Phase 1 Crohn's Pediatric Sub-study of MSC AFP
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|ClinicalTrials.gov Identifier: NCT03014219|
Recruitment Status : Withdrawn (FDA approval was not obtained until adult trial completes accrual)
First Posted : January 9, 2017
Last Update Posted : July 6, 2017
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.
|Condition or disease||Intervention/treatment||Phase|
|Perianal Fistula||Drug: Only 1 arm: treatment with MSC-AFP||Phase 1|
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 :||Interventional (Clinical Trial)|
|Actual Enrollment :||0 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase I Study of Autologous Mesenchymal Stromal Cell Coated Fistula Plug in Patients With Fistulizing Crohn's Disease: Pediatric Sub-study|
|Estimated Study Start Date :||July 2017|
|Estimated Primary Completion Date :||July 2019|
|Estimated Study Completion Date :||July 2020|
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.
Drug: Only 1 arm: treatment with MSC-AFP
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 Name: mesenchymal stromal cells
- 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.
- 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)
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03014219
|United States, Minnesota|
|Mayo Clinic in Rochester|
|Rochester, Minnesota, United States, 55905|
|Principal Investigator:||Michael C Stephens||Mayo Clinic|
|Principal Investigator:||William Faubion||Mayo Clinic|