Perianal FistulaPhase 1NCT02589119
Stem Cell Fistula Plug in Cryptoglandular Perianal Fistulas (MSC-AFP)
A Phase 1 study testing MSC-AFP in people with Perianal Fistula and Cryptoglandular Perianal Fistula, run by Mayo Clinic 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 Fistula Plug) in a Phase I study using a single dose of 20 million cells. 15 adult patients, ages 18 years and older with cryptoglandular fistulas will be enrolled. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Perianal Fistula, Cryptoglandular Perianal Fistula
- What is being tested?
- MSC-AFP
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2016to Sep 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.
- Single-tract, transsphincteric anal fistula of cryptoglandular origin. Primary fistulas (no previous surgical treatment) and those who have failed previous surgical repairs
- Have no contraindications to MR evaluations: e.g. pacemaker or magnetically active metal fragments, claustrophobia
- Ability to comply with protocol
- Competent and able to provide written informed consent
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
Show all 15Show fewer
- Pregnant or breast feeding.
- History of clinically significant auto-immunity (e.g. 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 (i.e. recto-vaginal, entero-vesicular)
- Multi-tract, suprasphincteric and extrasphincteric fistula tract extensions
- Active local infection associated with the fistula
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 High, Transsphincteric Cryptoglandular Perianal Fistulas
The investigators propose to study the safety of autologous mesenchymal stromal cell transfer using a biomatrix (the Gore Fistula Plug) in a Phase I study using a single dose of 20 million cells. 15 adult patients, ages 18 years and older with cryptoglandular 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; 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
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. 15 adult patients (age \> 18 years) with cryptoglandular 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. 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, Cryptoglandular Perianal Fistula
- Being tested
- MSC-AFP
- Keywords
- cryptoglandular, perianal fistula, cryptoglandular perianal fistulas
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MSC-AFPDrug
Also called mesenchymal stromal cell coated fistula plug
Eligible patients will be treated with a fistula plug that has been coated with autologous mesenchymal stromal cell.
| Study group | Role | What they receive |
|---|---|---|
| MSC-AFP | Other | Single Treatment Group Receives: Drug: MSC-AFP |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with treatment-related adverse events (safety and toxicity).
Participants will have a health assessment and blood work measured at each study visit to monitor for adverse events, such as worsening of the cryptoglandular fistulizing disease, abnormal laboratory values, or significant abnormalities in physical examination. The Outcome Measure will be the number of Participants With Abnormal Laboratory Values and/or Adverse Events That Are Related to Treatment
Measured: 2-24 months
Secondary outcomes (1)
Number of participants with response to the treatment regarding potential cessation of drainage from their fistula.
Participants will have a clinical assessment of fistula drainage. Participants will be assessed during an office examination if their fistula is draining or not. The Outcome Measure will be the presence or absence of fistula drainage.
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 posted28 Oct 2015 (estimated)
- Study startJan 2016
- Primary completionSep 2019
- Study completionSep 2019
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.
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Eric J. Dozois, M.D., Mayo Clinic
- Investigators
- Eric J Dozois, MD, Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02589119
- Sponsor's protocol ID
- 15-003200
- Status last verified
- Dec 2019
- First submitted
- 21 Oct 2015
- Last updated
- 6 Dec 2019
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 6 Dec 2019. 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 High, Transsphincteric Cryptoglandular Perianal Fistulas
- ClinicalTrials.gov ID
- NCT02589119
- Acronym / sponsor ID
- 15-003200
- Conditions
- Perianal Fistula, Cryptoglandular Perianal Fistula
- Keywords
- cryptoglandular, perianal fistula, cryptoglandular perianal fistulas
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Study start
- Jan 2016
- Primary completion
- Sep 2019 (actual)
- Study completion
- Sep 2019 (actual)
- First posted
- 28 Oct 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 investigators propose to study the safety of autologous mesenchymal stromal cell transfer using a biomatrix (the Gore Fistula Plug) in a Phase I study using a single dose of 20 million cells. 15 adult patients, ages 18 years and older with cryptoglandular 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; 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
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. 15 adult patients (age \> 18 years) with cryptoglandular 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. 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 |
|---|---|---|
| MSC-AFP | Other | Drug: MSC-AFPSingle Treatment Group |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Males and females 18-65 years of age.
- Residents of the United States.
- Single-tract, transsphincteric anal fistula of cryptoglandular origin. Primary fistulas (no previous surgical treatment) and those who have failed previous surgical repairs
- Have no contraindications to MR evaluations: e.g. pacemaker or magnetically active metal fragments, claustrophobia
- Ability to comply with protocol
Key exclusion criteria (15 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-AFPAlso: mesenchymal stromal cell coated fistula plugEligible patients will be treated with a fistula plug that has been coated with autologous mesenchymal stromal cell. | Drug | MSC-AFP |
Primary outcome
Number of participants with treatment-related adverse events (safety and toxicity).
Time frame: 2-24 months
Participants will have a health assessment and blood work measured at each study visit to monitor for adverse events, such as worsening of the cryptoglandular fistulizing disease, abnormal laboratory values, or significant abnormalities in physical examination. The Outcome Measure will be the number of Participants With Abnormal Laboratory Values and/or Adverse Events That Are Related to Treatment
1 secondary outcome
Number of participants with response to the treatment regarding potential cessation of drainage from their fistula.
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 6 Dec 2019.
- Mayo Clinic Clinical TrialsLink provided by the sponsor.
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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.
Eric J Dozois, 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 Fistula and Cryptoglandular Perianal Fistula, 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 6 inclusion and 15 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 Dec 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?
Number of participants with treatment-related adverse events (safety and toxicity). (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 Dec 2019.