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

NCT ID: NCT03549676

Last Updated: 2019-03-26

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE1
• Total Enrollment: 15 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-07-01
• Study Completion Date: 2020-12-31

Brief Summary

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.

Detailed 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.

Conditions

Fecal Microbiota Transplantation in GVHD

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

HSCT patients with refractory GVHD

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).

Group Type: EXPERIMENTAL

Fecal Microbiota Transplantation

Intervention Type: BIOLOGICAL

For patients who do not respond or partial respond for first time FMT treatment, a second time FMT treatment using different donor should be considered.

Interventions

Fecal Microbiota Transplantation

For patients who do not respond or partial respond for first time FMT treatment, a second time FMT treatment using different donor should be considered.

Intervention Type: BIOLOGICAL

Eligibility Criteria

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

• Minimum Eligible Age: 3 Years
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Shanghai Children's Medical Center

OTHER

Sponsor Role: lead

Responsible Party

Cao Qing

Director of Infectious Diseases

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Qing Cao, MD

Role: STUDY_DIRECTOR

Shanghai Children's Medical Center

Central Contacts

Qing Cao, MD

Role: CONTACT

caoqing@scmc.com.cn

+86 18930830511

Yue Tao, PhD

Role: CONTACT

taoyue@scmc.com.cn

+86 15121067906

Other Identifiers

FMT-GVHD

Identifier Type: -

Identifier Source: org_study_id