E-CEL UVEC as an Adjunct Cell Therapy for Treatment of Anal Fistulas

An Open-Label, Single-Center, Investigator Initiated Phase 1B Trial of E-CEL UVEC as an Adjunct Cell Therapy for Treatment of Anal Fistulas

The purpose of this study is to determine if endothelial cells derived from human umbilical vein are safe for use in conjunction with fistulotomy for the treatment of simple anal fistulas. Endothelial cells are a special kind of cell in the body that line the inside surface of blood vessels. The goal of the study is to evaluate the preliminary safety of human umbilical vein cells in anal fistula healing.

The purpose of this study is to test the safety of E-CEL UVEC® cells and see what effects (good and bad) it has on your anal fistula. It is hoped, that the E-CEL UVEC® cells may help to improve healing of your fistula after surgery.

E-CEL UVEC® cells are genetically engineered human endothelial cells that are taken from the umbilical cords of newborn babies. The endothelial cells are cells that line the inside of blood vessels including the umbilical cord. Human umbilical endothelial cells are collected from the umbilical cord of a healthy newborn baby. The cells are obtained under strict United States (U.S.) Food and Drug Administration (FDA) regulations. The endothelial cells are engineered in the laboratory, meaning an extra gene is added. A gene is taken from a virus (just a single gene, not the entire virus) and inserted into the endothelial cells.This causes the endothelial cells to be more stable and improves their growth capabilities. In animal studies, the endothelial cells were cleared from the body within a month. No negative side effects related to the endothelial cells were seen in animal studies. A higher than normal healing response was seen in animal studies.

This research study is being done because, in animal studies, E-CEL UVEC® cells have been shown to speed up healing in various tissues and organs. This study will test if it is safe to use E-CEL UVEC® cells and if they help to improve healing of your fistula after surgery.

Inclusion Criteria:

• Subject ages 18 or older
• Subject willing and able to provide informed consent
• Subject is medically eligible and have agreed to undergo a fistulotomy
• Subject with simple perianal fistula with 2 or fewer fistula tracts
• Maximum tract length of 3 inches
• Subject without history of Crohn's disease/ Ulcerative Colitis
• For female subjects of childbearing potential:
• A negative serum or urine pregnancy test at screening is required prior to enrollment
• Subject must be willing to use a highly effective method of contraception from the start of the screening period throughout the study period
• For males who can father a child and are having intercourse with females of childbearing potential who are not using adequate contraception:
• Subject must be willing to use a recommended method of contraception and refrain from sperm donation from the start of the conditioning therapy for at least 1 year after completion and discussion with a treating physician

Exclusion Criteria:

• Concomitant rectovaginal fistulas
• Subjects with an abscess
• Presence of active infections findings (e.g.; redness, swelling, tenderness or fever)
• Presence of rectal and/or anal stenosis
• The presence of setons unless removed prior to the treatment
• Subjects with ongoing steroid treatment or treated with steroids in the last 4 weeks
• Renal impairment defined by creatinine clearance below 90 mL/min calculated using Cockcroft-Gault formula or by serum creatinine ≥ 1.5 x upper limit of normality (ULN)
• Hepatic impairment defined by both of the following laboratory ranges:
• Total bilirubin ≥ 1.5 x ULN unless benign congenital hyperbilirubinemia
• Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≥ 2.5 x ULN
• Known history of abuse of alcohol or other addictive substances in the 6 months prior to enrollment
• Active malignant tumor within 5 years
• Current recent history of abnormal, severe, progressive, uncontrolled hepatic, hematological, gastrointestinal, endocrine, pulmonary, cardiac, neurological, psychiatric, or cerebral diseases
• Congenital or acquired immunodeficiencies including human immunodeficiency virus (HIV) infection or acquired immunodeficiency syndrome (AIDS)
• Major surgery or severe trauma within the previous 6 months
• Subjects who are candidates for solid organ transplantation or who may have a high likelihood of needing a solid organ transplant (ex. progressive heart failure)
• Females who are who are pregnant or breastfeeding or planning to become pregnant or breastfeed during treatment
• Subjects who have known hypersensitivity or documented allergy to DMSO
• Subjects who do not wish to or cannot comply with study procedures
• Subjects currently receiving, or having received any investigational drug within 3 months prior to E-CEL UVEC cell therapy