Anal FistulaPhase 1NCT04190862
E-CEL UVEC as an Adjunct Cell Therapy for Treatment of Anal Fistulas
A Phase 1 study testing E-CEL UVEC in people with Anal Fistula, run by Weill Medical College of Cornell University at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Anal Fistula
- What is being tested?
- E-CEL UVEC
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 39 participants
- Where
- 1 siteUnited States
- Study dates
- 22 Jan 2020to 30 Jun 2032
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Weill Cornell Medical College - NewYork-Presbyterian Hospital
New York, New York, 10065
KM Muktasid, 646-962-2789, kmm4010@med.cornell.edu
Ananya Sen, 646-962-6445, ase4003@med.cornell.edu
About this study
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
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.
Read the 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.
- Conditions
- Anal Fistula
- Being tested
- E-CEL UVEC
- Medical subject headings
- Rectal Fistula
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
E-CEL UVECDrug
Injection of E-CEL UVEC
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: 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. Receives: 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). Receives: 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. Receives: Drug: E-CEL UVEC |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: 2 weeks
Secondary outcomes (3)
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
(Applicable to all arms)
Measured: 6, 24 weeks after surgery
E-CEL UVEC Treatment Success, as measured by the combination of cessation and absence of fistula leakage
The success of E-CEL UVEC cells in subjects with anal fistulas will be assessed via digital pressure after completion of all treatments.
(Applicable to all arms)
Measured: 24 weeks after surgery
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 (Applicable to all arms)
Measured: 24 weeks after surgery
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Dec 2019
- Study start22 Jan 2020
- Primary completion30 Sep 2026 (estimated)
- Study completion30 Jun 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Anal FistulaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 39 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Weill Medical College of Cornell University
- Sponsor type
- University, hospital or other
- Collaborators
- Angiocrine Bioscience
- Responsible party
- Sponsor
- Investigators
- Jeffrey W Milsom, MD, Principal investigator, Weill Medical College of Cornell University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04190862
- Sponsor's protocol ID
- 19-04020122
- Status last verified
- Nov 2025
- First submitted
- 5 Dec 2019
- Last updated
- 18 Nov 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04190862
- Acronym / sponsor ID
- 19-04020122
- Conditions
- Anal Fistula
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 39 participants (estimated)
- Lead sponsor
- Weill Medical College of Cornell UniversityUniversity, hospital or other
- Collaborators
- Angiocrine Bioscience
- Study start
- 22 Jan 2020 (actual)
- Primary completion
- 30 Sep 2026 (estimated)
- Study completion
- 30 Jun 2032 (estimated)
- First posted
- 9 Dec 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Cell Therapy Treatment | Experimental | Drug: E-CEL UVECPatients 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. |
| Cell Therapy Treatment Part BS1 | Experimental | Drug: E-CEL UVECAdult 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. |
| Cell Therapy Treatment Part BS2 | Experimental | Drug: E-CEL UVECSubjects 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. |
| Cell Therapy Treatment Part BM1 | Experimental | Drug: E-CEL UVECSubjects 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). |
| Cell Therapy Treatment Part BM2 | Experimental | Drug: E-CEL UVECThis 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- 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
Key exclusion criteria (20 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| E-CEL UVECInjection of E-CEL UVEC | Drug | Cell Therapy Treatment, Cell Therapy Treatment Part BM1, Cell Therapy Treatment Part BM2, Cell Therapy Treatment Part BS1, Cell Therapy Treatment Part BS2 |
Primary outcome
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. (Applicable to all arms)
3 secondary outcomes
Efficacy of E-CEL UVEC, as measured the rate of subjects presenting with relapse in treated fistula
Time frame: 6, 24 weeks after surgery
E-CEL UVEC Treatment Success, as measured by the combination of cessation and absence of fistula leakage
Time frame: 24 weeks after surgery
Efficacy of E-CEL UVEC, as measured by time to complete healing of each side
Time frame: 24 weeks after surgery
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Nov 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Jeffrey W Milsom, MD principal investigator
Weill Medical College of Cornell University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing E-CEL UVEC in people with Anal Fistula, run by Weill Medical College of Cornell University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 18 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact KM Muktasid (646-962-2789, kmm4010@med.cornell.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the 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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Nov 2025.