Fecal Microbiota Transplant (FMT) for Relapsing C. Difficile Infection in Adults and Children Using a Frozen Inoculum

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Elizabeth L. Hohmann, MD, Massachusetts General Hospital
ClinicalTrials.gov Identifier:
NCT01704937
First received: October 4, 2012
Last updated: June 18, 2014
Last verified: June 2014
  Purpose

Fecal microbiota transplantation (FMT) is the reconstitution of normal flora by a "stool transplant" from a healthy individual to a C. difficile-infected recipient, and has long been a successful approach to recurrent/refractory C. difficile. The purpose of this project is to generate a frozen FMT inoculum from well-screened healthy volunteer donors which can be used repeatedly, particularly in those who do not have a healthy intimate partner or other related donor. Delivery of FMT has been performed colonoscopically, by fecal retention enema, or by the nasogastric route. This study will evaluate the safety and secondarily the efficacy of a frozen inoculum administered by nasogastric tube vs administered by colonoscope.

Subjects with recurrent/relapsing C. difficile infection (10 per group) will receive FMT via either:

  • colonoscopy
  • NGT

The primary endpoint is assessment of safety as measured by clinical events (GI, procedural, systemic). Efficacy will be defined as a resolution of diarrhea off antibiotics for C. difficile, in the absence of a need for OTHER systemic antibiotics, i.e. resumption of a normal bowel status for the individual. Secondary efficacy endpoints include weight, subjective well-being and relative clinical improvement per standardized questionnaire, and subject qualitative assessment of, and satisfaction with, the transplant procedures. Subjects will be monitored for clinical safety by history and standard exams and the follow-up questionnaire as well as followed closely by phone and in person.


Condition Intervention Phase
Clostridium Difficile Infection
Drug: Fecal Microbiota Transplant (FMT)
Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Fecal Microbiota Transplant (FMT) for Relapsing C. Difficile Infection in Adults and Children Using a Frozen Inoculum

Resource links provided by NLM:


Further study details as provided by Massachusetts General Hospital:

Primary Outcome Measures:
  • Safety [ Time Frame: up to 6 mo post FMT ] [ Designated as safety issue: Yes ]
    Safety is assessed by clinical symptoms, exam, signs (GI and systemic)


Secondary Outcome Measures:
  • Efficacy [ Time Frame: 3 months ] [ Designated as safety issue: No ]
    Efficacy is defined as resolution of C. Difficile signs and symptoms off antibiotics for C. difficile


Estimated Enrollment: 20
Study Start Date: November 2012
Study Completion Date: May 2014
Primary Completion Date: May 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Colonoscopy
Fecal microbiota transplant ("stool transplant") from healthy, unrelated donor via colonoscopy
Drug: Fecal Microbiota Transplant (FMT)
Reconstitution of normal flora by a "stool transplant" from a healthy individual to a C. difficile - infected recipient via colonoscopy or nasogastric tube
Experimental: Nasogastric Tube (NGT)
Fecal microbiota transplant ("stool transplant") from healthy, unrelated donor via NGT
Drug: Fecal Microbiota Transplant (FMT)
Reconstitution of normal flora by a "stool transplant" from a healthy individual to a C. difficile - infected recipient via colonoscopy or nasogastric tube

  Eligibility

Ages Eligible for Study:   7 Years to 90 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patients with recurrent or relapsing CDI defined as EITHER(13):

    • At least three episodes of mild-to-moderate CDI and failure of a 6-8 week taper with vancomycin with or without an alternative antibiotic (e.g. rifaximin, nitazoxanide).
    • At least two episodes of severe CDI resulting in hospitalization and associated with significant morbidity.
  • Willingness to accept risk of unrelated donor stool
  • Willingness to be randomized to NGT vs. colonoscopic delivery.
  • Able to consent for self, or parental assent/child assent as age appropriate.

Exclusion Criteria:

  • Anatomic contraindication to NGT
  • Delayed gastric emptying syndrome
  • Known chronic aspiration
  • Contraindication to colonoscopy (ASA 4 or more)
  • High risk of bacterial translocation (Immunosuppression, cirrhosis etc)
  • Pregnant or breastfeeding women
  • Acute unrelated infection or comorbid illness exaccerbation
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01704937

Locations
United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Sponsors and Collaborators
Massachusetts General Hospital
Investigators
Principal Investigator: Elizabeth L Hohmann, MD Massachusetts General Hospital
  More Information

Publications:
Responsible Party: Elizabeth L. Hohmann, MD, Principal Investigator, Massachusetts General Hospital
ClinicalTrials.gov Identifier: NCT01704937     History of Changes
Other Study ID Numbers: 2012-P-001657
Study First Received: October 4, 2012
Last Updated: June 18, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Massachusetts General Hospital:
Fecal Microbiota Transplant
Clostridium difficile
Diarrhea

Additional relevant MeSH terms:
Infection
Communicable Diseases

ClinicalTrials.gov processed this record on September 22, 2014