Fecal Microbiota Transplantation (FMT) for Treatment of Ulcerative Colitis in Children (FMT)
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|ClinicalTrials.gov Identifier: NCT01947101|
Recruitment Status : Completed
First Posted : September 20, 2013
Last Update Posted : June 14, 2016
Patients diagnosed with ulcerative colitis (UC), a specific type of inflammatory bowel disease, will be invited to take part in this study. The investigators do not know what causes UC. However, the microbes (such as bacteria and viruses), which normally live in our intestines are thought to play an important role in the development of UC. There are many treatment options for UC such as steroids,and other medications that decrease inflammation. However, none of these can cure the disease.
This study aims to treat pediatric UC in an alternative fashion by changing the microbes in the gut by giving the participant's stool specimens from healthy adult individuals. There is some evidence from a few cases of adult UC that this therapy may cure the disease or at least provide long lasting suppression (remission) of the symptoms. The investigators would like to test this therapeutic intervention in children with UC.
The purpose of this study is to (1) examine the microbiome of stool and colon (2) and to determine whether healthy-donated stool enemas can treat pediatric ulcerative colitis. (3) The investigators will also study the effects of the treatment on colonic gene expression.
|Condition or disease||Intervention/treatment||Phase|
|Ulcerative Colitis||Biological: Fecal Microbiota Transplant||Phase 1|
Participants will be recruited from the patient population treated by the Pediatric Gastroenterology, Hepatology, and Nutrition Section at Baylor College of Medicine/Texas Children's Hospital.
I. Activities prior to starting FMT therapy:
- Participants will be asked to discontinue or taper their current home medications for ulcerative colitis.
- Participants will be asked to complete enema training.
- The participant and family will be counseled in case emergency surgical and/or intensive care interventions are required.
- Survey Completion to assess clinical symptoms.
II. Initiation of FMT Therapy:
Pre-colonoscopy and FMT therapy preparation:
One to two days prior to scheduled colonoscopy the study participant will be asked to provide a stool sample. They will also undergo a bowel clean-out with Miralax for colonoscopy preparation. Colonoscopy (endoscopic examination of the large bowel) will be used to deliver the first donor fecal transplant at the beginning of the therapeutic protocol.
- FMT treatment and Initial colonoscopy:
Fecal microbiota transplantation is made possible by utilizing a donor (healthy adult) providing their stool sample. The donor's stool will be screened and tested for known disease-causing agents including viruses, bacteria and parasites to prevent spread of disease to the participant.
On the first day of treatment, the participant will undergo a colonoscopy to deliver the filtered donor stool directly into their colon. Additionally, biopsies will be taken during this procedure for routine diagnostic as well as research purposes.
III. Subsequent FMT treatments:
Enemas will be given periodically to complete a total of 1-year therapy.
IV. Follow-up endoscopy and samples:
A similar but more limited procedure (sigmoidoscopy) will be repeated at 14 weeks into the study period. Biopsies will be taken during this procedure for routine diagnostic as well as research purposes.
Potential healthy adult stool donors (between 18 and 45 years of age) will be recruited by the research staff. They will be asked to volunteer for the screening and regularly supply stool samples according to the study protocol.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||6 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase I Study of Fecal Microbiota Transplantation (FMT) in Immunomodulator Dependent Pediatric Ulcerative Colitis (UC)|
|Study Start Date :||February 2014|
|Actual Primary Completion Date :||December 2014|
|Actual Study Completion Date :||May 2016|
Experimental: Ulcerative Colitis
Fecal Microbiota Transplant
Biological: Fecal Microbiota Transplant
Fecal Microbiota Transplant will be endoscopically administered directly into the colon. Consecutive treatments will be given by rectal enema route.
Other Name: stool transplant
- Assess safety of FMT treatment by recording the frequency of adverse events. [ Time Frame: 12 months ]To determine the safety and associated toxicities of serial FMTs in pediatric UC following withdrawal from standard therapy.
- Assess efficacy of FMT treatment with the aid of the Pediatric Ulcerative Colitis Activity (PUCAI) Index, a validated measure of clinical disease severity. [ Time Frame: 1 year ]To preliminarily assess the efficacy of FMT in inducing or maintaining clinical remission of UC.
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): NCT01947101
|United States, Texas|
|Texas Children's Hospital|
|Houston, Texas, United States, 77030|
|Principal Investigator:||Richard Kellermayer, MD||Baylor College of Medicine/Texas Children's Hospital|