Inflammatory Bowel DiseasesPhase 1/2NCT02330653
Fecal Microbiota Transplant (FMT) in Pediatric Active Ulcerative Colitis and Pediatric Active Crohn's Colitis
A Phase 1/2 study testing Fecal Microbiota Transplant (FMT) in people with Inflammatory Bowel Diseases and Ulcerative Colitis, run by Stacy A. Kahn at 1 site in United States.
Trial summary
The primary aims of this phase I/II, randomized, placebo controlled study are the assessment of safety and tolerability of universal donor FMT compared to placebo in pediatric and young adult subjects (ages 5 years through 30 years) with active ulcerative colitis (UC) or active Crohn's colitis (CD) who have failed… Read more about this study
- Who is it for?
- Ages 5 to 30 years
- Conditions the study is for
- Inflammatory Bowel Diseases, Ulcerative Colitis, Crohn Disease
- What is being tested?
- Fecal Microbiota Transplant (FMT)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2015to 8 Apr 2019
- Phase
- Phase 1/2
- 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 5 to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
None listed.
Who cannot join
- Subjects who fall into any of the following exclusion criteria at the time of screening are not eligible for enrollment into the study.
- Patients with extensive and/or severe CD (i.e. fistulizing disease, abscess, small bowel obstruction, fevers).
- Patients in a clinical remission (PUCAI < 9) or (PCDAI <10).
- Patients with recent (within 4 weeks) dose change of biologics, 5-ASA, steroids or immunomodulators
- Patients considered to have toxic megacolon.
- Patients with a known drug allergy to vancomycin, metronidazole or polymyxin.
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- Patients with a history of aspiration, gastroparesis, surgery involving the upper gastrointestinal tract (that might affect upper gastrointestinal motility) or unable to swallow pills.
- Patients with esophageal dysmotility or swallowing dysfunction.
- Patients with known food allergies.
- Patients with positive test results for HBV, HCV, or HIV.
- Female patients with a positive test result on a urine hCG test.
- Patients unwilling or unable to give consent or participate in all study requirements.
- Patients unable or unwilling to receive a retention enema for purposes of induction therapy.
- Patients with recent (within 6 weeks) systemic antibiotic use.
- Patients who have testing consistent with active clostridium difficile.
- Patients with known prior experience with donor FMT.
Trial locations and contacts
1 site in 1 country.
United States1 site
Boston Children's Hospital
Boston, Massachusetts, 02115
About this study
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 and Pediatric Active Crohn's Colitis
The primary aims of this phase I/II, randomized, placebo controlled study are the assessment of safety and tolerability of universal donor FMT compared to placebo in pediatric and young adult subjects (ages 5 years through 30 years) with active ulcerative colitis (UC) or active Crohn's colitis (CD) who have failed, are intolerant to, or have refused traditional first-line maintenance therapy. Secondary objectives include the identification biomarkers in both donor and recipient that may confer a clinical response and to establish whether or not ongoing FMT maintenance therapy is required for maintenance of clinical benefit in pediatric UC or pediatric CD.
Read the detailed description
This is a single-center pilot, phase I/II, randomized, prospective, double-blinded, placebo-controlled study of FMT in the treatment of active pediatric UC and active pediatric CD. The primary aim is to assess safety and feasibility of a weekly FMT maintenance therapy. A total of 10 patients with active UC (as defined by PUCAI score of >9) and 10 patients with active CD (as defined by PDCAI 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 no more than 20 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 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.
- Being tested
- Fecal Microbiota Transplant (FMT), Placebo
- Keywords
- Colitis, Fecal Microbiota Transplant, Ulcerative colitis, inflammatory bowel disease, Crohn's Disease
- Medical subject headings
- Inflammatory Bowel Diseases, Colitis, Ulcerative, Crohn Disease, Colitis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Fecal Microbiota Transplant (FMT)Biological
Also called Screened, healthy human donor stool
The study intervention consists of frozen, bottled or encapsulated fecal microbiota preparations that have been screened and prepared to a uniform and rigorous standard by OpenBiome. FMT is performed by patients receiving a retention enema and swallowing capsules, introducing stool from a healthy donor into their intestinal tract.
PlaceboBiological
Placebo administration will consist of both a placebo retention enema and placebo capsules.
| Study group | Role | What they receive |
|---|---|---|
| Fecal Microbiota Transplant (FMT) | Experimental | Induction retention enema for the first week of treatment followed by once weekly administration of 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 7 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 of once weekly FMT capsule administration. Receives: Biological: Fecal Microbiota Transplant (FMT) |
| Placebo | Placebo comparator | Induction placebo enema for the first week of treatment followed by once weekly administration of 15 capsules of study placebo (administered within 60 minutes of thawing once weekly) for 7 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, beginning with a FMT induction enema followed by 7 weeks of weekly FMT capsule administration. Receives: Biological: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
1. Safety and Tolerability of Universal Donor FMT Compared to Placebo: FMT-related Adverse Events Grade 2 or Above
Number of participants with FMT-related adverse events grade 2 or above experienced in each arm.
Measured: At 8 weeks after start of FMT up to 6 months post treatment, an average of 10 months
Secondary outcomes (4)
Remission of Disease
Remission as defined by a PUCAI score of less than 9 (for UC) or by a PCDAI score of less than 10 (for CD)
Measured: At all intermediate timepoints until 6 month follow up post intervention, an average of 10 months
Improvement in Inflammatory Biomarkers
Improvement in inflammatory biomarkers (stool calprotectin, serum ESR, CRP, albumin, hematocrit) compared to baseline.
Measured: At End of Treatment (8 weeks) and at 6 month post treatment
Percentage of Donor Microbiome Present in Transplant Recipient
We will assess changes in microbial composition and the extent of microbial engraftment from the donor in the recipient by comparing the similarity of the microbiomes at two weeks at seven weeks after the induction enema.
Measured: At two weeks and seven weeks post induction enema
Number of Participants With Improvement in Disease Activity
5a. For UC - Improvement of Pediatric Ulcerative Colitis Activity Index (PUCAI) by 20 points or more.
Improvement in disease status as measured by improvement of PUCAI score by 20 points or more.
5b. For CD - Improvement of Pediatric Crohn's Disease Activity Index (PCDAI) by 12.5 points or more.
Improvement of disease status as measured by improvement of PCDAI score by 12.5 points or more.
Measured: At 8 weeks after start of FMT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jan 2015 (estimated)
- Study startNov 2015
- Primary completion8 Apr 2019
- Study completion8 Apr 2019
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 DiseasesStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stacy A. Kahn
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Stacy A. Kahn, Boston Children's Hospital
- Investigators
- Stacy A Kahn, MD, Principal investigator, Boston Childrens Hospital - GI & Nutrition
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02330653
- Sponsor's protocol ID
- P00014876
- Status last verified
- Oct 2023
- First submitted
- 29 Dec 2014
- Last updated
- 16 Oct 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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 and Pediatric Active Crohn's Colitis
- ClinicalTrials.gov ID
- NCT02330653
- Acronym / sponsor ID
- P00014876
- Keywords
- Colitis, Fecal Microbiota Transplant, Ulcerative colitis, inflammatory bowel disease, Crohn's Disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Stacy A. KahnUniversity, hospital or other
- Study start
- Nov 2015 (actual)
- Primary completion
- 8 Apr 2019 (actual)
- Study completion
- 8 Apr 2019 (actual)
- First posted
- 5 Jan 2015 (estimated)
- Results
- Posted 16 Oct 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary aims of this phase I/II, randomized, placebo controlled study are the assessment of safety and tolerability of universal donor FMT compared to placebo in pediatric and young adult subjects (ages 5 years through 30 years) with active ulcerative colitis (UC) or active Crohn's colitis (CD) who have failed, are intolerant to, or have refused traditional first-line maintenance therapy. Secondary objectives include the identification biomarkers in both donor and recipient that may confer a clinical response and to establish whether or not ongoing FMT maintenance therapy is required for maintenance of clinical benefit in pediatric UC or pediatric CD.
Detailed description
This is a single-center pilot, phase I/II, randomized, prospective, double-blinded, placebo-controlled study of FMT in the treatment of active pediatric UC and active pediatric CD. The primary aim is to assess safety and feasibility of a weekly FMT maintenance therapy. A total of 10 patients with active UC (as defined by PUCAI score of >9) and 10 patients with active CD (as defined by PDCAI 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 no more than 20 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 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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Fecal Microbiota Transplant (FMT) | Experimental | Biological: Fecal Microbiota Transplant (FMT)Induction retention enema for the first week of treatment followed by once weekly administration of 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 7 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 of once weekly FMT capsule administration. |
| Placebo | Placebo comparator | Biological: PlaceboInduction placebo enema for the first week of treatment followed by once weekly administration of 15 capsules of study placebo (administered within 60 minutes of thawing once weekly) for 7 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, beginning with a FMT induction enema followed by 7 weeks of weekly FMT capsule administration. |
Who may qualify
- Ages
- 5 to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (0 listed)
Key exclusion criteria (16 listed)
- Subjects who fall into any of the following exclusion criteria at the time of screening are not eligible for enrollment into the study.
- Patients with extensive and/or severe CD (i.e. fistulizing disease, abscess, small bowel obstruction, fevers).
- Patients in a clinical remission (PUCAI < 9) or (PCDAI <10).
- Patients with recent (within 4 weeks) dose change of biologics, 5-ASA, steroids or immunomodulators
- Patients considered to have toxic megacolon.
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 Transplant (FMT)Also: Screened, healthy human donor stoolThe study intervention consists of frozen, bottled or encapsulated fecal microbiota preparations that have been screened and prepared to a uniform and rigorous standard by OpenBiome. FMT is performed by patients receiving a retention enema and swallowing capsules, introducing stool from a healthy donor into their intestinal tract. | Biological | Fecal Microbiota Transplant (FMT) |
| PlaceboPlacebo administration will consist of both a placebo retention enema and placebo capsules. | Biological | Placebo |
Primary outcome
1. 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 up to 6 months post treatment, an average of 10 months
Number of participants with FMT-related adverse events grade 2 or above experienced in each arm.
4 secondary outcomes
Remission of Disease
Time frame: At all intermediate timepoints until 6 month follow up post intervention, an average of 10 months
Improvement in Inflammatory Biomarkers
Time frame: At End of Treatment (8 weeks) and at 6 month post treatment
Percentage of Donor Microbiome Present in Transplant Recipient
Time frame: At two weeks and seven weeks post induction enema
Number of Participants With Improvement in Disease Activity
Time frame: At 8 weeks after start of FMT
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Oct 2023.
- 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.
Stacy A Kahn, MD principal investigator
Boston Childrens Hospital - GI & Nutrition
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Fecal Microbiota Transplant (FMT) in people with Inflammatory Bowel Diseases and Ulcerative Colitis, run by Stacy A. Kahn 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?
The sponsor lists 0 inclusion and 16 exclusion criteria. 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 16 Oct 2023. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 16 Oct 2023). 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?
1. 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 up to 6 months post treatment, an average of 10 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 16 Oct 2023.