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FMT in Pediatric Crohn's Disease (FMTPCD)

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ClinicalTrials.gov Identifier: NCT03194529
Recruitment Status : Recruiting
First Posted : June 21, 2017
Last Update Posted : October 23, 2018
Sponsor:
Information provided by (Responsible Party):
Children's Hospital Los Angeles

Brief Summary:
The goal of this study is to evaluate the safety of Fecal Microbiota Transplantation (FMT) in children with Crohn's disease who are in remission. Safety will be the primary endpoint and Pediatric Crohn's Disease. Pediatric Crohn's Disease Activity Index (PCDAI) with other secondary endpoints including changes in gut microbial diversity will also be studied. All children will receive the equivalent of 50g of stools from a healthy donor into the jejunum through upper endoscopy. Also, 1-2 additional mucosal biopsies will be collected during patient's routine (standard of care) endoscopy. Subjects will have a total of 5 study visits within 24 weeks including phone call follow up on Day 7 after FMT.

Condition or disease Intervention/treatment Phase
Crohn Disease Pediatric Crohns Disease Biological: FMT Phase 1 Phase 2

Detailed Description:

The Investigators hypothesize that children with Crohn's disease who are in remission can receive a single endoscopic dose of FMT with no significant safety concerns.

All children will receive the equivalent of 50 g of stools from a healthy donor into the jejunum through upper endoscopy. Children will be seen at baseline, then 4 and 24 weeks after study drug administration begins. Stools will be collected and stored for gut microbial profiles during the study visit windows. In addition, a follow up telephone call will be performed 7 days after study drug is administered. If children undergo endoscopy as part of the standard of care, study staff will obtain 1-2 additional biopsies for evaluation of mucosal inflammation. This study will also capture any laboratory results if any of the subjects undergo laboratory testing as part of the standard of care. This study will define the effects of transplant on gut microbial profile using advanced molecular taxonomic approaches. Safety will be closely monitored by solicited (during defined telephone calls and study visits) and unsolicited adverse events (at all times). Safety will be the primary endpoint of this study. Secondary endpoints include Pediatric Crohn's Disease Activity Index (PCDAI), changes in gut microbial diversity - determined by gut microbial genomics and proteomics (16S ribosomal RNA, 16s rRNA), and outcome measures for mucosal inflammation and repair as reflected through laboratory testing such as the level for C-reactive protein (CRP), erythrocyte sedimentation rate (ESR), inflammatory cytokines of the colonic mucosa as well as the stool calprotectin level.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Intervention Model: Single Group Assignment
Intervention Model Description: Primary outcome is safety in children with Crohn's disease in remission.
Masking: None (Open Label)
Primary Purpose: Other
Official Title: Safety of FMT in Maintenance of Pediatric Crohn's Disease
Actual Study Start Date : October 9, 2017
Estimated Primary Completion Date : June 30, 2022
Estimated Study Completion Date : December 31, 2022

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: FMT in children with disease remission
All children (with Crohn's disease in remission) will receive the equivalent of 50 g of stools from a healthy donor (FMT) into the jejunum through upper endoscopy.
Biological: FMT
Fecal Microbiota Transplantation, single dose, 50g of stool, delivered via standard of care upper endoscopy into jejunum.
Other Name: Fecal Microbiota Transplantation




Primary Outcome Measures :
  1. Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] [ Time Frame: 24 weeks ]
    Safety will be closely monitored (during defined telephone calls and study visits) and adverse events will be documented, including mucosal inflammation episodes and standard of care laboratory test abnormalities.


Secondary Outcome Measures :
  1. Crohn's disease activity [ Time Frame: 24 weeks ]
    PCDAI scores will be collected

  2. Changes in gut microbiome [ Time Frame: 24 weeks ]
    The effects of transplant on the gut microbial profile will be determined using advanced molecular taxonomic approaches (gut microbial profiling).



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Ages Eligible for Study:   7 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Age: 7-21 who have been diagnosed with Crohn's disease
  2. Remission of disease defined as PCDAI <10
  3. Needs upper GI endoscopy

Exclusion Criteria:

  1. Unwilling to give informed consent/assent
  2. Pregnancy and breast feeding in patient subjects of childbearing potential
  3. Subjects with significant renal and liver dysfunction (creatinine > 2 mg/dl and direct bilirubin > 2 mg/dl)
  4. Subjects with congenital or acquired immunodeficiency, or who are immunosuppressed due to conditions other than Crohn's disease (such as neoplastic disease or organ transplantation), have received or are receiving chemotherapy, or have been diagnosed with HIV.
  5. Subjects with severe food allergies

Information from the National Library of Medicine

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): NCT03194529


Contacts
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Contact: Sonia Michail, MD (323) 361-1353 sonia.michail@hotmail.com
Contact: Roy Leong, CCRP 323-361-7877 rleong@chla.usc.edu

Locations
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United States, California
Children's Hospital Los Angeles Recruiting
Los Angeles, California, United States, 90027
Contact: Sonia Michail, MD    323-361-1353    sonia.michail@hotmail.com   
Sponsors and Collaborators
Children's Hospital Los Angeles
Investigators
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Principal Investigator: Sonia Michail, MD Children's Hospital Los Angeles

Additional Information:
Publications:
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Responsible Party: Children's Hospital Los Angeles
ClinicalTrials.gov Identifier: NCT03194529     History of Changes
Other Study ID Numbers: CHLA-17-00221
First Posted: June 21, 2017    Key Record Dates
Last Update Posted: October 23, 2018
Last Verified: October 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Children's Hospital Los Angeles:
Crohn's Disease
PCDAI
16s rRNA
Fecal Microbiota Transplantation
Gut Microbiome
FMT

Additional relevant MeSH terms:
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Crohn Disease
Inflammatory Bowel Diseases
Gastroenteritis
Gastrointestinal Diseases
Digestive System Diseases
Intestinal Diseases