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NCT Number: NCT07602751

FMT for Pediatric SR-aGVHD

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.

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Key information

Conditions

Acute Graft Versus Host Disease in Intestine Graft vs Host Disease Immune System Diseases

Age range

3 month–25 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Pediatric Hematology and Oncology, IRCCS Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.

Exclusion criteria

  • 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.

Treatment and study plan

Fecal Microbial Transplantation

Biological

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.

Primary outcomes

  1. 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

Secondary outcomes

  1. Efficacy: Overall Response Rate (ORR)

    Time frame: 28 days

    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).

  2. Microbiota modification

    Time frame: 56 days

    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).

  3. Cumulative incidence of infections

    Time frame: 28 days

    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.

  4. Efficacy on other GVHD target organs

    Time frame: 56 days

    Response in skin and liver GVHD involvement according to MAGIC criteria.

  5. Immune reconstitution

    Time frame: 90 days

    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.

Study contacts

Contact information is provided by the study sponsor or research team.

Pietro Merli, MD

CONTACT

[email protected]

+390668592623

Sponsors and collaborators

Lead sponsor

Bambino Gesù Hospital and Research Institute

Other

Collaborators

  • IRCCS Azienda Ospedaliero-Universitaria di Bologna
  • University Hospital, Padua, Italy

Registry information

Official study title

Fecal Microbiota Transplantation for the Treatment of Steroid-refractory Graft-versus-host Disease.

Important dates

Study start
2026
Primary completion
2028
Study completion
2029
First posted
May 22, 2026
Registry last updated
May 22, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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