FMT for Pediatric SR-aGVHD

NCT07602751 | Not Yet Recruiting Phase 1

• 1 other identifier: 3444_OPBG_2024
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 3

Brief 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. Eligible patients will receive 1-3 FMT via naso-jejunal tube or endoscopy.

Conditions

Acute Graft Versus Host Disease in Intestine

Keywords

graft-versus-host disease

Outcome Measures

Primary Outcomes (1)

• 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

  28 days

Secondary Outcomes (5)

• Efficacy: Overall Response Rate (ORR)

  28 days

• Microbiota modification

  56 days

• Cumulative incidence of infections

  28 days

• Efficacy on other GVHD target organs

  56 days

• Immune reconstitution

  90 days

Study Arms (1)

Treatment

EXPERIMENTAL

FMT

Biological: Fecal Microbial Transplantation

Interventions

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.

Treatment

Eligibility Criteria

• Age: 3 Months - 25 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Bambino Gesù Hospital and Research Institute: lead
• IRCCS Azienda Ospedaliero-Universitaria di Bologna: collaborator
• University Hospital, Padua, Italy: collaborator

Study Sites (3)

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

Bologna, 40138, Italy

Location

UOC Clinica di Oncoematologia Pediatrica, Azienda Ospedale-Università Padova

Padova, 35128, Italy

Location

Department of Hematology/Oncology, Cell and Gene Therapy, IRCCS Bambino Gesù Children's Hospital

Roma, 00165, Italy

Location

Related Publications (2)

• 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.

  PMID: 32801142 BACKGROUND

• Kakihana 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.

  PMID: 27461930 BACKGROUND

MeSH Terms

Conditions

Graft vs Host Disease

Condition Hierarchy (Ancestors)

Immune System Diseases

Central Study Contacts

Pietro Merli, MD

CONTACT

+390668592623; pietro.merli@opbg.net

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 11, 2026
• First Posted: May 22, 2026
• Study Start: May 18, 2026
• Primary Completion (Estimated): June 1, 2028
• Study Completion (Estimated): May 1, 2029
• Last Updated: May 22, 2026

Record last verified: 2026-05

Locations

IT (3)