Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03549676
Recruitment Status : Not yet recruiting
First Posted : June 8, 2018
Last Update Posted : March 26, 2019
Sponsor:
Information provided by (Responsible Party):
Cao Qing, Shanghai Children's Medical Center

Tracking Information
First Submitted Date  ICMJE May 15, 2018
First Posted Date  ICMJE June 8, 2018
Last Update Posted Date March 26, 2019
Estimated Study Start Date  ICMJE July 1, 2019
Estimated Primary Completion Date May 31, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 25, 2018)
  • Efficacy of FMT in the treatment of refractory GVHD on day 7 [ Time Frame: 7 days following FMT ]
    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.
  • Efficacy of FMT in the treatment of refractory GVHD on day 28 [ Time Frame: 28 days following FMT ]
    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.
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03549676 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: May 25, 2018)
  • Treatment-related AEs and SAEs [ Time Frame: up to 28 days following FMT ]
    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.
  • GVHD severity [ Time Frame: up to 28 days following FMT ]
    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.
  • Implantation rate [ Time Frame: through study completion, an average of 6 months ]
    The data will be compared with patients who had not participated in clinical trials.
  • Survival rate [ Time Frame: through study completion, an average of 6 months ]
    The data will be compared with patients who had not participated in clinical trials.
  • Change in biomarkers [ Time Frame: up to 28 days following FMT ]
    Change in levels of albumin and C-reactive protein between days 0 and days 28 will serve as a secondary endpoint.
  • Number of patients with infectious disorders [ Time Frame: through study completion, an average of 6 months ]
    Evaluation of FMT activity on infectious disorder.
  • Quality of Life [ Time Frame: up to 6 months following FMT ]
    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.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Fecal Microbiota Transplantation for Treatment of Refractory Graft Versus Host Disease-a Pilot Study
Official Title  ICMJE Fecal Microbiota Transplantation for Treatment of Refractory Graft Versus Host Disease-a Pilot Study
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Fecal Microbiota Transplantation in GVHD
Intervention  ICMJE Biological: 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.
Study Arms  ICMJE Experimental: 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).
Intervention: Biological: Fecal Microbiota Transplantation
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Not yet recruiting
Estimated Enrollment  ICMJE
 (submitted: May 25, 2018)
15
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 31, 2020
Estimated Primary Completion Date May 31, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 3 Years to 18 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Qing Cao, MD +86 18930830511 caoqing@scmc.com.cn
Contact: Yue Tao, PhD +86 15121067906 taoyue@scmc.com.cn
Listed Location Countries  ICMJE Not Provided
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03549676
Other Study ID Numbers  ICMJE FMT-GVHD
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party Cao Qing, Shanghai Children's Medical Center
Study Sponsor  ICMJE Shanghai Children's Medical Center
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Qing Cao, MD Shanghai Children's Medical Center
PRS Account Shanghai Children's Medical Center
Verification Date March 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP