- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03014219
Phase 1 Crohn's Pediatric Sub-study of MSC AFP
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.
Study Overview
Status
Conditions
Intervention / Treatment
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 Type
Phase
- Phase 1
Contacts and Locations
Study Locations
-
-
Minnesota
-
Rochester, Minnesota, United States, 55905
- Mayo Clinic in Rochester
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria
- 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
- 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
Exclusion Criteria
- 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;
a. 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
- 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)
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Only 1 arm: treatment with MSC-AFP
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.
|
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.
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
|
[Time Frame: 2-24 months
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Number of Subjects with healing in response to the study drug treatment
Time Frame: Time Frame: 2-24 months
|
The Outcome Measure will be the presence or absence of fistula drainage (Yes vs. No)
|
Time Frame: 2-24 months
|
Collaborators and Investigators
Sponsor
Investigators
- Principal Investigator: Michael C Stephens, Mayo Clinic
- Principal Investigator: William Faubion, Mayo Clinic
Study record dates
Study Major Dates
Study Start (Anticipated)
Primary Completion (Anticipated)
Study Completion (Anticipated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- 15-005574
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Perianal Fistula
-
William A. Faubion, M.D.CompletedPerianal Fistula | Cryptoglandular Perianal Fistula | Crohn's Perianal FistulaUnited States
-
Mayo ClinicCompletedPerianal Fistula | Cryptoglandular Perianal FistulaUnited States
-
Tigenix S.A.U.Completed
-
Nordic Drugs ABCompletedPerianal FistulasSweden
-
Instituto de Investigación Hospital Universitario...UnknownExtremely Complex Perianal FistulaeSpain
-
Jiangsu Topcel-KH Pharmaceutical Co., Ltd.Xiangya Hospital of Central South UniversityNot yet recruiting
-
TakedaTakeda Development Center Americas, Inc.RecruitingCrohn's Disease | Complex Perianal FistulaIsrael, Japan, Netherlands, Spain, Poland
-
Tigenix S.A.U.TerminatedCrohn Disease | Complex Perianal FistulaSpain, Netherlands, Austria
-
Tehran University of Medical SciencesActive, not recruitingPerianal Fistula in Patients With Crohn's DiseaseIran, Islamic Republic of
-
dr. IJM Han-GeurtsAcademisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)RecruitingDrain Abscess | Perianal Abscess | Perianal FistulaNetherlands
Clinical Trials on Only 1 arm: treatment with MSC-AFP
-
Boston Scientific CorporationCompletedSudden Cardiac DeathUnited States, Spain, Korea, Republic of, United Kingdom, Canada, Japan, Austria, Italy
-
Neovasc Inc.Shockwave Medical, Inc.RecruitingRefractory AnginaUnited States, Canada
-
Northwestern UniversityNational Institute of Mental Health (NIMH)Not yet recruiting
-
University Hospitals Cleveland Medical CenterWithdrawn
-
Moriel VandsburgerTerminatedMyocardial Fibrosis | Healthy | Chronic Kidney DiseaseUnited States
-
Centro Hospitalar NordesteCompletedColorectal Cancer
-
National Cancer Center, KoreaUnknownLimited Disease Small Cell Lung CancerKorea, Republic of
-
Kanyos Bio, Inc., a wholly-owned subsidiary of...PfizerRecruitingCeliac Disease | Coeliac DiseaseUnited States, Canada
-
metaMe HealthCompletedIrritable Bowel SyndromeUnited States
-
Helsinki University Central HospitalCompletedFracture | ClavicleFinland