Ulcerative ColitisPhase 1FMT_UCNCT02291523
The Effect of Therapeutic Fecal Transplant on the Gut Microbiome in Children With Ulcerative Colitis
A Phase 1 study testing Fecal Microbial Transplant in people with Ulcerative Colitis, run by Children's Hospital Los Angeles at 1 site in United States.
Trial summary
Ninety Six patients with mild to moderate ulcerative colitis will be randomized to double blind, placebo controlled study. The safety and efficacy of the intervention will be closely monitored. Read more about this study
- Who is it for?
- Ages 7 to 21 yearsHealthy volunteers can take part
- Conditions the study is for
- Ulcerative Colitis
- What is being tested?
- Fecal Microbial Transplant
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 101 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Nov 2016to Nov 2023
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 7 to 21 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Who may be able to join
- Age: 7-21 who have been diagnosed with ulcerative colitis
- Mild to moderate disease based on PUCAI with a score of 10-64
- Need for colonoscopy
Who cannot join
- Children who are known to be resistant to steroid therapy, immunomodulators and biologics, or on a steroid dose greater than 0.5 mg/kg/day (maximum 20 mg)
- Children with recent dose change of biologics (within 4 weeks), 5-ASA, steroids or immunomodulators (within 4 weeks)
- Allergy to or intolerance of mesalamine or 5-ASA products
- Any evidence of infectious colitis
- Concurrent infections that require anti-microbial therapy (such as abdominal abscess, pneumonia, etc…)
- Unable to give informed consent/assent
Show all 9Show fewer
- Have received probiotic preparations ≤ 4 weeks prior to randomization
- Pregnancy and breast feeding in patient subjects of childbearing potential
- Subjects with significant renal and liver dysfunction (creatinine > 2 mg/dl and direct bilirubin > 2 mg/dl), Subjects with congenital or acquired immunodeficiency, or who are immunosuppressed due to conditions other than ulcerative colitis (such as neoplastic disease or organ transplantation), have received or are receiving chemotherapy, or have been diagnosed with HIV.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Hospital Los Angeles
Los Angeles, California, 90027
About this study
Ninety Six patients with mild to moderate ulcerative colitis will be randomized to double blind, placebo controlled study. The safety and efficacy of the intervention will be closely monitored.
Read the detailed description
The enteric microbiota is now accepted as an important etiologic factor in the pathogenesis of human Inflammatory Bowel Disease (IBD) and immune-mediated chronic experimental intestinal inflammation, with ample data to implicate the microbiome as a main factor in the occurrence of IBD. This can be inferred from animals in germ-free environment which can protect from experimental colitis. In addition, increased gut permeability due to dysbiosis, is frequently seen in patients with IBD even in remission and, similarly, first degree relatives of IBD. Therefore, it is not surprising that therapeutic interventions aiming at modifying the gut microbiome would be of therapeutic benefit. Ulcerative colitis is a condition that is characterized by chronic inflammation of the colon. It is an important pediatric disease as 25% of all cases begin in childhood and its incidence is continuously on the rise. It is believed to be related to a genetically and environmentally-generated altered immune response to the enteric microbiome. Previous work in the PI's laboratory suggests that children harbor a unique gut microbial profile, which can predict therapeutic response. Therefore, modifying the gut microbiome may result in therapeutic benefit. However, attempts to modify the gut microbiome were largely unsuccessful until the advent of fecal transplant, which is a new approach in treating colitis. Fecal microbiota transplant (FMT) has been introduced several decades ago in an attempt to restore the gut microbial balance and it appears to be a more efficient method to effectively change and sustain the gut microbial composition. To date there have been a number of successful reports to suggest control of disease activity and in some cases cure of the disease. This study aims to further determine the safety and efficacy of FMT in treating children with ulcerative colitis
- Conditions
- Ulcerative Colitis
- Being tested
- Fecal Microbial Transplant
- Keywords
- Fecal transplant
- Medical subject headings
- Colitis, Ulcerative
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Fecal Microbial TransplantBiological
Also called FMT
Fecal Transplant via Colonoscopy.
| Study group | Role | What they receive |
|---|---|---|
| Patient Stool Transplant | Sham comparator | Arm 1 will get FMT (Fecal Microbial Transplant) placebo and high dose 5-ASA (Pentasa). The FMT is done through colonoscopy. Receives: Biological: Fecal Microbial Transplant |
| Donor Stool Transplant | Active comparator | Arm 2 will get FMT (Fecal Microbial Transplant) with Healthy Donor Stool and high dose 5-ASA (Pentasa). The FMT is done through colonoscopy. Receives: Biological: Fecal Microbial Transplant |
What is being measured
The primary outcome is the main question the study is designed to answer.
The primary endpoint is disease remission based on PUCAI scores (<10).
The primary outcome including results of disease activity, and safety measures.
Measured: 12 Months
Secondary outcomes (1)
Secondary endpoints will include change in mucosal inflammation reflected on laboratory studies.
Secondary endpoints include changes in gut microbial diversity - determined by gut microbial genomics and proteomics, and outcome measures for mucosal inflammation and repair including laboratory testing such as the level for C-reactive protein (CRP) and erythrocyte sedimentation rate (ESR) as well as the stool calprotectin level.
Measured: 12 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Nov 2014 (estimated)
- Study start1 Nov 2016
- Primary completionNov 2023 (estimated)
- Study completionNov 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Ulcerative ColitisStudies of the same condition, open and under way.
- More trials run by Children's Hospital Los AngelesThe sponsor's other registered studies.
- 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 101 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Los Angeles
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Sonia Michail, MD, Children's Hospital Los Angeles
- Investigators
- Sonia Michail, MD, Principal investigator, Children Hospital Los Angeles
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02291523
- Sponsor's protocol ID
- CHLA-16-00050
- Acronym
- FMT_UC
- Status last verified
- Oct 2023
- First submitted
- 11 Nov 2014
- Last updated
- 2 Nov 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Michail S, Bultron G, Depaolo RW. Genetic variants associated with Crohn's disease. Appl Clin Genet. 2013 Jul 16;6:25-32. doi: 10.2147/TACG.S33966. Print 2013.
PubMed 23935379Michail S, Durbin M, Turner D, Griffiths AM, Mack DR, Hyams J, Leleiko N, Kenche H, Stolfi A, Wine E. Alterations in the gut microbiome of children with severe ulcerative colitis. Inflamm Bowel Dis. 2012 Oct;18(10):1799-808. doi: 10.1002/ibd.22860. Epub 2011 Dec 14.
PubMed 22170749Hyams JS, Lerer T, Mack D, Bousvaros A, Griffiths A, Rosh J, Otley A, Evans J, Stephens M, Kay M, Keljo D, Pfefferkorn M, Saeed S, Crandall W, Michail S, Kappelman MD, Grossman A, Samson C, Sudel B, Oliva-Hemker M, Leleiko N, Markowitz J; Pediatric Infl ammatory Bowel Disease Collaborative Research Group Registry. Outcome following thiopurine use in children with ulcerative colitis: a prospective multicenter registry study. Am J Gastroenterol. 2011 May;106(5):981-7. doi: 10.1038/ajg.2010.493. Epub 2011 Jan 11.
PubMed 21224840Le J, Hakimjavadi H, Parsana R, Chamala S, Michail S. Fecal Microbiota Transplantation Induces Sustained Gut Microbiome Changes in Pediatric Ulcerative Colitis: A Combined Randomized and Open-Label Study. Gastro Hep Adv. 2025 Jul 11;4(10):100741. doi: 10.1016/j.gastha.2025.100741. eCollection 2025.
PubMed 40933007
Study at a glance
From the registry record, last updated by the sponsor on 2 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Effect of Therapeutic Fecal Transplant on the Gut Microbiome in Children With Ulcerative Colitis
- ClinicalTrials.gov ID
- NCT02291523
- Acronym / sponsor ID
- FMT_UC / CHLA-16-00050
- Conditions
- Ulcerative Colitis
- Keywords
- Fecal transplant
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 101 participants (estimated)
- Lead sponsor
- Children's Hospital Los AngelesUniversity, hospital or other
- Study start
- 1 Nov 2016 (actual)
- Primary completion
- Nov 2023 (estimated)
- Study completion
- Nov 2023 (estimated)
- First posted
- 14 Nov 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Ninety Six patients with mild to moderate ulcerative colitis will be randomized to double blind, placebo controlled study. The safety and efficacy of the intervention will be closely monitored.
Detailed description
The enteric microbiota is now accepted as an important etiologic factor in the pathogenesis of human Inflammatory Bowel Disease (IBD) and immune-mediated chronic experimental intestinal inflammation, with ample data to implicate the microbiome as a main factor in the occurrence of IBD. This can be inferred from animals in germ-free environment which can protect from experimental colitis. In addition, increased gut permeability due to dysbiosis, is frequently seen in patients with IBD even in remission and, similarly, first degree relatives of IBD. Therefore, it is not surprising that therapeutic interventions aiming at modifying the gut microbiome would be of therapeutic benefit. Ulcerative colitis is a condition that is characterized by chronic inflammation of the colon. It is an important pediatric disease as 25% of all cases begin in childhood and its incidence is continuously on the rise. It is believed to be related to a genetically and environmentally-generated altered immune response to the enteric microbiome. Previous work in the PI's laboratory suggests that children harbor a unique gut microbial profile, which can predict therapeutic response. Therefore, modifying the gut microbiome may result in therapeutic benefit. However, attempts to modify the gut microbiome were largely unsuccessful until the advent of fecal transplant, which is a new approach in treating colitis. Fecal microbiota transplant (FMT) has been introduced several decades ago in an attempt to restore the gut microbial balance and it appears to be a more efficient method to effectively change and sustain the gut microbial composition. To date there have been a number of successful reports to suggest control of disease activity and in some cases cure of the disease. This study aims to further determine the safety and efficacy of FMT in treating children with ulcerative colitis
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 |
|---|---|---|
| Patient Stool Transplant | Sham comparator | Biological: Fecal Microbial TransplantArm 1 will get FMT (Fecal Microbial Transplant) placebo and high dose 5-ASA (Pentasa). The FMT is done through colonoscopy. |
| Donor Stool Transplant | Active comparator | Biological: Fecal Microbial TransplantArm 2 will get FMT (Fecal Microbial Transplant) with Healthy Donor Stool and high dose 5-ASA (Pentasa). The FMT is done through colonoscopy. |
Who may qualify
- Ages
- 7 to 21 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Age: 7-21 who have been diagnosed with ulcerative colitis
- Mild to moderate disease based on PUCAI with a score of 10-64
- Need for colonoscopy
Key exclusion criteria (9 listed)
- Children who are known to be resistant to steroid therapy, immunomodulators and biologics, or on a steroid dose greater than 0.5 mg/kg/day (maximum 20 mg)
- Children with recent dose change of biologics (within 4 weeks), 5-ASA, steroids or immunomodulators (within 4 weeks)
- Allergy to or intolerance of mesalamine or 5-ASA products
- Any evidence of infectious colitis
- Concurrent infections that require anti-microbial therapy (such as abdominal abscess, pneumonia, etc…)
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 Microbial TransplantAlso: FMTFecal Transplant via Colonoscopy. | Biological | Donor Stool Transplant, Patient Stool Transplant |
Primary outcome
The primary endpoint is disease remission based on PUCAI scores (<10).
Time frame: 12 Months
The primary outcome including results of disease activity, and safety measures.
1 secondary outcome
Secondary endpoints will include change in mucosal inflammation reflected on laboratory studies.
Time frame: 12 Months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Nov 2023.
- Background: PubMed 23935379Michail S, Bultron G, Depaolo RW. Genetic variants associated with Crohn's disease. Appl Clin Genet. 2013 Jul 16;6:25-32. doi: 10.2147/TACG.S33966. Print 2013.
- Background: PubMed 22170749Michail S, Durbin M, Turner D, Griffiths AM, Mack DR, Hyams J, Leleiko N, Kenche H, Stolfi A, Wine E. Alterations in the gut microbiome of children with severe ulcerative colitis. Inflamm Bowel Dis. 2012 Oct;18(10):1799-808. doi: 10.1002/ibd.22860. Epub 2011 Dec 14.
- Background: PubMed 21224840Hyams JS, Lerer T, Mack D, Bousvaros A, Griffiths A, Rosh J, Otley A, Evans J, Stephens M, Kay M, Keljo D, Pfefferkorn M, Saeed S, Crandall W, Michail S, Kappelman MD, Grossman A, Samson C, Sudel B, Oliva-Hemker M, Leleiko N, Markowitz J; Pediatric Infl ammatory Bowel Disease Collaborative Research Group Registry. Outcome following thiopurine use in children with ulcerative colitis: a prospective multicenter registry study. Am J Gastroenterol. 2011 May;106(5):981-7. doi: 10.1038/ajg.2010.493. Epub 2011 Jan 11.
- Derived from this study: PubMed 40933007Le J, Hakimjavadi H, Parsana R, Chamala S, Michail S. Fecal Microbiota Transplantation Induces Sustained Gut Microbiome Changes in Pediatric Ulcerative Colitis: A Combined Randomized and Open-Label Study. Gastro Hep Adv. 2025 Jul 11;4(10):100741. doi: 10.1016/j.gastha.2025.100741. eCollection 2025.
- Individual participant dataSharing planned: No
Related trials for Ulcerative Colitis
Not yet recruitingPhase 1
An Open-label, DDI Study to Investigate the Effects of Duvakitug on the PK of Selected Cytochrome P450 Substrates
Ulcerative Colitis, Crohn's Disease
RecruitingObservational study
Longitudinal Immunophenotyping of Patients With Inflammatory Bowel Disease
Inflammatory Bowel Disease, Crohn's Disease
RecruitingObservational study
Ibd CAncer and seRious Infections in France (I-CARE 2)
IBD, Ulcerative Colitis
RecruitingObservational study
Characterization of Colorectal Dysplasic Lesions and Stratification of Their Evolving Risk in Inflammatory Bowel Disease Patients: a Prospective, Observational and Multicenter Study
Crohn Disease (CD), Colitis Ulcerative
Enrolling by invitationPhase 4
MOdel-Informed Precision Dosing of Ustekinumab and VEdolizumab in Inflammatory Bowel Disease
Inflammatory Bowel Diseases, Crohn Disease
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.
Sonia Michail, MD principal investigator
Children Hospital Los Angeles
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 Microbial Transplant in people with Ulcerative Colitis, run by Children's Hospital Los Angeles 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 3 inclusion and 9 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: status not confirmed, as of the sponsor's last update on 2 Nov 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 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?
Results are not available in the source record.
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?
The primary endpoint is disease remission based on PUCAI scores (<10). (time frame: 12 Months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02291523, which also lists 4 linked publications.
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 2 Nov 2023.