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

NCT03549676 | Unknown Phase 1 DMC

• 1 other identifier: FMT-GVHD
• Study Type: interventional
• Target: 15
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Fecal Microbiota Transplantation in GVHD

Keywords

FMT; GVHD; HSCT

Outcome Measures

Primary Outcomes (2)

• Efficacy of FMT in the treatment of refractory GVHD on day 7

  Participants will be evaluated on days 7 following FMT. The volume and frequency of daily diarrhoea will be continuous observed and recorded in 7 days.

  7 days following FMT

• Efficacy of FMT in the treatment of refractory GVHD on day 28

  Participants will be evaluated on days 28 following FMT. The volume and frequency of daily diarrhoea will be continuous observed and recorded in 28 days.

  28 days following FMT

Secondary Outcomes (7)

• Treatment-related AEs and SAEs

  up to 28 days following FMT

• GVHD severity

  up to 28 days following FMT

• Implantation rate

  through study completion, an average of 6 months

• Survival rate

  through study completion, an average of 6 months

• Change in biomarkers

  up to 28 days following FMT

Study Arms (1)

HSCT patients with refractory GVHD

EXPERIMENTAL

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).

Biological: Fecal Microbiota Transplantation

Interventions

Fecal Microbiota Transplantation 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.

HSCT patients with refractory GVHD

Eligibility Criteria

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

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Shanghai Children's Medical Center: lead

MeSH Terms

Interventions

Fecal Microbiota Transplantation

Intervention Hierarchy (Ancestors)

Biological Therapy; Therapeutics

Study Officials

• Qing Cao, MD

  Shanghai Children's Medical Center

  STUDY DIRECTOR

Central Study Contacts

Qing Cao, MD

CONTACT

+86 18930830511; caoqing@scmc.com.cn

Yue Tao, PhD

CONTACT

+86 15121067906; taoyue@scmc.com.cn

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director of Infectious Diseases

Study Record Dates

• First Submitted: May 15, 2018
• First Posted: June 8, 2018
• Study Start: July 1, 2019
• Primary Completion: May 31, 2020
• Study Completion: December 31, 2020
• Last Updated: March 26, 2019

Record last verified: 2019-03