Clinical trial NCT01915927: Stem Cell Fistula Plug in Perianal Crohn's Disease
Official title: A Phase 1 Study of Autologous Mesenchymal Stromal Cell Coated Fistula Plug in Patients With Fistulizing Crohn's Disease.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Perianal Crohn's Disease
Lead sponsor: Mayo Clinic
Sponsor class: OTHER
First posted: 2013-08-05
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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. 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.


Eligibility criteria: Inclusion Criteria

* 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
* Ability to comply with protocol
* Competent and able to provide written informed consent
* Must have failed standard medical therapy including anti-TNF agents

Exclusion Criteria

* 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
* 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)

Primary outcomes:
measure: To determine the safety and toxicity of using autologous MSC coated fistula plug in patients with fistulizing Crohn's Disease.
timeFrame: 2-24 months
Secondary outcomes:
measure: To assess in preliminary fashion the response of fistula healing induced by the GORE plug containing MSC
timeFrame: 2-24 months
Other outcomes:
Locations:
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
Study officials:
affiliation: Mayo Clinic
name: William Faubion, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.