Acute Graft Versus Host Disease in IntestinePhase 1/2NCT07602751
FMT for Pediatric SR-aGVHD
A Phase 1/2 study testing Fecal Microbial Transplantation in people with Acute Graft Versus Host Disease in Intestine, run by Bambino Gesù Hospital and Research Institute at 3 sites in Italy.
Trial summary
This is a pilot, prospective, non-profit, multicenter, uncontrolled, open-label study to evaluate the safety and feasibility of FMT in patients aged between 3 months and 25 years suffering from acute intestinal GVHD resistant to conventional steroid therapy. Read more about this study
- Who is it for?
- Ages 3 months to 25 years
- Conditions the study is for
- Acute Graft Versus Host Disease in Intestine
- What is being tested?
- Fecal Microbial Transplantation
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 3 sitesItaly
- Study dates
- 17 Sep 2026to May 2029
- Phase
- Phase 1/2
- 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 3 months to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis of hematological disease, malignant or non-malignant;
- Patients undergoing allogeneic stem cell transplantation from a familial or unrelated donor;
- Presence of acute GVHD with intestinal involvement (grade II-IV), steroid-resistant (i.e., with progression after 3 days of high-dose steroid therapy (methylprednisolone > 2 mg/kg), no response after 7 days, progression during steroid tapering, or failure to achieve remission on day 28 from the start of steroid therapy), for those who have no indication for other second- or third-line therapies;
- Signed informed consent.
Who cannot join
- Presence of concurrent bacterial infections requiring systemic antibiotic therapy;
- Positivity for anti-HIV or anti-HCV antibodies, or for HbsAg with HBV-DNA positive by PCR;
- Presence of severe mucositis;
- History of chronic inflammatory bowel disease.
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Italy3 sites, 3 recruiting
- Recruiting
Pediatric Hematology and Oncology, IRCCS Azienda Ospedaliero-Universitaria di Bologna
Bologna, 40138
Riccardo Masetti, MD, PhD, +39 051 2144016, riccardo.masetti5@unibo.it
- Not yet recruiting
UOC Clinica di Oncoematologia Pediatrica, Azienda Ospedale-Università Padova
Padova, 35128
Maria Gabelli, MD, +39 049 8213579, maria.gabelli@aopd.veneto.it
- Recruiting
Department of Hematology/Oncology, Cell and Gene Therapy, IRCCS Bambino Gesù Children's Hospital
Roma, 00165
Pietro Merli, MD, +390668592623, pietro.merli@opbg.net
About this study
Official title: Fecal Microbiota Transplantation for the Treatment of Steroid-refractory Graft-versus-host Disease.
This is a pilot, prospective, non-profit, multicenter, uncontrolled, open-label study to evaluate the safety and feasibility of FMT in patients aged between 3 months and 25 years suffering from acute intestinal GVHD resistant to conventional steroid therapy.
Eligible patients will receive 1-3 FMT via naso-jejunal tube or endoscopy.
- Being tested
- Fecal Microbial Transplantation
- Keywords
- graft-versus-host disease
- 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 Microbial TransplantationBiological
The administration of the fecal preparation (from a related donor or a third party donor) will be carried out via esophagogastroduodenoscopy, with the release of the fecal preparation into the duodenum; or via a nasoduodenal tube, with the release of the fecal preparation into the duodenum at a 'dose' of 7-12 ml/kg, up to a maximum of 250 ml/administration; or via colonoscopy. In the latter case, mucosal biopsies will not be performed to reduce the risk of bacterial translocation. In some subjects, the possibility of administering the emulsion via ENEMA will be evaluated. Cases will be selected based on specific clinical indications.
In the case of a partial response, after evaluating the risk/benefit ratio, a second infusion can be performed after 3 days. The procedure can be repeated a third time later (7 days) in case of a new flare of intestinal GVHD after initial improvement.
| Study group | Role | What they receive |
|---|---|---|
| Treatment | Experimental | FMT Receives: Biological: Fecal Microbial Transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety (AE and treatment-related AE, according to CTCAE v5.0)
Number of Participants With Adverse Events and Treatment-Related Adverse Events as Assessed by CTCAE v5.0
Measured: 28 days
Secondary outcomes (5)
Efficacy: Overall Response Rate (ORR)
Number of participants with Complete Response (CR, defined as a score of 0 for the aGvHD grading in all evaluable organs that indicates complete resolution of all signs and symptoms of aGvHD in all evaluable organs without administration of additional systemic therapy for any earlier progression, mixed response or non-response of aGvHD) or Partial Response (PR, defined as improvement of 1 stage in 1 or more organs involved with aGvHD signs or symptoms without progression in other organs or sites without administration of additional systemic therapy for an earlier progression, mixed response or non-response of aGvHD) at day +28 according to MAGIC criteria (ORR=CR+PR).
Measured: 28 days
Microbiota modification
Comparison of alpha-diversity (according to Chao And Simpson indexes) and of beta-diversity (according to Bray-Curtis and UniFrac dissimilarity indexes) of patient samples taken before and after FMT (16s sequencing).
Measured: 56 days
Cumulative incidence of infections
Cumulative incidence of clinically-relevant infections (i.e., ≥ grade 3 according to CTCAE v 5.0) after FMT; relapse or start of a new immunosuppressive treatment will be considered competing events.
Measured: 28 days
Efficacy on other GVHD target organs
Response in skin and liver GVHD involvement according to MAGIC criteria.
Measured: 56 days
Immune reconstitution
Number of participants with CD3+ counts > 500/mcl at day +90 after treatment. Number of participants with CD4+ counts > 50/mcl at day +90 after treatment.
Measured: 90 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 May 2026
- Study start17 Sep 2026
- Primary completionJun 2028 (estimated)
- Study completionMay 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Acute Graft Versus Host Disease in IntestineStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Bambino Gesù Hospital and Research Institute
- Sponsor type
- University, hospital or other
- Collaborators
- IRCCS Azienda Ospedaliero-Universitaria di Bologna, University Hospital, Padua, Italy
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07602751
- Sponsor's protocol ID
- 3444_OPBG_2024
- Status last verified
- Oct 2026
- First submitted
- 11 May 2026
- Last updated
- 20 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
van Lier YF, Davids M, Haverkate NJE, de Groot PF, Donker ML, Meijer E, Heubel-Moenen FCJI, Nur E, Zeerleder SS, Nieuwdorp M, Blom B, Hazenberg MD. Donor fecal microbiota transplantation ameliorates intestinal graft-versus-host disease in allogeneic hematopoietic cell transplant recipients. Sci Transl Med. 2020 Aug 12;12(556):eaaz8926. doi: 10.1126/scitranslmed.aaz8926.
PubMed 32801142Kakihana K, Fujioka Y, Suda W, Najima Y, Kuwata G, Sasajima S, Mimura I, Morita H, Sugiyama D, Nishikawa H, Hattori M, Hino Y, Ikegawa S, Yamamoto K, Toya T, Doki N, Koizumi K, Honda K, Ohashi K. Fecal microbiota transplantation for patients with steroid-resistant acute graft-versus-host disease of the gut. Blood. 2016 Oct 20;128(16):2083-2088. doi: 10.1182/blood-2016-05-717652. Epub 2016 Jul 26.
PubMed 27461930
Study at a glance
From the registry record, last updated by the sponsor on 20 Oct 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 Graft-versus-host Disease.
- ClinicalTrials.gov ID
- NCT07602751
- Acronym / sponsor ID
- 3444_OPBG_2024
- Keywords
- graft-versus-host disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Bambino Gesù Hospital and Research InstituteUniversity, hospital or other
- Collaborators
- IRCCS Azienda Ospedaliero-Universitaria di Bologna, University Hospital, Padua, Italy
- Study start
- 17 Sep 2026 (actual)
- Primary completion
- Jun 2028 (estimated)
- Study completion
- May 2029 (estimated)
- First posted
- 22 May 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.
This is a pilot, prospective, non-profit, multicenter, uncontrolled, open-label study to evaluate the safety and feasibility of FMT in patients aged between 3 months and 25 years suffering from acute intestinal GVHD resistant to conventional steroid therapy.
Eligible patients will receive 1-3 FMT via naso-jejunal tube or endoscopy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment | Experimental | Biological: Fecal Microbial TransplantationFMT |
Who may qualify
- Ages
- 3 months to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Diagnosis of hematological disease, malignant or non-malignant;
- Patients undergoing allogeneic stem cell transplantation from a familial or unrelated donor;
- Presence of acute GVHD with intestinal involvement (grade II-IV), steroid-resistant (i.e., with progression after 3 days of high-dose steroid therapy (methylprednisolone > 2 mg/kg), no response after 7 days, progression during steroid tapering, or failure to achieve remission on day 28 from the start of steroid therapy), for those who have no indication for other second- or third-line therapies;
- Signed informed consent.
Key exclusion criteria (4 listed)
- Presence of concurrent bacterial infections requiring systemic antibiotic therapy;
- Positivity for anti-HIV or anti-HCV antibodies, or for HbsAg with HBV-DNA positive by PCR;
- Presence of severe mucositis;
- History of chronic inflammatory bowel disease.
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 Microbial TransplantationThe administration of the fecal preparation (from a related donor or a third party donor) will be carried out via esophagogastroduodenoscopy, with the release of the fecal preparation into the duodenum; or via a nasoduodenal tube, with the release of the fecal preparation into the duodenum at a 'dose' of 7-12 ml/kg, up to a maximum of 250 ml/administration; or via colonoscopy. In the latter case, mucosal biopsies will not be performed to reduce the risk of bacterial translocation. In some subjects, the possibility of administering the emulsion via ENEMA will be evaluated. Cases will be selected based on specific clinical indications. In the case of a partial response, after evaluating the risk/benefit ratio, a second infusion can be performed after 3 days. The procedure can be repeated a third time later (7 days) in case of a new flare of intestinal GVHD after initial improvement. | Biological | Treatment |
Primary outcome
Safety (AE and treatment-related AE, according to CTCAE v5.0)
Time frame: 28 days
Number of Participants With Adverse Events and Treatment-Related Adverse Events as Assessed by CTCAE v5.0
5 secondary outcomes
Efficacy: Overall Response Rate (ORR)
Time frame: 28 days
Microbiota modification
Time frame: 56 days
Cumulative incidence of infections
Time frame: 28 days
Efficacy on other GVHD target organs
Time frame: 56 days
Immune reconstitution
Time frame: 90 days
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 20 Oct 2026.
- Background: PubMed 32801142van Lier YF, Davids M, Haverkate NJE, de Groot PF, Donker ML, Meijer E, Heubel-Moenen FCJI, Nur E, Zeerleder SS, Nieuwdorp M, Blom B, Hazenberg MD. Donor fecal microbiota transplantation ameliorates intestinal graft-versus-host disease in allogeneic hematopoietic cell transplant recipients. Sci Transl Med. 2020 Aug 12;12(556):eaaz8926. doi: 10.1126/scitranslmed.aaz8926.
- Background: PubMed 27461930Kakihana K, Fujioka Y, Suda W, Najima Y, Kuwata G, Sasajima S, Mimura I, Morita H, Sugiyama D, Nishikawa H, Hattori M, Hino Y, Ikegawa S, Yamamoto K, Toya T, Doki N, Koizumi K, Honda K, Ohashi K. Fecal microbiota transplantation for patients with steroid-resistant acute graft-versus-host disease of the gut. Blood. 2016 Oct 20;128(16):2083-2088. doi: 10.1182/blood-2016-05-717652. Epub 2016 Jul 26.
- 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.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Fecal Microbial Transplantation in people with Acute Graft Versus Host Disease in Intestine, run by Bambino Gesù Hospital and Research Institute at 3 sites in Italy.
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 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 20 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Italy. 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 combined 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.
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 Pietro Merli, MD (+390668592623, pietro.merli@opbg.net), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety (AE and treatment-related AE, according to CTCAE v5.0) (time frame: 28 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 NCT07602751, which also lists 2 linked publications.
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 20 Oct 2026.