Fecal Microbiota Transplant (FMT) in Pediatric Active Crohn's Colitis
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|ClinicalTrials.gov Identifier: NCT02330211|
Recruitment Status : Recruiting
First Posted : January 1, 2015
Last Update Posted : November 14, 2017
|Condition or disease||Intervention/treatment||Phase|
|Inflammatory Bowel Diseases Crohn Disease||Biological: Fecal Microbiota Transplant Biological: Placebo||Phase 1 Phase 2|
This is a single-center, phase I/II, randomized, prospective, double-blinded, placebo-controlled study of FMT in the treatment of active pediatric CD. The primary aim is to assess safety and feasibility of a weekly FMT maintenance therapy. A total of 60 patients with active CD (as defined by PCDAI score of >10) will be enrolled and randomized to receive FMT or placebo-FMT (study treatment) by retention enema for 1 week and oral, frozen encapsulated inocula/placebo for 7 weeks. After the first 8 weeks, subjects on FMT who improve or subjects on placebo-FMT who do not improve will have the option to continue on study treatment or switch to open-label FMT until the end of 4 months from study initiation. Subjects will be followed by telephone to assess adverse events for a total of 6 months after their last FMT dose.
An initial subset of 10 subjects will be enrolled in the study (will be limited to only those patients 12 years of age or older and to those who have mild to moderate Crohn's Disease) and randomized to receive FMT or placebo. We'd expect short term adverse events to occur within 7 days of FMT administration. Individual subject safety data will be reviewed by the PIto assess whether FMT appears to be safe in the subject before continuing the subject towards open-label use of FMT.
Patient metadata and stool samples will be collected at key time points. The patient-reported metadata collection technique will allow for numerous clinical correlations to be parsed out using the random forest machine learning capabilities of synthetic learning in microbial ecology (SLiME) to identify taxonomic features associated with important clinical parameters.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||60 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||A Phase I/II, Double Blinded, Placebo Controlled, Single-center Study of Fecal Microbiota Transplant (FMT) for the Treatment of Active Pediatric Crohn's Colitis|
|Actual Study Start Date :||July 17, 2017|
|Estimated Primary Completion Date :||March 2018|
|Estimated Study Completion Date :||August 2018|
Experimental: Fecal Microbiota Transplant
Induction retention enema for the first week of treatment followed by 15 capsules of study treatment (the equivalent of 7.5 grams of human stool) will be (administered within 60 minutes of thawing once weekly for a total of 7 weeks) for a total of 8 weeks. After completing 8 weeks of blinded study treatment, study subjects on FMT who have shown improvement will be given the option to receive open-label maintenance FMT weekly for an additional 8 weeks.
Biological: Fecal Microbiota Transplant
The study intervention consists of frozen, bottled or encapsulated fecal microbiota preparations that have been screened and prepared to a uniform and rigorous standards by the Microbiome Health Research Institute Inc. FMT is performed by patients receiving a retention enema and swallowing capsules, introducing stool from a healthy donor into their intestinal tract.
Other Name: Screened, healthy human donor stool
Placebo Comparator: Placebo
Induction placebo enema for the first week of treatment followed by 15 capsules of study placebo (administered within 60 minutes of thawing once weekly for a total of 7 weeks) for a total of 8 weeks. After completing 8 weeks of blinded study placebo, study subjects on placebo who DO NOT demonstrate improvement will be given the option to receive open-label maintenance FMT weekly for an additional 8 weeks.
Placebo administration will consist of both a placebo retention enema and placebo capsules.
- Safety and tolerability of universal donor FMT compared to placebo: FMT-related adverse events of grade 2 or above [ Time Frame: At 8 weeks after start of FMT ]Proportion of subjects with any FMT-related adverse events of grade 2 or above.
- Improvement Pediatric Crohn's Disease Activity Index (PCDAI) by 12.5 points or more [ Time Frame: At 8 weeks after start of FMT ]Improvement of disease status as measured by improvement of PCDAI score of 12.5 points or more.
- Remission of disease [ Time Frame: At 8 weeks and 1 year after start of FMT ]Remission of disease as defined by PCDAI score of 10 or less
- Identify biomarkers that may confer a clinical response [ Time Frame: At 8 weeks and 1 year after start of FMT ]Improvement in inflammatory biomarkers (stool calprotectin, stool lactoferrin, serum ESR/CRP, albumin, Hematocrit) compared to baseline.
- Safety and tolerability of universal donor FMT compared to placebo: FMT-related adverse events of grade 2 or above [ Time Frame: At 1 year after start of FMT ]Proportion of subjects with any FMT-related adverse events of grade 2 or above.
- Improvement Pediatric Crohn's Disease Activity Index (PCDAI) by 12.5 points or more [ Time Frame: At 1 year after start of FMT ]Improvement of disease status as measured by improvement of PCDAI score of 12.5 points or more.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02330211
|Contact: McKenzie M Leierfirstname.lastname@example.org|
|United States, Massachusetts|
|Boston Children's Hospital||Recruiting|
|Boston, Massachusetts, United States, 02115|
|Contact: McKenzie M Leier 617-919-4609 email@example.com|
|Principal Investigator:||Stacy A Kahn, MD||Boston Childrens Hospital - GI & Nutrition|