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

NCT04190862 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: 19-04020122
• Study Type: interventional
• Target: 39
• Locations: 1 country
• Sites: 1

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

Outcome Measures

Primary Outcomes (1)

• 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. (Applicable to all arms)

  2 weeks

Secondary Outcomes (3)

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

  6, 24 weeks after surgery

• E-CEL UVEC Treatment Success, as measured by the combination of cessation and absence of fistula leakage

  24 weeks after surgery

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

  24 weeks after surgery

Study Arms (5)

Cell Therapy Treatment

EXPERIMENTAL

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.

Drug: E-CEL UVEC

Cell Therapy Treatment Part BS1

EXPERIMENTAL

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.

Drug: E-CEL UVEC

Cell Therapy Treatment Part BS2

EXPERIMENTAL

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.

Drug: E-CEL UVEC

Cell Therapy Treatment Part BM1

EXPERIMENTAL

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).

Drug: E-CEL UVEC

Cell Therapy Treatment Part BM2

EXPERIMENTAL

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.

Drug: E-CEL UVEC

Interventions

E-CEL UVEC DRUG

Injection of E-CEL UVEC

Cell Therapy Treatment; Cell Therapy Treatment Part BM1; Cell Therapy Treatment Part BM2; Cell Therapy Treatment Part BS1; Cell Therapy Treatment Part BS2

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Weill Medical College of Cornell University: lead
• Angiocrine Bioscience: collaborator

Study Sites (1)

Weill Cornell Medical College - NewYork-Presbyterian Hospital

New York, New York, 10065, United States

RECRUITING

MeSH Terms

Conditions

Rectal Fistula

Condition Hierarchy (Ancestors)

Intestinal Fistula; Digestive System Fistula; Digestive System Diseases; Intestinal Diseases; Gastrointestinal Diseases; Rectal Diseases; Fistula; Pathological Conditions, Anatomical; Pathological Conditions, Signs and Symptoms

Study Officials

• Jeffrey W Milsom, MD

  Weill Medical College of Cornell University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

KM Muktasid

CONTACT

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

Ananya Sen

CONTACT

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

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 5, 2019
• First Posted: December 9, 2019
• Study Start: January 22, 2020
• Primary Completion (Estimated): September 30, 2026
• Study Completion (Estimated): June 30, 2032
• Last Updated: November 18, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)