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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04190862
Recruitment Status : Recruiting
First Posted : December 9, 2019
Last Update Posted : March 3, 2020
Sponsor:
Collaborator:
Angiocrine Bioscience
Information provided by (Responsible Party):
Weill Medical College of Cornell University

Tracking Information
First Submitted Date  ICMJE December 5, 2019
First Posted Date  ICMJE December 9, 2019
Last Update Posted Date March 3, 2020
Actual Study Start Date  ICMJE January 22, 2020
Estimated Primary Completion Date December 31, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 5, 2019)
Safety of dose escalation, as measured by incidence rate of treatment emergent adverse events following the administration of E-CEL UVEC [ Time Frame: 2 weeks ]
The short term safety of escalating dose of E-CEL UVEC cells in subjects with anal fistulas will be assessed by monitoring and recording all adverse events for 2 weeks following the administration of E-CEL UVEC.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 5, 2019)
  • Long-Term Safety of E-CEL UVEC, as measured by the number of adverse events at 6 weeks following the administration of E-CEL UVEC [ Time Frame: 6 weeks ]
    The long term safety of E-CEL UVEC cells in subjects with anal fistulas will be by assessed by monitoring and recording adverse events for 24 weeks following the administration of E-CEL UVEC.
  • Long-Term Safety of E-CEL UVEC, as measured by the number of adverse events for 24 weeks following the administration of E-CEL UVEC [ Time Frame: 24 weeks ]
    The long term safety of E-CEL UVEC cells in subjects with anal fistulas will be by assessed by monitoring and recording adverse events for 24 weeks following the administration of E-CEL UVEC.
  • Efficacy of E-CEL UVEC, as measured the rate of subjects presenting with relapse in treated fistula [ Time Frame: 6, 24 weeks after surgery ]
    Efficacy of E-CEL UVEC, as measured the rate of subjects presenting with relapse in treated fistula at week 6 and 24
  • Efficacy of E-CEL UVEC, as measured by time to complete healing of each side [ Time Frame: 24 weeks after surgery ]
    Efficacy of E-CEL UVEC, as measured by time to complete healing of each side of injection site
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE E-CEL UVEC as an Adjunct Cell Therapy for Treatment of Anal Fistulas
Official Title  ICMJE An Open-Label, Single-Center, Investigator Initiated Phase 1B Trial of E-CEL UVEC as an Adjunct Cell Therapy for Treatment of Anal Fistulas
Brief Summary 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.
Detailed Description

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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Anal Fistula
Intervention  ICMJE Drug: E-CEL UVEC
Injection of E-CEL UVEC
Study Arms  ICMJE Experimental: Cell Therapy Treatment
Patients who present with simple anal fistula and elect to undergo fistulotomy for treatment will be eligible to have E-CEL UVEC injected into the fistula at the time of fistulotomy to aid in healing.
Intervention: Drug: E-CEL UVEC
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: December 5, 2019)
6
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 31, 2022
Estimated Primary Completion Date December 31, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
  • Subjects with HbA1c ≥ 7.0
  • 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
  • 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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Lily Hwang 212-746-5578 euh4001@med.cornell.edu
Contact: Koianka Ivanova-Trencheva 646-962-2342 kivanova@med.cornell.edu
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04190862
Other Study ID Numbers  ICMJE 19-04020122
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Weill Medical College of Cornell University
Study Sponsor  ICMJE Weill Medical College of Cornell University
Collaborators  ICMJE Angiocrine Bioscience
Investigators  ICMJE
Principal Investigator: Jeffrey W Milsom, MD Weill Medical College of Cornell University
PRS Account Weill Medical College of Cornell University
Verification Date March 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP