results observed from the study.
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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.
Leland Metheny, MD principal investigator
Case Medical Center, University Hospitals Seidman Cancer Center, Case Comprehensive Cancer Center
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 Fecal Microbiota Transplantation (FMT) in people with Gastro-Intestinal Acute Graft Versus Host Disease (GI-aGVHD), run by Leland Metheny 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 13 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: withdrawn, as of the sponsor's last update on 7 Jul 2022. 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?
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?
The study was withdrawn before enrolling participants, so there are no results.
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?
Number of participants experiencing toxicity (time frame: up to 6 months from start of treatment). 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 7 Jul 2022.