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

Primary Purpose

Anal Fistula

Status

Recruiting

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

E-CEL UVEC

About this trial

This is an interventional treatment trial for Anal Fistula

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Weill Cornell Medical College - NewYork-Presbyterian HospitalRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Arm Label

Cell Therapy Treatment

Cell Therapy Treatment Part B

Arm Description

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.

Adult subjects with simple perianal fistula who meet all eligibility criteria to participate in Part B of the study will be treated in the outpatient setting with curettage and E-CEL UVEC cells without fistulotomy. E-CEL UVEC cells will be injected along the two sides (180 degrees apart from each other) of the whole length of the curetted anal fistula tract.

Outcomes

Primary Outcome Measures

Safety of dose escalation, as measured by incidence rate of treatment emergent adverse events following the administration of E-CEL UVEC

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.

Secondary Outcome Measures

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

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

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

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

Efficacy of E-CEL UVEC, as measured by time to complete healing of each side of injection site

Full Information

NCT ID

NCT04190862

First Posted

December 5, 2019

Last Updated

June 6, 2023

Sponsor

Weill Medical College of Cornell University

Collaborators

Angiocrine Bioscience

1. Study Identification

Unique Protocol Identification Number

NCT04190862

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

June 2023

Overall Recruitment Status

Recruiting

Study Start Date

January 22, 2020 (Actual)

Primary Completion Date

December 31, 2024 (Anticipated)

Study Completion Date

December 31, 2031 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Weill Medical College of Cornell University

Collaborators

Angiocrine Bioscience

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Anal Fistula

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Sequential Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

12 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Cell Therapy Treatment

Arm Type

Experimental

Arm Description

Arm Title

Cell Therapy Treatment Part B

Arm Type

Experimental

Arm Description

Intervention Type

Drug

Intervention Name(s)

E-CEL UVEC

Intervention Description

Injection of E-CEL UVEC

Primary Outcome Measure Information:

Title

Safety of dose escalation, as measured by incidence rate of treatment emergent adverse events following the administration of E-CEL UVEC

Description

Time Frame

2 weeks

Secondary Outcome Measure Information:

Title

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

Description

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.

Time Frame

6 weeks

Title

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

Description

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.

Time Frame

24 weeks

Title

Efficacy of E-CEL UVEC, as measured the rate of subjects presenting with relapse in treated fistula

Description

Efficacy of E-CEL UVEC, as measured the rate of subjects presenting with relapse in treated fistula at week 6 and 24

Time Frame

6, 24 weeks after surgery

Title

Efficacy of E-CEL UVEC, as measured by time to complete healing of each side

Description

Efficacy of E-CEL UVEC, as measured by time to complete healing of each side of injection site

Time Frame

24 weeks after surgery

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

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

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Rohit Rasane

Phone

646-962-2789

rkr4004@med.cornell.edu

First Name & Middle Initial & Last Name or Official Title & Degree

Julianna Brouwer, MPH

Phone

6469622394

jub2024@med.cornell.edu

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Jeffrey W Milsom, MD

Organizational Affiliation

Weill Medical College of Cornell University

Official's Role

Principal Investigator

Facility Information:

Facility Name

Weill Cornell Medical College - NewYork-Presbyterian Hospital

City

New York

State/Province

New York

ZIP/Postal Code

10065

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Rohit Rasane

Phone

646-962-2789

rkr4004@med.cornell.edu

First Name & Middle Initial & Last Name & Degree

Julianna Brouwer

Phone

646-962-2394

jub2024@med.cornell.edu

First Name & Middle Initial & Last Name & Degree

Heather Yeo, MD

First Name & Middle Initial & Last Name & Degree

Kelly Garrett, MD

First Name & Middle Initial & Last Name & Degree

Lea Lowenfeld, MD

First Name & Middle Initial & Last Name & Degree

Alessio Pigazzi, MD, PhD

First Name & Middle Initial & Last Name & Degree

Mehraneh Jafari, MD

First Name & Middle Initial & Last Name & Degree

Jeffrey Milsom, MD

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

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

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