Fistula in AnoPhase 1NCT03449069
Pediatric MSC-AFP Sub-study for Crohn's Fistula
A Phase 1 study testing MSC-AFP in people with Fistula in Ano and Crohn Disease, run by Mayo Clinic at 1 site in United States.
Trial summary
The purpose of this study is to determine the safety of using an autologous mesenchymal stromal cell (MSC) coated fistula plug in people with fistulizing Crohn's disease. Autologous means these cells to coat the plug come from the patient. Read more about this study
- Who is it for?
- Ages 12 to 17 years
- Conditions the study is for
- Fistula in Ano, Crohn Disease
- What is being tested?
- MSC-AFP
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- 23 Jul 2018to 31 Dec 2023
- 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-aminosalicylate (5-ASA) drugs, thiopurines, methotrexate (MTX), antibiotics, and anti-tumor necrosis factor (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 purpose of this study is to determine the safety of using an autologous mesenchymal stromal cell (MSC) coated fistula plug in people with fistulizing Crohn's disease. Autologous means these cells to coat the plug come from the 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. 5 patients (age 12 to 17 years) with Crohn's 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.
- Conditions
- Fistula in Ano, Crohn Disease
- Being tested
- MSC-AFP
- Medical subject headings
- Rectal Fistula, Crohn Disease
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 anal fistula plug
Patients will have a mesenchymal stromal cell coated anal fistula plug placed in their Crohn's perianal fistula tract.
| Study group | Role | What they receive |
|---|---|---|
| MSC-AFP | Experimental | This is a single treatment group study. All patients will receive treatment of a stem cell coated fistula plug. 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 done at each study visit to monitor for adverse events.
Measured: 2 months to approximately at 24 months
Secondary outcomes (2)
Number of participants with response to the treatment regarding cessation of drainage from the treated fistula.
Participants will have a clinical assessment of fistula drainage at each study visit.
Measured: 2 months to approximately at 24 months
Number of participants with radiographic response to the treatment regarding the treated fistula.
Participants will have an assessment of healing radiographically by magnetic resonance imaging (MRI) at Week 2 visit, Week 8, and Week 24.
Measured: Week 2, Week 8, and Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Feb 2018
- Study start23 Jul 2018
- Primary completion31 Dec 2023
- Study completion31 Dec 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fistula in AnoStudies 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
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Michael C. Stephens, Mayo Clinic
- Investigators
- Michael C Stephens, Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03449069
- Sponsor's protocol ID
- 17-010539
- Status last verified
- Mar 2024
- First submitted
- 22 Feb 2018
- Last updated
- 13 Mar 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Mar 2024. 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
- NCT03449069
- Acronym / sponsor ID
- 17-010539
- Conditions
- Fistula in Ano, Crohn Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Study start
- 23 Jul 2018 (actual)
- Primary completion
- 31 Dec 2023 (actual)
- Study completion
- 31 Dec 2023 (actual)
- First posted
- 28 Feb 2018 (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 purpose of this study is to determine the safety of using an autologous mesenchymal stromal cell (MSC) coated fistula plug in people with fistulizing Crohn's disease. Autologous means these cells to coat the plug come from the 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. 5 patients (age 12 to 17 years) with Crohn's 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.
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 | Experimental | Drug: MSC-AFPThis is a single treatment group study. All patients will receive treatment of a stem cell coated fistula plug. |
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-aminosalicylate (5-ASA) drugs, thiopurines, methotrexate (MTX), antibiotics, and anti-tumor necrosis factor (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 |
|---|---|---|
| MSC-AFPAlso: mesenchymal stromal cell coated anal fistula plugPatients will have a mesenchymal stromal cell coated anal fistula plug placed in their Crohn's perianal fistula tract. | Drug | MSC-AFP |
Primary outcome
Number of participants with treatment-related adverse events (safety and toxicity)
Time frame: 2 months to approximately at 24 months
Participants will have a health assessment and blood work done at each study visit to monitor for adverse events.
2 secondary outcomes
Number of participants with response to the treatment regarding cessation of drainage from the treated fistula.
Time frame: 2 months to approximately at 24 months
Number of participants with radiographic response to the treatment regarding the treated fistula.
Time frame: Week 2, Week 8, and Week 24
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 2024.
- Mayo Clinic Clinical TrialsLink provided by the sponsor.
- 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.
Michael C Stephens 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 Fistula in Ano and Crohn Disease, 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 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: finished, as of the sponsor's last update on 13 Mar 2024. 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 months to approximately at 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 13 Mar 2024.