Clinical trial NCT03549676: Fecal Microbiota Transplantation for Treatment of Refractory Graft Versus Host Disease-a Pilot Study
Official title: Fecal Microbiota Transplantation for Treatment of Refractory Graft Versus Host Disease-a Pilot Study
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
Conditions: Fecal Microbiota Transplantation in GVHD
Keywords: FMT
GVHD
HSCT
Lead sponsor: Shanghai Children's Medical Center
Sponsor class: OTHER
First posted: 2018-06-08
Enrollment: 15
Sex: ALL
Minimum age (years): 3
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Efficacy of FMT in the treatment of refractory GVHD on day 7
timeFrame: 7 days following FMT
measure: Efficacy of FMT in the treatment of refractory GVHD on day 28
timeFrame: 28 days following FMT
Secondary outcomes:
measure: Treatment-related AEs and SAEs
timeFrame: up to 28 days following FMT
measure: GVHD severity
timeFrame: up to 28 days following FMT
measure: Implantation rate
timeFrame: through study completion, an average of 6 months
measure: Survival rate
timeFrame: through study completion, an average of 6 months
measure: Change in biomarkers
timeFrame: up to 28 days following FMT
measure: Number of patients with infectious disorders
timeFrame: through study completion, an average of 6 months
measure: Quality of Life
timeFrame: up to 6 months following FMT
Other outcomes:
Locations:
Study officials:
affiliation: Shanghai Children's Medical Center
name: Qing Cao, MD
role: STUDY_DIRECTOR
References: