Inflammatory Bowel Diseases (IBD)Phase 1FMTNCT02108821
Fecal Microbiota Transplantation in Pediatric Patients
A Phase 1 study testing Fecal Microbiota Transplantation (FMT) in people with Inflammatory Bowel Diseases (IBD) and Crohn's Disease (CD), run by Children's Mercy Hospital Kansas City at 1 site in United States.
Trial summary
A disturbance in the diversity of gut bacterial composition could be linked to several immune mediated diseases including inflammatory bowel diseases (IBD). IBD can be classified into Crohn's Disease (CD) and Ulcerative Colitis (UC). Read more about this study
- Who is it for?
- Ages 2 to 22 yearsHealthy volunteers can take part
- Conditions the study is for
- Inflammatory Bowel Diseases (IBD), Crohn's Disease (CD), Ulcerative Colitis (UC)
- What is being tested?
- Fecal Microbiota Transplantation (FMT)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 23 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2014to 30 Sep 2016
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 22 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
STUDY SUBJECT INCLUSION CRITERIA:
Current IBD patients who have:
- UC patients with a flare due to failure of current therapy and have to undergo esophagogastroduodenoscopy (EGD) and colonoscopy for restaging the disease and escalation of therapy.
- CD patients with ileo colonic or colonic disease who require an EGD and colonoscopy for disease assessment due to a flare or poor control.
- The ability to safely undergo colonoscopy (physical status classification used by the American Society of Anesthesiologists).
- PUCAI score less than sixty five.
- PCDAI score less than forty.
STUDY SUBJECTS EXCLUSION CRITERIA:
- Patients with Crohn's disease: complications like an abscess, phlegmon, stricture, small bowel obstruction, perforation, internal or external fistulization or infection as causes for flare up before being deemed eligible for recruitment to the study. We will check for these complications if a recent study has not been done.
- Severe immunosuppression: Biologicals with concomitant steroids (>30 mg/day).
- Central Line.
- Pressor or ventilatory support.
- On antibiotics.
- Patients with Crohn's disease found to have complications like an abscess, phlegmon, stricture, small bowel obstruction, perforation, internal or external fistulization or infection.
- Not willing to consent or follow guidelines throughout research trial.
- Screening labs in either donor or recipient reveal problems with performing fecal microbiome transplantation because inclusion requirements are no longer met.
- Physician discretion.
- Participant request.
DONOR EXCLUSION CRITERIA:
- A history of antibiotic treatment during the 3 months preceding donation.
- A history of intrinsic gastrointestinal illnesses.
- A history of autoimmune or atopic illness or modulating therapy.
- A history of chronic pain syndromes, or neurologic or neurodevelopmental disorders.
- Metabolic syndrome or malnutrition or obesity.
- A history of exposure to infectious agents.
- Diarrhea or other symptoms of an intestinal infection within two weeks prior to, or on the day of, stool donation.
- A history of malignant illnesses or ongoing oncologic therapy.
- Weight less than 15 kgs.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Mercy Hospital
Kansas City, Missouri, 64108
About this study
Official title: A Study of Fecal Microbiota Transplantation in Pediatric Patients With Relapsed Inflammatory Bowel Disease.
A disturbance in the diversity of gut bacterial composition could be linked to several immune mediated diseases including inflammatory bowel diseases (IBD). IBD can be classified into Crohn's Disease (CD) and Ulcerative Colitis (UC). Both these diseases occur from abnormal immune reaction to resident gut bacteria.The process of fecal microbiota transplantation (FMT) where fecal bacteria from a healthy individual is transferred into a recipient, has recently received attention as an alternative therapy for individuals affected with these life-altering diseases. In this study, the investigators will perform fecal transplantation on the subjects meeting inclusion criteria, to determine the efficacy and safety of this therapy in subjects with IBD (CD and UC) who are not responding to first line therapy, and are in a flare.
Read the detailed description
50 subjects (25 subjects with Crohn's Disease and 25 subjects with Ulcerative Colitis) who are 2 to 22 years of age will be enrolled in the trial over 3 years. The fecal donors, preferably a parent or sibling, will be extensively screened for infectious diseases prior to providing stool for the transplant. Patients who are failing primary therapy, are in a flare, and require restaging of their IBD by an endoscopy and colonoscopy will be approached for the study. Standard of care endoscopy and colonoscopy will be performed on each subject and 2 additional biopsies will be taken for analysis. Microbiota analysis will also be performed on both the donor and recipient stool sample prior to transplantation, and on the recipient sample at 1 week, 1 month, and 6 months post transplantation. The primary objective will be to study the safety of FMT in all enrolled subjects. The study will also correlate efficacy and patient outcomes with the fecal microbiome prior to, and after FMT. The secondary objectives are to examine the efficacy of FMT in the treatment of children with IBD using the Pediatric Ulcerative Colitis Activity Index (PUCAI) and the Pediatric Crohn Disease Activity Index (PCDAI) analysis. Correlate the patient outcomes with the fecal microbiome prior to, and after FMT.
- Being tested
- Fecal Microbiota Transplantation (FMT)
- Keywords
- Inflammatory Bowel Diseases (IBD), Crohn's Disease (CD), Ulcerative Colitis (UC)
- Medical subject headings
- Inflammatory Bowel Diseases, Crohn Disease, Colitis, Ulcerative
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Fecal Microbiota Transplantation (FMT)Biological
Also called Fecal Transplantation
The process of fecal microbiota transplantation (FMT), where fecal bacteria from a healthy individual is transferred into a recipient as an alternative therapy for individuals affected with these life-altering diseases: Crohn's Disease (CD), and/or Ulcerative Colitis (UC).
| Study group | Role | What they receive |
|---|---|---|
| Fecal Microbiome Transplantation | Experimental | Fecal Microbiome Transplantation will be done at the time of EGD and colonoscopy. A parent or sibling or a healthy relative will be tested for several infections like hepatitis, H. Pylori, HIV, syphilis, ova and parasites, culture and C.diff. They will fill out a donor questionnaire used for blood donors prior to the sample collection. After eligibility criteria have been met, appropriate consent has been obtained, and the screening labs have been assessed, the fecal transplant procedure will take place in the procedure center at Children's Hospital of Pittsburgh. Fresh stool sample will be obtained from the donor. The fecal sample will be prepared for transplantation in a designated area in the procedure center. Frequency: once. Duration: Approximately 1 hour Receives: Biological: Fecal Microbiota Transplantation (FMT) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Occurrences of Adverse Events at 6 Months According to Adverse Event Term
Adverse events recorded to determine safety of Fecal Microbiome Transplant in the treatment of children with IBD.
Measured: 6 months
Secondary outcomes (1)
Examine the Efficacy of Fecal Microbiome Transplant in the Treatment of Children With IBD Using the Pediatric Ulcerative Colitis Activity Index (PUCAI) and the Pediatric Crohn Disease Activity Index (PCDAI) Analysis.
Efficacy outcomes scored via Pediatric Ulcerative Colitis Activity Index (PUCAI) and the Pediatric Crohn Disease Activity Index (PCDAI) analysis.
Scored at baseline, day 30, and at day 180 for responders.
The PUCAI score range is 0-85, where the higher the number the more severe the disease (A score of 65 and up is considered severe, a score of 35-64 is considered moderate, and a score of 10-34 is considered mild). The PUCAI is the scoring system used for patients with ulcerative colitis/indeterminate colitis.
The PCDAI score range is 0-100, where the higher the number the more severe the disease (A score of greater than 30 is considered moderate to severe, a score of 11-30 is considered mild, and a score of 10 or less is considered inactive disease). The PCDAI is the scoring system used for patients with crohn's disease.
Measured: Baseline, Day 30, Day 180
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Apr 2014 (estimated)
- Study startMar 2014
- Primary completion30 Sep 2016
- Study completion30 Sep 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Inflammatory Bowel Diseases (IBD)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 23 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Mercy Hospital Kansas City
- Sponsor type
- University, hospital or other
- Collaborators
- University of Pittsburgh, Stanford University
- Responsible party
- Sponsor
- Investigators
- Alka Goyal, MD, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02108821
- Sponsor's protocol ID
- PRO13030081
- Acronym
- FMT
- Status last verified
- Feb 2021
- First submitted
- 21 Mar 2014
- Last updated
- 4 Feb 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 4 Feb 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study of Fecal Microbiota Transplantation in Pediatric Patients With Relapsed Inflammatory Bowel Disease.
- ClinicalTrials.gov ID
- NCT02108821
- Acronym / sponsor ID
- FMT / PRO13030081
- Keywords
- Inflammatory Bowel Diseases (IBD), Crohn's Disease (CD), Ulcerative Colitis (UC)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 23 participants (actual)
- Lead sponsor
- Children's Mercy Hospital Kansas CityUniversity, hospital or other
- Collaborators
- University of Pittsburgh, Stanford University
- Study start
- Mar 2014 (actual)
- Primary completion
- 30 Sep 2016 (actual)
- Study completion
- 30 Sep 2016 (actual)
- First posted
- 9 Apr 2014 (estimated)
- Results
- Posted 4 Feb 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A disturbance in the diversity of gut bacterial composition could be linked to several immune mediated diseases including inflammatory bowel diseases (IBD). IBD can be classified into Crohn's Disease (CD) and Ulcerative Colitis (UC). Both these diseases occur from abnormal immune reaction to resident gut bacteria.The process of fecal microbiota transplantation (FMT) where fecal bacteria from a healthy individual is transferred into a recipient, has recently received attention as an alternative therapy for individuals affected with these life-altering diseases. In this study, the investigators will perform fecal transplantation on the subjects meeting inclusion criteria, to determine the efficacy and safety of this therapy in subjects with IBD (CD and UC) who are not responding to first line therapy, and are in a flare.
Detailed description
50 subjects (25 subjects with Crohn's Disease and 25 subjects with Ulcerative Colitis) who are 2 to 22 years of age will be enrolled in the trial over 3 years. The fecal donors, preferably a parent or sibling, will be extensively screened for infectious diseases prior to providing stool for the transplant. Patients who are failing primary therapy, are in a flare, and require restaging of their IBD by an endoscopy and colonoscopy will be approached for the study. Standard of care endoscopy and colonoscopy will be performed on each subject and 2 additional biopsies will be taken for analysis. Microbiota analysis will also be performed on both the donor and recipient stool sample prior to transplantation, and on the recipient sample at 1 week, 1 month, and 6 months post transplantation. The primary objective will be to study the safety of FMT in all enrolled subjects. The study will also correlate efficacy and patient outcomes with the fecal microbiome prior to, and after FMT. The secondary objectives are to examine the efficacy of FMT in the treatment of children with IBD using the Pediatric Ulcerative Colitis Activity Index (PUCAI) and the Pediatric Crohn Disease Activity Index (PCDAI) analysis. Correlate the patient outcomes with the fecal microbiome prior to, and after FMT.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Fecal Microbiome Transplantation | Experimental | Biological: Fecal Microbiota Transplantation (FMT)Fecal Microbiome Transplantation will be done at the time of EGD and colonoscopy. A parent or sibling or a healthy relative will be tested for several infections like hepatitis, H. Pylori, HIV, syphilis, ova and parasites, culture and C.diff. They will fill out a donor questionnaire used for blood donors prior to the sample collection. After eligibility criteria have been met, appropriate consent has been obtained, and the screening labs have been assessed, the fecal transplant procedure will take place in the procedure center at Children's Hospital of Pittsburgh. Fresh stool sample will be obtained from the donor. The fecal sample will be prepared for transplantation in a designated area in the procedure center. Frequency: once. Duration: Approximately 1 hour |
Who may qualify
- Ages
- 2 to 22 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Fecal Microbiota Transplantation (FMT)Also: Fecal TransplantationThe process of fecal microbiota transplantation (FMT), where fecal bacteria from a healthy individual is transferred into a recipient as an alternative therapy for individuals affected with these life-altering diseases: Crohn's Disease (CD), and/or Ulcerative Colitis (UC). | Biological | Fecal Microbiome Transplantation |
Primary outcome
Number of Occurrences of Adverse Events at 6 Months According to Adverse Event Term
Time frame: 6 months
Adverse events recorded to determine safety of Fecal Microbiome Transplant in the treatment of children with IBD.
1 secondary outcome
Examine the Efficacy of Fecal Microbiome Transplant in the Treatment of Children With IBD Using the Pediatric Ulcerative Colitis Activity Index (PUCAI) and the Pediatric Crohn Disease Activity Index (PCDAI) Analysis.
Time frame: Baseline, Day 30, Day 180
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Feb 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Alka Goyal, MD principal investigator
Stanford University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Fecal Microbiota Transplantation (FMT) in people with Inflammatory Bowel Diseases (IBD) and Crohn's Disease (CD), run by Children's Mercy Hospital Kansas City at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Feb 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 4 Feb 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Occurrences of Adverse Events at 6 Months According to Adverse Event Term (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Feb 2021.