Fecal Microbiota Transplant (FMT) in Pediatric Active Ulcerative Colitis
|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: NCT02330653|
Recruitment Status : Recruiting
First Posted : January 5, 2015
Last Update Posted : October 4, 2018
|Condition or disease||Intervention/treatment||Phase|
|Inflammatory Bowel Diseases Ulcerative Colitis||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 UC. The primary aim is to assess safety and feasibility of a weekly FMT maintenance therapy. A total of 60 patients with active UC (as defined by PUCAI score of >9) 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 PI to 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 Ulcerative Colitis|
|Actual Study Start Date :||November 2015|
|Estimated Primary Completion Date :||December 2018|
|Estimated Study Completion Date :||May 2019|
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 OpenBiome. 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 grade 2 or above [ Time Frame: At 8 weeks after start of FMT ]Proportion of FMT-related adverse events grade 2 or above experienced in each arm.
- Remission of disease [ Time Frame: At 8 weeks and 1 year after start of FMT ]Remission as defined by a PUCAI score of less than 9
- Improvement in inflammatory biomarkers [ 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.
- Changes in gut microbial composition [ Time Frame: At 8 weeks and at 1 year after start of FMT ]We will assess changes in microbial composition and microbial engraftment from donor to recipient.
- Improvement in the Pediatric Ulcerative Colitis Activity Index (PUCAI) by 20 points or more. [ Time Frame: At 8 weeks and at 1 year after start of FMT ]Improvement in disease status as measured by improvement in PUCAI score by 20 points or more.
- Engraftment of donor microbes [ Time Frame: At 8 weeks and at 1 year after start of FMT ]Assessment of engraftment of donor microbes into recipients
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): NCT02330653
|Contact: McKenzie M Leierfirstname.lastname@example.org|
|United States, Massachusetts|
|Boston Children's Hospital||Recruiting|
|Boston, Massachusetts, United States, 02115|
|Contact: McKenzie M Leier email@example.com|
|Principal Investigator:||Stacy A Kahn, MD||Boston Childrens Hospital - GI & Nutrition|