Crohn DiseasePhase 1/2FMTPCDNCT03194529
FMT in Pediatric Crohn's Disease
A Phase 1/2 study testing FMT in people with Crohn Disease and Pediatric Crohns Disease, run by Children's Hospital Los Angeles at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 7 to 21 years
- Conditions the study is for
- Crohn Disease, Pediatric Crohns Disease
- What is being tested?
- FMT
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteUnited States
- Study dates
- 9 Oct 2017to 31 Jul 2019
- Phase
- Phase 1/2
- 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
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age: 7-21 who have been diagnosed with Crohn's disease
- Remission of disease defined as PCDAI <10
- Needs upper GI endoscopy
Who cannot join
- Unwilling to give informed consent/assent
- 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 Crohn's disease (such as neoplastic disease or organ transplantation), have received or are receiving chemotherapy, or have been diagnosed with HIV.
- Subjects with severe food allergies
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Hospital Los Angeles
Los Angeles, California, 90027
About this study
Official title: Safety of FMT in Maintenance of Pediatric Crohn's Disease
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.
Read the 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.
- Conditions
- Crohn Disease, Pediatric Crohns Disease
- Being tested
- FMT
- Keywords
- Crohn's Disease, PCDAI, 16s rRNA, Fecal Microbiota Transplantation, Gut Microbiome, FMT
- Medical subject headings
- Crohn Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FMTBiological
Also called Fecal Microbiota Transplantation
Fecal Microbiota Transplantation, single dose, 50g of stool, delivered via standard of care upper endoscopy into jejunum.
| Study group | Role | What they receive |
|---|---|---|
| FMT in children with disease remission | Experimental | 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. Receives: Biological: FMT |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
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.
Measured: 24 weeks
Secondary outcomes (2)
Crohn's disease activity
PCDAI scores will be collected
Measured: 24 weeks
Changes in gut microbiome
The effects of transplant on the gut microbial profile will be determined using advanced molecular taxonomic approaches (gut microbial profiling).
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jun 2017
- Study start9 Oct 2017
- Primary completion30 Jun 2019
- Study completion31 Jul 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Crohn DiseaseStudies 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 1/2
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Primary outcome is safety in children with Crohn's disease in remission.
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Los Angeles
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Sonia Michail, MD, Principal investigator, Children's Hospital Los Angeles
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03194529
- Sponsor's protocol ID
- CHLA-17-00221
- Acronym
- FMTPCD
- Status last verified
- Mar 2020
- First submitted
- 19 Jun 2017
- Last updated
- 13 Mar 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Rinawi F, Assa A, Hartman C, Mozer Glassberg Y, Friedler VN, Rosenbach Y, Silbermintz A, Zevit N, Shamir R. Incidence of Bowel Surgery and Associated Risk Factors in Pediatric-Onset Crohn's Disease. Inflamm Bowel Dis. 2016 Dec;22(12):2917-2923. doi: 10.1097/MIB.0000000000000937.
PubMed 27755214Shi Y, Dong Y, Huang W, Zhu D, Mao H, Su P. Fecal Microbiota Transplantation for Ulcerative Colitis: A Systematic Review and Meta-Analysis. PLoS One. 2016 Jun 13;11(6):e0157259. doi: 10.1371/journal.pone.0157259. eCollection 2016.
PubMed 27295210Sartor RB. Microbial influences in inflammatory bowel diseases. Gastroenterology. 2008 Feb;134(2):577-94. doi: 10.1053/j.gastro.2007.11.059.
PubMed 18242222Bakhtiar SM, LeBlanc JG, Salvucci E, Ali A, Martin R, Langella P, Chatel JM, Miyoshi A, Bermudez-Humaran LG, Azevedo V. Implications of the human microbiome in inflammatory bowel diseases. FEMS Microbiol Lett. 2013 May;342(1):10-7. doi: 10.1111/1574-6968.12111. Epub 2013 Mar 15.
PubMed 23431991D'Inca R, Annese V, di Leo V, Latiano A, Quaino V, Abazia C, Vettorato MG, Sturniolo GC. Increased intestinal permeability and NOD2 variants in familial and sporadic Crohn's disease. Aliment Pharmacol Ther. 2006 May 15;23(10):1455-61. doi: 10.1111/j.1365-2036.2006.02916.x.
PubMed 16669960
Study at a glance
From the registry record, last updated by the sponsor on 13 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety of FMT in Maintenance of Pediatric Crohn's Disease
- ClinicalTrials.gov ID
- NCT03194529
- Acronym / sponsor ID
- FMTPCD / CHLA-17-00221
- Conditions
- Crohn Disease, Pediatric Crohns Disease
- Keywords
- Crohn's Disease, PCDAI, 16s rRNA, Fecal Microbiota Transplantation, Gut Microbiome, FMT
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Children's Hospital Los AngelesUniversity, hospital or other
- Study start
- 9 Oct 2017 (actual)
- Primary completion
- 30 Jun 2019 (actual)
- Study completion
- 31 Jul 2019 (actual)
- First posted
- 21 Jun 2017 (actual)
- 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.
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.
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.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Primary outcome is safety in children with Crohn's disease in remission.
| Arm | Type | Receives |
|---|---|---|
| FMT in children with disease remission | Experimental | Biological: FMTAll 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. |
Who may qualify
- Ages
- 7 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Age: 7-21 who have been diagnosed with Crohn's disease
- Remission of disease defined as PCDAI <10
- Needs upper GI endoscopy
Key exclusion criteria (5 listed)
- Unwilling to give informed consent/assent
- 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 Crohn's disease (such as neoplastic disease or organ transplantation), have received or are receiving chemotherapy, or have been diagnosed with HIV.
- Subjects with severe food allergies
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 |
|---|---|---|
| FMTAlso: Fecal Microbiota TransplantationFecal Microbiota Transplantation, single dose, 50g of stool, delivered via standard of care upper endoscopy into jejunum. | Biological | FMT in children with disease remission |
Primary outcome
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.
2 secondary outcomes
Crohn's disease activity
Time frame: 24 weeks
Changes in gut microbiome
Time frame: 24 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Mar 2020.
- Background: PubMed 27755214Rinawi F, Assa A, Hartman C, Mozer Glassberg Y, Friedler VN, Rosenbach Y, Silbermintz A, Zevit N, Shamir R. Incidence of Bowel Surgery and Associated Risk Factors in Pediatric-Onset Crohn's Disease. Inflamm Bowel Dis. 2016 Dec;22(12):2917-2923. doi: 10.1097/MIB.0000000000000937.
- Background: PubMed 27295210Shi Y, Dong Y, Huang W, Zhu D, Mao H, Su P. Fecal Microbiota Transplantation for Ulcerative Colitis: A Systematic Review and Meta-Analysis. PLoS One. 2016 Jun 13;11(6):e0157259. doi: 10.1371/journal.pone.0157259. eCollection 2016.
- Background: PubMed 18242222Sartor RB. Microbial influences in inflammatory bowel diseases. Gastroenterology. 2008 Feb;134(2):577-94. doi: 10.1053/j.gastro.2007.11.059.
- Background: PubMed 23431991Bakhtiar SM, LeBlanc JG, Salvucci E, Ali A, Martin R, Langella P, Chatel JM, Miyoshi A, Bermudez-Humaran LG, Azevedo V. Implications of the human microbiome in inflammatory bowel diseases. FEMS Microbiol Lett. 2013 May;342(1):10-7. doi: 10.1111/1574-6968.12111. Epub 2013 Mar 15.
- Background: PubMed 16669960D'Inca R, Annese V, di Leo V, Latiano A, Quaino V, Abazia C, Vettorato MG, Sturniolo GC. Increased intestinal permeability and NOD2 variants in familial and sporadic Crohn's disease. Aliment Pharmacol Ther. 2006 May 15;23(10):1455-61. doi: 10.1111/j.1365-2036.2006.02916.x.
- Individual participant dataSharing planned: No
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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.
Sonia Michail, MD principal investigator
Children's 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 1/2 study testing FMT in people with Crohn Disease and Pediatric Crohns Disease, 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 5 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 13 Mar 2020. 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 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] (time frame: 24 weeks). 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 NCT03194529, which also lists 5 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 13 Mar 2020.