Graft vs Host DiseasePhase 2NCT07364617
Fecal Microbiota Transplantation for Steroid-Refractory Acute GI GVHD
A Phase 2 study testing Fecal microbiota transplantation (FMT) in people with Graft vs Host Disease, run by Chang Gung Memorial Hospital at 1 site in Taiwan.
Trial summary
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an effective treatment for various hematologic diseases. However, one of the major challenges of allo-HSCT is the occurrence of graft-versus-host disease (GvHD), particularly acute gastrointestinal GvHD (GI-GvHD). Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Graft vs Host Disease
- What is being tested?
- Fecal microbiota transplantation (FMT)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 35 participants
- Where
- 1 siteTaiwan
- Study dates
- 29 Apr 2026to 28 Feb 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Stage II to IV steroid refractory acute GI-GvHD in allo-HSCT recipients
- Stage II to IV acute GI-GVHD subjects, having >1000 mL stool per day, diarrhea > 5 times/day, or abdominal cramping, bleeding or ileus, AND
- Resistant to a first-line therapy with corticosteroids (CS)
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- Lack of improvement after 5 days of treatment with CS at 2 mg/kg/d methylprednisolone or other CS with equivalent dose,
- Progression after 3 days of treatment with CS at 2 mg/kg/d methylprednisolone or other CS with equivalent dose.
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- Age ≥ 18 years old.
- Allo-HSCT with any type of donor, stem cell source, GvHD prophylaxis or conditioning regimen.
- Allow vancomycin-resistant enterococcus (VRE) colonization and asymptomatic cytomegalovirus (CMV) viremia, which is defined as a detectable CMV viral load in plasma but without tissue-invasive disease.
- Patients able to have a minimum of 12 hours discontinuation of systemic antibiotics in order to perform the allogeneic FMT (antiviral and antifungal agents are allowed)
- Signature of informed and written consent by the subject or by the subject's legally acceptable representative for patients under guardianship or trusteeship. Subject must understand and voluntarily sign an informed consent form prior to any study-related assessments/procedures being conducted.
Who cannot join
- Absolute neutrophil count < 500 cells/uL.
- Absolute platelet count < 30000 /uL which is not correctable by transfusion
- Hemodynamically unstable status with the following conditions: systolic blood pressure < 90 mm Hg, pulse oximeter oxygen saturation (SpO2) < 90%, PaO2 < 60 mm Hg, or respiratory rate > 22/minute.
- Uncontrolled and active infection from bacteria, virus, or fungus as determined by the investigators.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Taiwan1 site, 1 recruiting
- Recruiting
Chang Gung Memorial Hospital at Linkou
Taoyuan, 333423
Hsiao-Wen Kao, M.D., +886-3-3281200 ext. 2524, hsiaowen@cgmh.org.tw
About this study
Official title: Fecal Microbiota Transplantation for the Treatment of Steroid Refractory Acute Gastrointestinal Graft-Versus-Host Disease in Patients After Allogeneic Hematopoietic Stem Cell Transplantation
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an effective treatment for various hematologic diseases. However, one of the major challenges of allo-HSCT is the occurrence of graft-versus-host disease (GvHD), particularly acute gastrointestinal GvHD (GI-GvHD). GvHD occurs when donor T cells recognize the recipient's tissue as foreign and mount an immune attack against it. Acute GI-GvHD is a common complication following allo-HSCT and a significant cause of mortality. If the initial steroid treatment for acute GvHD fails, mortality rates can reach as high as 81%.
Recent studies have shown a strong association between reduced gut microbiota diversity and high mortality in patients with acute GI-GvHD, highlighting the critical role of the gut microbiome in regulating immune responses and maintaining intestinal homeostasis. Consequently, fecal microbiota transplantation (FMT) has emerged as a potential therapeutic strategy aimed at restoring a healthy gut microbiome and improving clinical outcomes in patients with acute GI-GvHD.
This study aims to evaluate the efficacy and safety of FMT in patients with steroid-refractory or steroid-resistant acute GI-GvHD. The findings of this research will contribute to establishing FMT as a potential and effective treatment option for managing severe acute GI-GvHD, thereby improving patient outcomes and reducing transplant-related mortality.
Read the detailed description
This is a prospective, single-arm, open-label, phase II clinical trial evaluating the safety and efficacy of fecal microbiota transplantation (FMT) in adult patients with steroid-refractory acute gastrointestinal graft-versus-host disease (acute GI-GvHD) following allogeneic hematopoietic stem cell transplantation (allo-HSCT). Eligible patients will receive 250 cc microbiota fluid prepared from healthy screened donors, delivered into the terminal ileum or cecum via ileocolonoscopy, or into the terminal duodenum via panendoscopy. The study aims to assess event-free survival, overall and complete response rates, survival outcomes, changes in steroid exposure, adverse events, and microbiological/immunological biomarkers. A total of 35 participants will be enrolled over a planned 2-year study period.
- Conditions
- Graft vs Host Disease
- Being tested
- Fecal microbiota transplantation (FMT)
- Keywords
- Acute Gastrointestinal Graft-versus-Host Disease, fecal microbiota transplantation, steroid-refractory
- 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
About 250 mL microbiota fluid, containing approximately 60 cm³ of stool materials; 6x10¹³ bacteria, will be delivered to the terminal ileum/cecum via ileocolonoscopy or to the duodenum via panendoscopy, with a second FMT given 7-21 days later and an optional third dose based on response.
| Study group | Role | What they receive |
|---|---|---|
| single arm | Experimental | Participants receive 250 mL microbiota fluid delivered to the terminal ileum/cecum via ileocolonoscopy or to the duodenum via panendoscopy, with a second FMT given 7-21 days later and an optional third dose based on response. Receives: Biological: Fecal microbiota transplantation (FMT) |
What is being measured
The primary outcome is the main question the study is designed to answer.
event-free survival
Measured: From first fecal microbiota transplantation to the first event or up to 6 months
overall response rate
at least one stage improvement in acute GI-GvHD
Measured: on day-28 after first FMT
complete response rate
improvement of acute GI-GvHD to stage 0
Measured: on day-28 after first FMT
Secondary outcomes (7)
Overall response rate
at least one stage improvement in acute GI-GvHD
Measured: on day-56 after first FMT
Complete response rate
improvement of acute GI-GvHD to stage 0
Measured: on day 56 after first FMT
Time to response
Measured: from the time of first FMT to the time of at least one stage improvement in acute GI-GvHD
Relapse rate
Relapse defined as at least one stage acute gastrointestinal GvHD progression.
Measured: at least two-year follow-up after first FMT
Overall survival
Measured: from the time of first FMT to the time of death.
Non-relapse mortality
death without prior acute gastrointestinal GvHD relapse
Measured: at least two-year follow-up after first FMT
Incidence of adverse events
adverse events include but not limited to infection, cytopenia, viral reactivation, gastrointestinal bleeding, bowel rupture
Measured: at least two-year follow-up after first FMT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jan 2026
- Study start29 Apr 2026
- Primary completion28 Feb 2028 (estimated)
- Study completion28 Feb 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Graft vs Host DiseaseStudies of the same condition, open and under way.
- More trials run by Chang Gung Memorial 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
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- This prospective, single-arm, open-label phase II study evaluates fecal microbiota transplantation (FMT) as treatment for adults with steroid-refractory stage II-IV acute gastrointestinal graft-versus-host disease (GI-GvHD) after allogeneic hematopoietic stem cell transplantation. Participants receive 250 mL microbiota fluid delivered to the terminal ileum/cecum via ileocolonoscopy or to the duodenum via panendoscopy, with a second FMT given 7-21 days later and an optional third dose based on response. The study assesses event-free survival, overall and complete response rates at Days 28 and 56, steroid reduction, relapse, survival, and safety including infections and procedure-related complications. Serial clinical, microbiologic, and immunologic evaluations are performed to characterize treatment effects and adverse events. Total planned enrollment is 35 patients.
- Masking
- None (open label)
- Enrolment
- 35 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chang Gung Memorial Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- National Taiwan University Hospital
- Responsible party
- Principal investigator, Hsiaowen Kao, Chang Gung Memorial Hospital
- Investigators
- Hsiao-Wen KAO, M.D., Principal investigator, Chang Gung Memorial Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07364617
- Sponsor's protocol ID
- 202401777A0
- Other IDs
- LK20250520001
- Status last verified
- May 2026
- First submitted
- 2 Dec 2025
- Last updated
- 22 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: The study team plans to share de-identified individual participant data (IPD), including clinical outcomes, laboratory results, and response assessments related to fecal microbiota transplantation for acute GI-GvHD. IPD will be made available after publication of the primary results and following completion of all planned analyses. Data will be shared in a de-identified, password-protected format and will be accessible upon reasonable request to the principal investigator through a formal data-sharing agreement specifying intended use, data protection measures, and prohibition of re-identification. Supporting documents (such as the protocol and statistical analysis plan) may also be available upon request. No public repository upload is currently planned.
Study at a glance
From the registry record, last updated by the sponsor on 22 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Fecal Microbiota Transplantation for the Treatment of Steroid Refractory Acute Gastrointestinal Graft-Versus-Host Disease in Patients After Allogeneic Hematopoietic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT07364617
- Acronym / sponsor ID
- 202401777A0
- Conditions
- Graft vs Host Disease
- Keywords
- Acute Gastrointestinal Graft-versus-Host Disease, fecal microbiota transplantation, steroid-refractory
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 35 participants (estimated)
- Lead sponsor
- Chang Gung Memorial HospitalUniversity, hospital or other
- Collaborators
- National Taiwan University Hospital
- Study start
- 29 Apr 2026 (actual)
- Primary completion
- 28 Feb 2028 (estimated)
- Study completion
- 28 Feb 2028 (estimated)
- First posted
- 23 Jan 2026 (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.
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an effective treatment for various hematologic diseases. However, one of the major challenges of allo-HSCT is the occurrence of graft-versus-host disease (GvHD), particularly acute gastrointestinal GvHD (GI-GvHD). GvHD occurs when donor T cells recognize the recipient's tissue as foreign and mount an immune attack against it. Acute GI-GvHD is a common complication following allo-HSCT and a significant cause of mortality. If the initial steroid treatment for acute GvHD fails, mortality rates can reach as high as 81%.
Recent studies have shown a strong association between reduced gut microbiota diversity and high mortality in patients with acute GI-GvHD, highlighting the critical role of the gut microbiome in regulating immune responses and maintaining intestinal homeostasis. Consequently, fecal microbiota transplantation (FMT) has emerged as a potential therapeutic strategy aimed at restoring a healthy gut microbiome and improving clinical outcomes in patients with acute GI-GvHD.
This study aims to evaluate the efficacy and safety of FMT in patients with steroid-refractory or steroid-resistant acute GI-GvHD. The findings of this research will contribute to establishing FMT as a potential and effective treatment option for managing severe acute GI-GvHD, thereby improving patient outcomes and reducing transplant-related mortality.
Detailed description
This is a prospective, single-arm, open-label, phase II clinical trial evaluating the safety and efficacy of fecal microbiota transplantation (FMT) in adult patients with steroid-refractory acute gastrointestinal graft-versus-host disease (acute GI-GvHD) following allogeneic hematopoietic stem cell transplantation (allo-HSCT). Eligible patients will receive 250 cc microbiota fluid prepared from healthy screened donors, delivered into the terminal ileum or cecum via ileocolonoscopy, or into the terminal duodenum via panendoscopy. The study aims to assess event-free survival, overall and complete response rates, survival outcomes, changes in steroid exposure, adverse events, and microbiological/immunological biomarkers. A total of 35 participants will be enrolled over a planned 2-year study period.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- This prospective, single-arm, open-label phase II study evaluates fecal microbiota transplantation (FMT) as treatment for adults with steroid-refractory stage II-IV acute gastrointestinal graft-versus-host disease (GI-GvHD) after allogeneic hematopoietic stem cell transplantation. Participants receive 250 mL microbiota fluid delivered to the terminal ileum/cecum via ileocolonoscopy or to the duodenum via panendoscopy, with a second FMT given 7-21 days later and an optional third dose based on response. The study assesses event-free survival, overall and complete response rates at Days 28 and 56, steroid reduction, relapse, survival, and safety including infections and procedure-related complications. Serial clinical, microbiologic, and immunologic evaluations are performed to characterize treatment effects and adverse events. Total planned enrollment is 35 patients.
| Arm | Type | Receives |
|---|---|---|
| single arm | Experimental | Biological: Fecal microbiota transplantation (FMT)Participants receive 250 mL microbiota fluid delivered to the terminal ileum/cecum via ileocolonoscopy or to the duodenum via panendoscopy, with a second FMT given 7-21 days later and an optional third dose based on response. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (11 listed)
- Stage II to IV steroid refractory acute GI-GvHD in allo-HSCT recipients
- <!-- -->
- Age ≥ 18 years old.
- Allo-HSCT with any type of donor, stem cell source, GvHD prophylaxis or conditioning regimen.
- Allow vancomycin-resistant enterococcus (VRE) colonization and asymptomatic cytomegalovirus (CMV) viremia, which is defined as a detectable CMV viral load in plasma but without tissue-invasive disease.
Key exclusion criteria (4 listed)
- Absolute neutrophil count < 500 cells/uL.
- Absolute platelet count < 30000 /uL which is not correctable by transfusion
- Hemodynamically unstable status with the following conditions: systolic blood pressure < 90 mm Hg, pulse oximeter oxygen saturation (SpO2) < 90%, PaO2 < 60 mm Hg, or respiratory rate > 22/minute.
- Uncontrolled and active infection from bacteria, virus, or fungus as determined by the investigators.
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)About 250 mL microbiota fluid, containing approximately 60 cm³ of stool materials; 6x10¹³ bacteria, will be delivered to the terminal ileum/cecum via ileocolonoscopy or to the duodenum via panendoscopy, with a second FMT given 7-21 days later and an optional third dose based on response. | Biological | single arm |
Primary outcome
event-free survival
Time frame: From first fecal microbiota transplantation to the first event or up to 6 months
overall response rate
Time frame: on day-28 after first FMT
at least one stage improvement in acute GI-GvHD
complete response rate
Time frame: on day-28 after first FMT
improvement of acute GI-GvHD to stage 0
7 secondary outcomes
Overall response rate
Time frame: on day-56 after first FMT
Complete response rate
Time frame: on day 56 after first FMT
Time to response
Time frame: from the time of first FMT to the time of at least one stage improvement in acute GI-GvHD
Relapse rate
Time frame: at least two-year follow-up after first FMT
Overall survival
Time frame: from the time of first FMT to the time of death.
Non-relapse mortality
Time frame: at least two-year follow-up after first FMT
Incidence of adverse events
Time frame: at least two-year follow-up after first FMT
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 May 2026.
- Individual participant dataSharing planned: Yes. The study team plans to share de-identified individual participant data (IPD), including clinical outcomes, laboratory results, and response assessments related to fecal microbiota transplantation for acute GI-GvHD. IPD will be made available after publication of the primary results and following completion of all planned analyses. Data will be shared in a de-identified, password-protected format and will be accessible upon reasonable request to the principal investigator through a formal data-sharing agreement specifying intended use, data protection measures, and prohibition of re-identification. Supporting documents (such as the protocol and statistical analysis plan) may also be available upon request. No public repository upload is currently planned.
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Graft vs Host Disease
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.
Hsiao-Wen KAO, M.D. principal investigator
Chang Gung Memorial 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 Fecal microbiota transplantation (FMT) in people with Graft vs Host Disease, run by Chang Gung Memorial Hospital at 1 site in Taiwan.
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 11 inclusion and 4 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: looking for participants now, as of the sponsor's last update on 22 May 2026. 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 Taiwan. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Hsiao-Wen Kao, M.D. (+886-3-3281200 ext. 2524, hsiaowen@cgmh.org.tw), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
event-free survival (time frame: From first fecal microbiota transplantation to the first event or up to 6 months). The study lists 3 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 22 May 2026.