Perianal Crohn's DiseasePhase 1MSC-AFPNCT01915927
Stem Cell Fistula Plug in Perianal Crohn's Disease
A Phase 1 study testing MSC-AFP in people with Perianal Crohn's Disease, 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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Perianal Crohn's Disease
- What is being tested?
- MSC-AFP
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2013to Dec 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Males and females 18-65 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
- Must have failed standard medical therapy including anti-TNF agents
Who cannot join
- Inability to give informed consent.
- Clinically significant medical conditions within the six months before administration of MSCs: e.g. myocardial infarction, active angina, congestive heart failure 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
- A resident outside the United States
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- 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 liposuction is not technically feasible
- Allergic to local anesthetics
- Pregnant patients or trying to become pregnant.
- Non-enterocutaneous tracts (ie recto-vaginal, entero-vesicular)
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 1 Study of Autologous Mesenchymal Stromal Cell Coated Fistula Plug in Patients With Fistulizing Crohn's Disease.
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. Twenty adult patients (age 18 years or older) with refractory, complicated perianal fistulizing Crohn's disease will be enrolled. Subjects will undergo standard adjuvant therapy including drainage of infection and placement of a draining seton with continuation of pre-existing anti-Crohn's therapy. Six weeks post placement of the draining seton, the seton will be replaced with the MSC loaded Gore® Bio-A® 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.
- Conditions
- Perianal Crohn's Disease
- Being tested
- MSC-AFP
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MSC-AFPDrug
| Study group | Role | What they receive |
|---|---|---|
| 1 | Other | Only 1 arm: treatment with MSC-AFP Receives: Drug: MSC-AFP |
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine the safety and toxicity of using autologous MSC coated fistula plug in patients with fistulizing Crohn's Disease.
The primary endpoint of this study is to determine the safety and feasibility 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: 2-24 months
Secondary outcomes (1)
To assess in preliminary fashion the response of fistula healing induced by the GORE plug containing MSC
To assess in preliminary fashion the response of fistula healing induced by the GORE plug containing MSC i) Clinically by drainage assessment and ii) Radiographically by MRI, the gold standard test for assessment of healing.
Measured: 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 posted5 Aug 2013 (estimated)
- Study startJul 2013
- Primary completionDec 2019
- Study completionDec 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Perianal Crohn's DiseaseStudies 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
- 20 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
- William Faubion, MD, Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01915927
- Sponsor's protocol ID
- 12-009716
- Acronym
- MSC-AFP
- Status last verified
- Dec 2019
- First submitted
- 31 Jul 2013
- Last updated
- 3 Jan 2020
- Results posted
- No
- Data monitoring committee
- No
Publications
Dietz AB, Dozois EJ, Fletcher JG, Butler GW, Radel D, Lightner AL, Dave M, Friton J, Nair A, Camilleri ET, Dudakovic A, van Wijnen AJ, Faubion WA. Autologous Mesenchymal Stem Cells, Applied in a Bioabsorbable Matrix, for Treatment of Perianal Fistulas in Patients With Crohn's Disease. Gastroenterology. 2017 Jul;153(1):59-62.e2. doi: 10.1053/j.gastro.2017.04.001. Epub 2017 Apr 9.
PubMed 28400193
Study at a glance
From the registry record, last updated by the sponsor on 3 Jan 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Study of Autologous Mesenchymal Stromal Cell Coated Fistula Plug in Patients With Fistulizing Crohn's Disease.
- ClinicalTrials.gov ID
- NCT01915927
- Acronym / sponsor ID
- MSC-AFP / 12-009716
- Conditions
- Perianal Crohn's Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- William A. Faubion, M.D.University, hospital or other
- Study start
- Jul 2013 (actual)
- Primary completion
- Dec 2019 (actual)
- Study completion
- Dec 2019 (actual)
- First posted
- 5 Aug 2013 (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. Twenty adult patients (age 18 years or older) with refractory, complicated perianal fistulizing Crohn's disease will be enrolled. Subjects will undergo standard adjuvant therapy including drainage of infection and placement of a draining seton with continuation of pre-existing anti-Crohn's therapy. Six weeks post placement of the draining seton, the seton will be replaced with the MSC loaded Gore® Bio-A® 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | Other | Drug: MSC-AFPOnly 1 arm: treatment with MSC-AFP |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Males and females 18-65 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 (13 listed)
- Inability to give informed consent.
- Clinically significant medical conditions within the six months before administration of MSCs: e.g. myocardial infarction, active angina, congestive heart failure 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
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 |
|---|---|---|
| MSC-AFP | Drug | 1 |
Primary outcome
To determine the safety and toxicity of using autologous MSC coated fistula plug in patients with fistulizing Crohn's Disease.
Time frame: 2-24 months
The primary endpoint of this study is to determine the safety and feasibility 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
To assess in preliminary fashion the response of fistula healing induced by the GORE plug containing MSC
Time frame: 2-24 months
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 3 Jan 2020.
- Background: PubMed 28400193Dietz AB, Dozois EJ, Fletcher JG, Butler GW, Radel D, Lightner AL, Dave M, Friton J, Nair A, Camilleri ET, Dudakovic A, van Wijnen AJ, Faubion WA. Autologous Mesenchymal Stem Cells, Applied in a Bioabsorbable Matrix, for Treatment of Perianal Fistulas in Patients With Crohn's Disease. Gastroenterology. 2017 Jul;153(1):59-62.e2. doi: 10.1053/j.gastro.2017.04.001. Epub 2017 Apr 9.
- Mayo Clinic Clinical TrialsLink provided by the sponsor.
Related trials for Perianal Crohn's Disease
Active, not recruitingPhase 3
A Study of Guselkumab in Participants With Fistulizing, Perianal Crohn's Disease
Fistulizing Crohns Disease, Perianal Crohns Disease
RecruitingPhase 4
Risankizumab for Refractory Perianal Fistulizing Crohn's Disease
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Recruiting
A Study Comparing Transperineal Ultrasound, Magnetic Resonance Imaging, Endoscopic Ultrasound, and Examination Under Anesthesia to Detect and Classify Perianal Fistulas in Patients With Crohn's Disease or Fistulas of Unknown Cause
Crohn Disease (CD), Cryptoglandular Perianal Fistula
Not yet recruitingPhase 1/2
Hyperbaric Oxygen Therapy for Perianal Fistulizing Crohn's Disease Not Responsive or Intolerant to Conventional Care: HYPNOTIC Pilot Study
Inflammatory Bowel Disease (IBD), Crohn Disease
Not yet recruitingPhase 4
MIRICURE: Mirikizumab With Curettage and Repair (Closure) for Perianal Fistulizing Crohn's Disease
Crohn's Disease, Perianal Fistula
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.
William Faubion, 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 MSC-AFP in people with Perianal Crohn's Disease, 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 13 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 3 Jan 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 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?
To determine the safety and toxicity of using autologous MSC coated fistula plug in patients with fistulizing Crohn's Disease. (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?
On the ClinicalTrials.gov record for NCT01915927, which also lists 1 linked publication.
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 3 Jan 2020.