Clinical trial NCT03812705: Fecal Microbiota Transplantation for Steroid Resistant/Dependent Acute GI GVHD
Official title: Fecal Microbiota Transplantation for the Treatment of Steroid Resistant/Dependent Acute Gastrointestinal Graft Versus Host Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Hematopoietic and Lymphoid Cell Neoplasm
Keywords: allogeneic hematopoeitic stem cell transplantation
acute gastrointestinal graft versus host disease
fecal microbiome transplantation
Lead sponsor: Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
Sponsor class: OTHER
First posted: 2019-01-23
Enrollment: 6
Sex: ALL
Minimum age (years): 14
Maximum age (years): 60
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: fecal microbiome transplantation for patients with steroid resistant/dependent acute gastroinstestinal graft versus host disease
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate the efficacy and safety of fecal microbiome transplantation in patients with steroid resistant/dependent acute gastrointestinal graft-versus-host disease (GVHD). The patient will cease antibiotics treatment 1 day before FMT, and stop taking food 6 hours before FMT. Patients will be given Ondansetron intravenously 1 hour before FMT. Patients will be injected 200\~300 ml fecal microbiome fluid to left colon by Colonoscopy or duodenum through duodenal tube by gastroscopy. If patient's condition is stable or improved within 1 week, second FMT may be performed 1 week later, up to 4 times will be performed if patient response. If patient's condition is not improved after the second FMT, ceasing FMT.

Detailed description: The purpose of this study is to evaluate the efficacy and safety of fecal microbiome transplantation in patients with steroid resistant/dependent acute gastrointestinal graft-versus-host disease (GVHD).

Inclusion criteria:

1. Age\>= 14 yrs ≤60 yrs.
2. Diagnosed with hematological diseases.
3. Recipients of allogeneic peripheral blood stem cell transplantation.
4. Steroid resistant/dependent acute intestinal graft-versus-host disease (GVHD) within 100 days post-transplantation. The definition of steroid resistant/dependent acute intestinal GVHD is progression within 3 days of starting treatment or an incomplete response by 7 days or recurrence after initial dose reduction.
5. ECOG score ≤2;
6. signed consent form.

Exclusion criteria:

1. Complicated with uncontrolled severe infection except intestine and colon.
2. High risk of hemorrhage, dysfunction of coagulation, active gastrointestinal hemorrhage;
3. Absolute neutrophil count (ANC) \<0.5×10e9/L or platelet count (PLT) \< 20×10e9/L
4. Severe organ dysfunction, including heart failure, respiratory failure, renal failure, epilepsy or central nervous system dysfunction.
5. Participating other clinical trials.
6. Pregnant women.

Treatment:

1. Stop antibiotics treatment 1 day before FMT;
2. Fasting food 6 hours before FMT;
3. Give Ondansetron intravenously 1 hour before FMT for vomiting prevention;
4. Injection of 200\~300 ml fecal microbiome fluid to left colon by Colonoscopy or duodenum through duodenum tube by gastroscopy;
5. If patient's condition is stable or improved within 1 week, second FMT may be performed 1 week later, up to 4 times will be performed if patient response;
6. If patient's condition is not improved after the second FMT, stop FMT.

Major endpoint Response rate of acute gastrointestinal GVHD with 12 weeks after FMT, including complete response and partial response.

Minor endpoints:

1. Time to response of acute gastrointestinal GVHD;
2. Duration of response of acute gastrointestinal GVHD.

Eligibility criteria: Inclusion Criteria:

1. Age\>= 14 yrs ≤60 yrs.
2. Diagnosed with hematological diseases.
3. Recipients of allogeneic peripheral blood stem cell transplantation.
4. Steroid resistant/dependent acute intestinal graft-versus-host disease (GVHD) within 100 days post-transplantation. The definition of steroid resistant/dependent acute intestinal GVHD is progression within 3 days of starting treatment or an incomplete response by 7 days or recurrence after initial dose reduction.
5. Eastern Cooperative Oncology Group (ECOG) score ≤2;
6. Signed consent form.

Exclusion Criteria:

1. Complicated with uncontrolled severe infection except intestine and colon.
2. High risk of hemorrhage, dysfunction of coagulation, active gastrointestinal hemorrhage;
3. Absolute neutrophil count (ANC) \<0.5×10e9/L or platelet count (PLT) \< 20×10e9/L
4. Severe organ dysfunction, including heart failure, respiratory failure, renal failure, epilepsy or central nervous system dysfunction.
5. Participating other clinical trials.
6. Pregnant women.

Primary outcomes:
measure: Response rate
timeFrame: 12 weeks within FMT
Secondary outcomes:
measure: Time to response
timeFrame: 12 weeks within FMT
measure: Duration of response
timeFrame: 12 weeks within FMT
Other outcomes:
Locations:
city: Shanghai
country: China
facility: Shanghai Jiao Tong University Affilated First People's Hospital
state: Shanghai
city: Shanghai
country: China
facility: Shanghai Jiao Tong University Affilated Shanghai General Hospital
state: Shanghai
Study officials:
affiliation: Shanghai Jiao Tong University Affiliated Shanghai General Hospiatal
name: Liping Wan, MD
role: PRINCIPAL_INVESTIGATOR
References: