Fecal Microbiota Transplantation for Treatment of Refractory Graft Versus Host Disease-a Pilot Study

Shanghai Jiao Tong University

Status and phase: Unknown; Phase 1

Conditions: Fecal Microbiota Transplantation in GVHD

Treatments: Biological: Fecal Microbiota Transplantation

Study type: Interventional

Funder types: Other

Identifiers

NCT03549676

FMT-GVHD

Details and patient eligibility

About

The study evaluates safety and efficacy of fecal microbiota transplantation (FMT) for the treatment of refractory graft-versus-host-disease (GVHD) of the gut. FMT might be a beneficial treatment in this clinical situation with a poor prognosis and limited therapeutic options.

Full description

Graft-versus-host-disease (GVHD) is a major complication after hematopoietic stem cell transplantation (HSCT). Gut is the most vulnerable target organ of acute GVHD. Patients who have a gastrointestinal acute GVHD received a first-line standard treatment of corticosteroids. For patients who do not respond or progress after an initial response have a high mortality. Therefore, the investigation of effective second line therapy for these patients are in need. The study evaluates safety and efficacy of fecal microbiota transplantation (FMT) for the treatment of refractory GVHD of the gut. FMT might be a beneficial treatment in this clinical situation with a poor prognosis and limited therapeutic options.

Enrollment: 15 estimated patients

Sex: All

Ages: 3 to 18 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• 3-18 years of age
• Allo-SCT patients with acute steroid-resistant GI-related GVHD grade III-IV. Steroid-resistant GI-related GVHD will be defined as lack of improvement (same stage) or worsening of GI symptoms after 7 days of steroid therapy (≥ 2 ml/kg of IV methylprednisolone)
• No definite contraindication for gastrointestinal endoscopy
• Signature of informed consent by the legal guardians of patients

Exclusion criteria

• Prior inclusion to an interventional study
• Previous Allo-SCT
• Known multi-drug resistance carriage prior to stool collection
• Severe colitis of any etiology or a history of inflammatory bowel disease (IBD)
• Uncontrolled infection (hemodynamic instability, ongoing high fever or bacteremia within 3 days after antibiotics administration)
• Active GI bleeding
• Absolute neutrophil count < 500 cells/microL
• Absolute platelet count< 10 x 109 /L
• Patients who cannot give informed consent

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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15 participants in 1 patient group

HSCT patients with refractory GVHD

Experimental group

Description:

Patients will accept FilmArray Gastrointestinal (GI) panel test before pre-treatment of HSCT and 28±3 days post-HSCT. Patients will receive 50ml fecal microbiota from unrelated healthy donors through nasojejunal tube and monitored under gastroscopy. Patients receiving FMT treatment will be followed for at least 6 months. The ideal follow up time is 2 year. Stool and blood samples will be serially collected and tested (before pre-treatment, 1/3/6/12 months after FMT).

Treatment:

Biological: Fecal Microbiota Transplantation