Perianal FistulaPhase 1NCT03014219
Phase 1 Crohn's Pediatric Sub-study of MSC AFP
A Phase 1 study testing Only 1 arm: treatment with MSC-AFP in people with Perianal Fistula, run by William A. Faubion, M.D. at 1 site in United States.
Trial summary
The investigators propose to study the safety of autologous mesenchymal stromal cell transfer using a biomatrix (the Gore Bio-A Fistula Plug) in a Phase I study using a single dose of 20 million cells. 20 patients (age 12 to 17 years) with Crohns perianal fistulas will be enrolled. Read more about this study
- Who is it for?
- Ages 12 to 17 years
- Conditions the study is for
- Perianal Fistula
- What is being tested?
- Only 1 arm: treatment with MSC-AFP
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2017to Jul 2020
- 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 12 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Males and females 12-17 years of age.
- Residents of the United States.
- Crohn's disease with single or multiple draining complex perianal fistulae (definition as below) for at least three months despite standard therapy (definition below).
- Concurrent therapies with corticosteroids, 5-ASA drugs, thiopurines, MTX, antibiotics, and anti-TNF therapy are permitted.
- All patients should have undergone a colonoscopy in last 12 months to rule out malignant or premalignant condition
- Have no contraindications to MR evaluations: e.g. pacemaker or magnetically active metal fragments, claustrophobia
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- Ability to comply with protocol
- Competent and able to provide written informed consent (and assent where appropriate).
- Must have failed standard medical therapy including anti-TNF agents
Who cannot join
- Inability to obtain informed consent (and assent where appropriate).
- Clinically significant medical conditions within the six months before administration of MSCs: e.g. sepsis, pneumonia active serious infection or other conditions that would, in the opinion of the investigators, compromise the safety of the patient.
- Specific exclusions;
- Evidence of hepatitis B, C, or HIV
- History of cancer including melanoma (with the exception of localized skin cancers)
- Investigational drug within thirty (30) days of baseline
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- A resident outside the United States
- Pregnant or trying to become pregnant, or breast feeding.
- History of clinically significant auto-immunity (other than Crohn's disease) or any previous example of fat-directed autoimmunity
- Previous allergic reaction to a perianal fistula plug.
- If adipose tissue is not technically feasible
- Weight less than 35 kg
- Allergic to local anesthetics
- Non-enterocutaneous tracts (i.e. recto-vaginal, entero-vesicular)
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 Phase I Study of Autologous Mesenchymal Stromal Cell Coated Fistula Plug in Patients With Fistulizing Crohn's Disease: Pediatric Sub-study
The investigators propose to study the safety of autologous mesenchymal stromal cell transfer using a biomatrix (the Gore Bio-A Fistula Plug) in a Phase I study using a single dose of 20 million cells. 20 patients (age 12 to 17 years) with Crohns perianal fistulas will be enrolled.
Subjects will undergo standard adjuvant therapy including drainage of infection and placement of a draining seton. Six weeks post placement of the draining seton, the seton will be replaced with the MSC loaded Gore fistula plug as per current clinical practice. The subjects will be subsequently followed for fistula response and closure for 24 months. This is an autologous product derived from the patient and used only for the same patient.
Read the detailed description
Visit 1: Patients will be evaluated for eligibility (inclusion/exclusion checklist) and written, informed consent will be obtained. Patients will undergo general exam with vital signs. Patients will be scheduled for a fat biopsy to collect the tissue needed to grow MSC. In the event there is no cell growth from the tissue obtained from the first biopsy, one further attempt will made from a second tissue sample from this patient. However, if the second attempt fails to grow cells, no further attempts will be made, and the subject will not continue in the study.
The subjects will be subsequently followed for fistula response and closure for 24 months. Study visits are Day 1, Week 2, Week 4, Week 8, Week 12, Week 24, Week 52, and Week 104. This is an autologous product derived from the patient and used only for the same patient.
- Conditions
- Perianal Fistula
- Being tested
- Only 1 arm: treatment with MSC-AFP
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Only 1 arm: treatment with MSC-AFPDrug
Also called mesenchymal stromal cells
Eligible patients will be treated with a fistula plug that has been coated with autologous mesenchymal stromal cell. Drug study. Single dose of 20 million cells.
| Study group | Role | What they receive |
|---|---|---|
| Only 1 arm: treatment with MSC-AFP | Experimental | Single Treatment Group: Eligible patients will be treated with a Gore Bio-A Fistula Plug that has been coated with autologous mesenchymal stromal cells. This is a drug study, specifically phase 1 study of autologous mesenchymal stromal cells. Single dose of 20 million cells. Receives: Drug: Only 1 arm: treatment with MSC-AFP |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Subjects with any Adverse Events that are related to study drug
The primary endpoint of this study is to determine the safety of using adipose derived, autologous mesenchymal stromal cells (MSC) bound to the Gore Bio-A Fistula Plug for treatment of refractory CD perianal fistulae.
Measured: [Time Frame: 2-24 months
Secondary outcomes (1)
Number of Subjects with healing in response to the study drug treatment
The Outcome Measure will be the presence or absence of fistula drainage (Yes vs. No)
Measured: Time Frame: 2-24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jan 2017 (estimated)
- Study startJul 2017 (estimated)
- Primary completionJul 2019 (estimated)
- Study completionJul 2020 (estimated)
Why it stopped: FDA approval was not obtained until adult trial completes accrual
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Perianal FistulaStudies 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
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- William A. Faubion, M.D.
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, William A. Faubion, M.D., Mayo Clinic
- Investigators
- Michael C Stephens, Principal investigator, Mayo ClinicWilliam Faubion, Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03014219
- Sponsor's protocol ID
- 15-005574
- Status last verified
- Jul 2017
- First submitted
- 4 Jan 2017
- Last updated
- 6 Jul 2017
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Jul 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Study of Autologous Mesenchymal Stromal Cell Coated Fistula Plug in Patients With Fistulizing Crohn's Disease: Pediatric Sub-study
- ClinicalTrials.gov ID
- NCT03014219
- Acronym / sponsor ID
- 15-005574
- Conditions
- Perianal Fistula
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: FDA approval was not obtained until adult trial completes accrual
- Enrolment
- 0 participants (actual)
- Lead sponsor
- William A. Faubion, M.D.University, hospital or other
- Study start
- Jul 2017 (estimated)
- Primary completion
- Jul 2019 (estimated)
- Study completion
- Jul 2020 (estimated)
- First posted
- 9 Jan 2017 (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 investigators propose to study the safety of autologous mesenchymal stromal cell transfer using a biomatrix (the Gore Bio-A Fistula Plug) in a Phase I study using a single dose of 20 million cells. 20 patients (age 12 to 17 years) with Crohns perianal fistulas will be enrolled.
Subjects will undergo standard adjuvant therapy including drainage of infection and placement of a draining seton. Six weeks post placement of the draining seton, the seton will be replaced with the MSC loaded Gore fistula plug as per current clinical practice. The subjects will be subsequently followed for fistula response and closure for 24 months. This is an autologous product derived from the patient and used only for the same patient.
Detailed description
Visit 1: Patients will be evaluated for eligibility (inclusion/exclusion checklist) and written, informed consent will be obtained. Patients will undergo general exam with vital signs. Patients will be scheduled for a fat biopsy to collect the tissue needed to grow MSC. In the event there is no cell growth from the tissue obtained from the first biopsy, one further attempt will made from a second tissue sample from this patient. However, if the second attempt fails to grow cells, no further attempts will be made, and the subject will not continue in the study.
The subjects will be subsequently followed for fistula response and closure for 24 months. Study visits are Day 1, Week 2, Week 4, Week 8, Week 12, Week 24, Week 52, and Week 104. This is an autologous product derived from the patient and used only for the same patient.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Only 1 arm: treatment with MSC-AFP | Experimental | Drug: Only 1 arm: treatment with MSC-AFPSingle Treatment Group: Eligible patients will be treated with a Gore Bio-A Fistula Plug that has been coated with autologous mesenchymal stromal cells. This is a drug study, specifically phase 1 study of autologous mesenchymal stromal cells. Single dose of 20 million cells. |
Who may qualify
- Ages
- 12 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- Males and females 12-17 years of age.
- Residents of the United States.
- Crohn's disease with single or multiple draining complex perianal fistulae (definition as below) for at least three months despite standard therapy (definition below).
- Concurrent therapies with corticosteroids, 5-ASA drugs, thiopurines, MTX, antibiotics, and anti-TNF therapy are permitted.
- All patients should have undergone a colonoscopy in last 12 months to rule out malignant or premalignant condition
Key exclusion criteria (14 listed)
- Inability to obtain informed consent (and assent where appropriate).
- Clinically significant medical conditions within the six months before administration of MSCs: e.g. sepsis, pneumonia active serious infection or other conditions that would, in the opinion of the investigators, compromise the safety of the patient.
- Specific exclusions;
- History of cancer including melanoma (with the exception of localized skin cancers)
- Investigational drug within thirty (30) days of baseline
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 |
|---|---|---|
| Only 1 arm: treatment with MSC-AFPAlso: mesenchymal stromal cellsEligible patients will be treated with a fistula plug that has been coated with autologous mesenchymal stromal cell. Drug study. Single dose of 20 million cells. | Drug | Only 1 arm: treatment with MSC-AFP |
Primary outcome
Number of Subjects with any Adverse Events that are related to study drug
Time frame: [Time Frame: 2-24 months
The primary endpoint of this study is to determine the safety of using adipose derived, autologous mesenchymal stromal cells (MSC) bound to the Gore Bio-A Fistula Plug for treatment of refractory CD perianal fistulae.
1 secondary outcome
Number of Subjects with healing in response to the study drug treatment
Time frame: Time Frame: 2-24 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jul 2017.
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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.
Michael C Stephens principal investigator
Mayo Clinic
William Faubion 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 Only 1 arm: treatment with MSC-AFP in people with Perianal Fistula, run by William A. Faubion, M.D. 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 9 inclusion and 14 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: withdrawn, as of the sponsor's last update on 6 Jul 2017. 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 was withdrawn before enrolling participants, so there are no results.
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 Subjects with any Adverse Events that are related to study drug (time frame: [Time Frame: 2-24 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 6 Jul 2017.