Fecal Microbiota Transplantation in GVHD Clinical Trial
Official title:
Fecal Microbiota Transplantation for Treatment of Refractory Graft Versus Host Disease-a Pilot Study
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.
| Status | Not yet recruiting |
| Enrollment | 15 |
| Est. completion date | December 31, 2020 |
| Est. primary completion date | May 31, 2020 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 3 Years to 18 Years |
| Eligibility |
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 |
| Country | Name | City | State |
|---|---|---|---|
| n/a | |||
| Lead Sponsor | Collaborator |
|---|---|
| Shanghai Children's Medical Center |
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | 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 | |
| Primary | 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 | Treatment-related AEs and SAEs | The overall safety of the study will be evaluated with the incidence of all Adverse Events (AEs) and Serious Adverse Events (SAEs) within 28 days following FMT. The relationship of any kind AEs and FMT will be seriously evaluated. Non-serious AEs include: dyspepsia, abdominal pain, nausea, vomiting, diarrhea, constipation, fever, inhalation without mechanical ventilation, etc. SAEs include: death, sepsis, aspiration pneumonia, gastrointestinal hemorrhage, septic shock, etc. | up to 28 days following FMT | |
| Secondary | GVHD severity | Patients will be evaluated on days 7 and 28 following transplantation for severity of GVHD. GVHD severity is graded by the International Bone Marrow Transplant Registry Severity Index grading system. | up to 28 days following FMT | |
| Secondary | Implantation rate | The data will be compared with patients who had not participated in clinical trials. | through study completion, an average of 6 months | |
| Secondary | Survival rate | The data will be compared with patients who had not participated in clinical trials. | through study completion, an average of 6 months | |
| Secondary | Change in biomarkers | Change in levels of albumin and C-reactive protein between days 0 and days 28 will serve as a secondary endpoint. | up to 28 days following FMT | |
| Secondary | Number of patients with infectious disorders | Evaluation of FMT activity on infectious disorder. | through study completion, an average of 6 months | |
| Secondary | Quality of Life | The quality of life of patients will be measured using a standardized quality of life questionnaire (EORTC QLQ-C30) prior to transplantation and on days 7, 14, 21, 28 and 180 following transplantation. | up to 6 months following FMT |