Recruiting
Phase 1

E-CEL UVEC

Sponsor:

Weill Medical College of Cornell University

Code:

NCT04190862

Conditions

Anal Fistula

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

E-CEL UVEC

Study Details

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.

Conditions

Anal Fistula

Study ID

NCT04190862

Start date

Jan 22, 2020

Status verified date

Nov, 2025

Completion date

Jun 30, 2032

Anticipated

Primary completion date

Sep 30, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

39 participants

Anticipated

Allocation

Non randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

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.

experimental: Cell Therapy Treatment Part BS1

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.

experimental: Cell Therapy Treatment Part BS2

Subjects in Part B2-S will have the same eligibility criteria and would undergo the same procedure as described in Part B1, except that B2-S subjects will also have suturing and injection of the internal opening.

experimental: Cell Therapy Treatment Part BM1

Subjects in Part BM1 will have the same eligibility criteria and would undergo the same procedure as described in Part B1, except that B2-M subjects in this cohort are eligible for up to 4 treatments (versus 1 in Part BS2).

experimental: Cell Therapy Treatment Part BM2

This cohort comprises of multiple (M) treatments with E-CEL UVEC injections. The difference between Part BM1 and Part BM2, is that the subjects in the latter cohort receive double the dose of E-CEL UVEC cells per treatment. Subjects in Part BM2 will have the same eligibility criteria as Part BS1, BS2 and BM1 cohorts and would undergo the same treatment procedure as described in Parts BS1, BS2 and BM1.

Every treatment consists of the percutaneous injection along both sides of the fistula tract plus injection of the internal opening via direct anoscopic visualization (described in the next paragraph). Dosing is 30 x 106 viable E-CEL UVEC cells into the area of the internal opening and minimum 30 x 106 viable E-CEL UVEC cells per centimeter along the fistula tract (15x106 viable E-CEL UVEC cells, each side per centimeter of the tract).

All subjects in this cohort receive up to 6 treatments over 6 months. The six treatments are administered on visits WK 0, 3. 6, 9, 12 and 24.

Interventions

E-CEL UVEC

Injection of E-CEL UVEC

Primary outcome measure

  • 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 ]

Central Contacts and Locations

Central contacts

KM Muktasid

646-962-2789kmm4010@med.cornell.edu

Ananya Sen

646-962-6445ase4003@med.cornell.edu

Locations

Weill Cornell Medical College - NewYork-Presbyterian Hospital

Recruiting

New York, New York, United States, 10065

Contacts

KM Muktasid

646-962-2789kmm4010@med.cornell.edu

Ananya Sen

646-962-6445ase4003@med.cornell.edu

Principal Investigator:

Jeffrey Milsom, MD

More Information

Sponsor

Weill Medical College of Cornell University

Last update posted

Nov 18, 2025

Last verified

Nov, 2025

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Weill Medical College of Cornell University on 2025-11-18.