IRB Study Number 19-081
Status Recruiting
Phase Phase 3
Location Cleveland Clinic Main Campus
Institute Digestive Disease & Surgery Institute
Description
The study objective is to evaluate the efficacy and safety of adipose derived mesenchymal stem cells, Cx601 compared to placebo for the treatment of complex perianal fistula(s) patients with Crohn’s disease (CD) at Week 24 with a follow-up period up to 52 weeks. The treatment is anticipated to prevent one of the main causes of anal incontinence, diminish recurrence of the fistula disease and reduce drastically the significant disorders provoked by the standard fistula surgery in these patients. The procedure does not require hospital stay and avoids the work leave of classic procedures.
Inclusion Criteria
Subject must answer ++YES++ to inclusion criteria to be enrolled in study:
Male or Female ≥18 years and ≤75 years,
Crohn’s Disease diagnosed at least 6 months prior to Screening Visit in accordance with accepted clinical, endoscopic, histological, and/or radiological criteria,
Presence of complex perianal fistula defined by protocol
MRI to confirm location
Fistula draining for at least 6 weeks prior to screening visit
Clinically controlled, non-active, or mildly active Crohn’s Disease for last 6 months
PRO-2 Score <14
Colonoscopy documenting the absence of ulcers larger than 0.5 cm in colonic mucosa
All Crohn’s disease medication has been at a stable dose for at least 3 months
Previously treated perianal fistula with inadequate response, loss of response, or documented intolerance to any of the following: Yes 3 month treatment with azathioprine, 6-mercaptopurine, or subcutaneous/intramuscular methotrexate, OR 14 week treatment with Infliximab, Adalimumab, Certolizumab, or anti-integrins, OR 16 week treatment with anti-IL-12/23s prior to screening
Women of Childbearing Potential (WCBP) with a negative serum pregnancy test
Exclusion Criteria
Subject must answer ++NO++ to ++ALL++ exclusion criteria to be enrolled in study:
Concomitant rectovaginal or rectovesical fistula(s)
Patient naïve to prior treatment for perianal fistulas
Presence of perianal collection >2cm in at least 2 dimensions from central reading of MRI at Screening Visit and not considered adequately drained by surgeon at Preparation procedure
Severe rectal and/or anal stenosis and/or severe proctitis
Presence of diverting stomas
Active, uncontrolled infection requiring parenteral antibiotics
Ongoing systemic or rectal steroids for CD in the last 2 weeks prior to Preparation Visit
Major alteration to any of the following laboratory tests:
Serum creatinine levels >1.5 times ULN
Total bilirubin >1.5 times ULN (Unless non-conjugated due to history of Gilbert’s syndrome)
AST/ALT >3.0 times the ULN
Hemoglobin <10.0 g/dL
Platelets <75.0 x 109/L
Albuminedmia <3.0g/dL
Suspected or documented infectious enterocolitis within 2 weeks prior to Screening Visit
Prior invasive malignancy diagnosed within 5 years prior to Screening Visit
Current or recent (within 6 months prior to Screening Visit) history of illness/disease that could increase risk and/or lack of compliance with study procedures
Primary sclerosing cholangitis
Known chronically active hepatopathy of any origin
Congenital or acquired immunodeficiency
Known allergies or hypersensitivity to penicillin or aminoglycosides; DMEM; bovine serum; local anaesthetics or gadolinium (MRI Contrast)
Contraindication to MRI scan
Severe trauma within 6 months prior to Screening Visit
Pregnant or breastfeeding woman
Does not wish or cannot comply with study procedures
Currently receiving, or having received any investigational drug within 3 months prior to Screening Visit
Previously treated with Cx601 or other allogenic stem-cell therapy
Any major surgery of the GI tract within 6 months prior to screening or any minor surgery within 3 months prior to screening
Local perianal surgery other than drainage of fistula within 6 months prior to Screening Visit, or may need surgery in the perianal region for reasons other than fistulas at the time of inclusion
Contraindication to anesthetic procedure
