Clostridium DifficilePhase 1NCT02423967
Fresh Versus Frozen Stool for Fecal Transplant in Children
A Phase 1 study testing Transplant uses frozen anonymous stool and Transplant uses fresh familial stool in people with Clostridium Difficile, run by Mayo Clinic at 1 site in United States.
Trial summary
The primary goal of this study will be to assess whether stool collected and frozen from anonymous screened unrelated donors can be as effective as stool freshly collected from recipient's parents when used in Fecal Microbial Transplant for the eradication of recurrent Clostridium difficile infections in children. Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Clostridium Difficile
- What is being tested?
- Transplant uses frozen anonymous stool and Transplant uses fresh familial stool
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 28 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2015to 4 Mar 2019
- 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 1 year to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age 1-18 years
- Diarrhea
- Positive Clostridium difficile infection using stool toxin testing
- Diarrhea that has improved on antibiotics but recurs when antibiotics are stopped.
- Willingness to undergo Fecal Microbial Transplant using frozen stool from anonymous screened donors.
Who cannot join
- Age under 1 or over 18
- No diarrhea
Trial locations and contacts
1 site in 1 country.
United States1 site
Mayo Clinic in Rochester
Rochester, Minnesota, 55905
About this study
Official title: A Comparison of Efficacy of Fresh Versus Frozen Donor Stool for Fecal Microbial Transplant in Children With Recurrent Clostridium Difficile Infections
The primary goal of this study will be to assess whether stool collected and frozen from anonymous screened unrelated donors can be as effective as stool freshly collected from recipient's parents when used in Fecal Microbial Transplant for the eradication of recurrent Clostridium difficile infections in children. In the current protocols, which are more than 90% effective, each child who is receiving a fecal transplant has to provide their own donor stool, usually from a parent or close relative. This requires considerable screening costs for each case and is logistically complicated as the donor must be present and must stool just prior to the transplant. The investigators hope to show that a small number of healthy donors can provide stool samples which can be frozen and banked and then thawed for use in numerous patients. The primary goal is to show that Clostridium difficile will be eradicated as effectively (Greater than 90% success) when using the stool from the frozen donors.
The study will also evaluate the inflammatory response and intestinal microbiome in young children aged 1-3 years with Clostridium difficile infections to better predict which ones will respond to fecal transplantation and which ones have incidental infections. For this question the investigators will gather stool samples to check for lactoferrin, calprotectin, and alpha1antitrypsin, and 16s ribosomal RNA analysis in children before and after the fecal transplants. The goal is to see if there is an intestinal microbiome that predisposes some children to getting sick from Clostridium difficile versus just having it incidentally.
Read the detailed description
The study is designed to enroll forty children aged 1-18 with recurrent Clostridium difficile Infection (CDI). Recurrent CDI is defined as an infection that persists after three rounds of appropriate antibiotics. Enrollees will be randomly assigned to receive Fecal Microbial Transplant using stool from either a screened relative as per current protocols versus using frozen stool that has been collected from healthy volunteers.
Primary outcome will be the eradication of the Clostridium difficile as defined by elimination of diarrhea with a negative Clostridium difficile toxin stool test.
Secondary outcomes will include a measure of quality of life pre and post Fecal Microbial Transplant using a validated quality of life survey instrument, the Health Act Child Health Questionnaire. The investigators will also assess for inflammation in the stool using lactoferrin and calprotectin and evaluate the stool microbiome in recipients pre and post transplant using sequencing to look for any pattern that predicts successful eradication.
- Conditions
- Clostridium Difficile
- Being tested
- Transplant uses frozen anonymous stool, Transplant uses fresh familial stool
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Transplant uses frozen anonymous stoolBiological
Also called fecal microbial transplant
The intervention is using frozen anonymous donor stool instead of fresh stool from family members for the fecal microbial transplant to treat recurrent CDI.
Transplant uses fresh familial stoolBiological
Also called fecal microbila transplant
The intervention is using fresh familial donor stool for the fecal microbial transplant to treat recurrent CDI
| Study group | Role | What they receive |
|---|---|---|
| Transplant uses fresh familial stool | Active comparator | Undergoes Fecal Microbial Transplant using fresh stool from a screened family member Followed up and assessed for eradication of Clostridium difficile Quality of life assessed Stool assessed for inflammation and microbiome Receives: Biological: Transplant uses fresh familial stool |
| Transplant uses frozen anonymous stool | Experimental | Undergoes Fecal Microbial Transplant using stool collected from screened anonymous donor that has been frozen until time of Fecal Microbial Transplant Followed up and assessed for eradication of Clostridium difficile Quality of life assessed Stool assessed for inflammation and microbiome Receives: Biological: Transplant uses frozen anonymous stool |
What is being measured
The primary outcome is the main question the study is designed to answer.
Clostridium difficile (CD) Eradication; percentage of patients with negative CD toxin in stool following Fecal Microbiota Transplant
Will analyze stool sample collected 4 weeks after fecal microbiota transplant (FMT) for presence or absence of Clostridium difficile toxin. In addition, will note whether presenting symptoms improved or not after FMT.
Measured: 4 weeks
Secondary outcomes (3)
Changes in quality of life questionnaire scores after Fecal Microbiota Transplant
Will assess quality of life using standardized Child health Questionnaire which provides a raw score of 0-100. Investigators will compare scores of all patients before Fecal Transplant and 12 weeks after in order to mark the impact of procedure outcome on children's physical and psychological health
Measured: 12 weeks
percentage of patients with stool Calprotectin levels exceeding 50 mcg/g after Fecal Microbiota Transplant
Will assess fecal Calprotectin levels in stools prior to fecal transplant and 4 weeks after
Measured: 4 weeks
percentage of patients with positive Lactoferrin inflammatory marker in stools after Fecal Microbiota Transplant
Analyze stool samples for presence or absence Lactoferrin before Fecal transplant and 4 weeks after
Measured: 4 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Apr 2015 (estimated)
- Study startApr 2015
- Primary completion4 Mar 2019
- Study completion4 Mar 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Clostridium DifficileStudies of the same condition, open and under way.
- More trials run by Mayo ClinicThe 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
- None (open label)
- Enrolment
- 28 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Mark G. Bartlett, M.D., Mayo Clinic
- Investigators
- Mark Bartlett, MD, Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02423967
- Sponsor's protocol ID
- 14-004472
- Status last verified
- Mar 2019
- First submitted
- 6 Apr 2015
- Last updated
- 6 Mar 2019
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Comparison of Efficacy of Fresh Versus Frozen Donor Stool for Fecal Microbial Transplant in Children With Recurrent Clostridium Difficile Infections
- ClinicalTrials.gov ID
- NCT02423967
- Acronym / sponsor ID
- 14-004472
- Conditions
- Clostridium Difficile
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 28 participants (actual)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Study start
- Apr 2015
- Primary completion
- 4 Mar 2019 (actual)
- Study completion
- 4 Mar 2019 (actual)
- First posted
- 22 Apr 2015 (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.
The primary goal of this study will be to assess whether stool collected and frozen from anonymous screened unrelated donors can be as effective as stool freshly collected from recipient's parents when used in Fecal Microbial Transplant for the eradication of recurrent Clostridium difficile infections in children. In the current protocols, which are more than 90% effective, each child who is receiving a fecal transplant has to provide their own donor stool, usually from a parent or close relative. This requires considerable screening costs for each case and is logistically complicated as the donor must be present and must stool just prior to the transplant. The investigators hope to show that a small number of healthy donors can provide stool samples which can be frozen and banked and then thawed for use in numerous patients. The primary goal is to show that Clostridium difficile will be eradicated as effectively (Greater than 90% success) when using the stool from the frozen donors.
The study will also evaluate the inflammatory response and intestinal microbiome in young children aged 1-3 years with Clostridium difficile infections to better predict which ones will respond to fecal transplantation and which ones have incidental infections. For this question the investigators will gather stool samples to check for lactoferrin, calprotectin, and alpha1antitrypsin, and 16s ribosomal RNA analysis in children before and after the fecal transplants. The goal is to see if there is an intestinal microbiome that predisposes some children to getting sick from Clostridium difficile versus just having it incidentally.
Detailed description
The study is designed to enroll forty children aged 1-18 with recurrent Clostridium difficile Infection (CDI). Recurrent CDI is defined as an infection that persists after three rounds of appropriate antibiotics. Enrollees will be randomly assigned to receive Fecal Microbial Transplant using stool from either a screened relative as per current protocols versus using frozen stool that has been collected from healthy volunteers.
Primary outcome will be the eradication of the Clostridium difficile as defined by elimination of diarrhea with a negative Clostridium difficile toxin stool test.
Secondary outcomes will include a measure of quality of life pre and post Fecal Microbial Transplant using a validated quality of life survey instrument, the Health Act Child Health Questionnaire. The investigators will also assess for inflammation in the stool using lactoferrin and calprotectin and evaluate the stool microbiome in recipients pre and post transplant using sequencing to look for any pattern that predicts successful eradication.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Transplant uses fresh familial stool | Active comparator | Biological: Transplant uses fresh familial stoolUndergoes Fecal Microbial Transplant using fresh stool from a screened family member Followed up and assessed for eradication of Clostridium difficile Quality of life assessed Stool assessed for inflammation and microbiome |
| Transplant uses frozen anonymous stool | Experimental | Biological: Transplant uses frozen anonymous stoolUndergoes Fecal Microbial Transplant using stool collected from screened anonymous donor that has been frozen until time of Fecal Microbial Transplant Followed up and assessed for eradication of Clostridium difficile Quality of life assessed Stool assessed for inflammation and microbiome |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Age 1-18 years
- Diarrhea
- Positive Clostridium difficile infection using stool toxin testing
- Diarrhea that has improved on antibiotics but recurs when antibiotics are stopped.
- Willingness to undergo Fecal Microbial Transplant using frozen stool from anonymous screened donors.
Key exclusion criteria (2 listed)
- Age under 1 or over 18
- No diarrhea
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 |
|---|---|---|
| Transplant uses frozen anonymous stoolAlso: fecal microbial transplantThe intervention is using frozen anonymous donor stool instead of fresh stool from family members for the fecal microbial transplant to treat recurrent CDI. | Biological | Transplant uses frozen anonymous stool |
| Transplant uses fresh familial stoolAlso: fecal microbila transplantThe intervention is using fresh familial donor stool for the fecal microbial transplant to treat recurrent CDI | Biological | Transplant uses fresh familial stool |
Primary outcome
Clostridium difficile (CD) Eradication; percentage of patients with negative CD toxin in stool following Fecal Microbiota Transplant
Time frame: 4 weeks
Will analyze stool sample collected 4 weeks after fecal microbiota transplant (FMT) for presence or absence of Clostridium difficile toxin. In addition, will note whether presenting symptoms improved or not after FMT.
3 secondary outcomes
Changes in quality of life questionnaire scores after Fecal Microbiota Transplant
Time frame: 12 weeks
percentage of patients with stool Calprotectin levels exceeding 50 mcg/g after Fecal Microbiota Transplant
Time frame: 4 weeks
percentage of patients with positive Lactoferrin inflammatory marker in stools after Fecal Microbiota Transplant
Time frame: 4 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 6 Mar 2019.
- Mayo Clinic Clinical TrialsLink provided by the sponsor.
Related trials for Clostridium Difficile
Not yet recruitingObservational study
Epidemiology of Clostridioides Difficile Infection (CDI) in Long-term Care Facilities, Assessment of Professional Practices Related to CDI Risk Prevention, and Strategies for Improving Care.
Clostridioides Difficile Infection
Active, not recruitingPhase 2
Autologous Fecal Microbiota Transplantation (Auto-FMT) for Prophylaxis of Clostridium Difficile Infection in Recipients of Allogeneic Hematopoietic Stem Cell Transplantation
Allogeneic Hematopoietic Stem Cell
RecruitingPhase 3
A Study to Learn About a Clostridioides Difficile Vaccine in People 65 Years of Age and Older
Clostridoides Difficile Associated Disease
Active, not recruitingObservational study
C. Difficile Toxin Levels in Stool From Healthy Individuals Following Standard of Care Antibiotic Treatment for CDI.
Clostridioides Difficile Infection Recurrence
RecruitingPhase 3
VE303 for Prevention of Recurrent Clostridioides Difficile Infection
Clostridium Difficile, Clostridium Difficile Infections
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.
Mark Bartlett, MD principal investigator
Mayo Clinic
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 Transplant uses frozen anonymous stool and Transplant uses fresh familial stool in people with Clostridium Difficile, run by Mayo Clinic 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 5 inclusion and 2 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 6 Mar 2019. 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?
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?
Clostridium difficile (CD) Eradication; percentage of patients with negative CD toxin in stool following Fecal Microbiota Transplant (time frame: 4 weeks). 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 6 Mar 2019.