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Urothelial CarcinomaPhase 2NCT05868265

A Study of Enfortumab Vedotin in People With Urothelial Carcinoma of the Upper Urinary Tract

A Phase 2 study testing Enfortumab Vedotin and Radical surgery in people with Urothelial Carcinoma, run by Memorial Sloan Kettering Cancer Center at 8 sites in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

The purpose of this study is to find out whether the study drug, enfortumab vedotin, is an effective and safe treatment for people who have urothelial carcinoma of the upper urinary tract. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Urothelial Carcinoma
What is being tested?
Enfortumab Vedotin and Radical surgery
Type of treatment
Surgery or procedure
Could I receive a placebo?
No, everyone receives an active treatment
How many people
9 participants
Where
8 sitesUnited States
Study dates
2 Jun 2023to 2 Jun 2027
Phase
Phase 2
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

  • Histologically confirmed high grade urothelial carcinoma of the upper tract and/or radiographically visible tumor stage T2-T4a N0/x M0 disease with positive selective urinary cytology. Hydronephrosis associated with tumor on imaging or biopsy will be considered invasive by definition. (Variant histology is acceptable if there is a predominant urothelial component. Discussion with PI strongly encouraged.) Retroperitoneal lymph nodes <15 mm in the short axis will be eligible.
  • Patients who are
  • o Ineligible for cisplatin based on any of the following criteria:
  • Estimated or calculated creatinine clearance ≥ 30ml/min but < 60 ml/min
  • Grade 2 or above audiometric hearing loss (per CTCAE v5.0) or
  • Declined cisplatin-based neoadjuvant chemotherapy, as documented in medical chart
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  • Availability of tumor specimen block, cell block or 30 unstained slides from diagnosis. Patients with fewer than 30 slides available may be enrolled after discussion with the Principal Investigator.Additional research biopsy is not required.
  • Karnofsky performance status ≥ 70%.
  • Medically appropriate candidate for radical surgery (nephroureterectomy, nephrectomy, or ureterectomy), as per treating Attending Urologic Oncologist
  • Age ≥ 18 years.
  • Required initial laboratory values:
  • Absolute neutrophil count ≥ 1.5 x 10^9 /L
  • Platelets ≥ 100 x 109 /L
  • Bilirubin ≤1.5 times the upper limit of normal (x ULN)
  • Aspartate transaminase (AST) and alanine transaminase (ALT) ≤ 2.5 x ULN PTT/PT ≤1.5 x ULN or INR < 1.7 x ULN for patients who are not receiving therapeutic anticoagulation. Patients receiving therapeutic anticoagulation should be on a stable dose
  • If patients are HIV (+) they are eligible as long as they have: cd4 >200, undetectable viral load and on HAART therapy.

Who cannot join

  • Presence of metastatic disease.
  • Prior treatment with systemic chemotherapy or radiotherapy for urothelial cancer of the bladder within the last 2 years. (Prior intravesical treatment such as BCG is allowed).
  • Grade 2 or higher peripheral neuropathy.
  • Patients with active keratitis or corneal ulcerations. Patients with superficial punctate keratitis are allowed if the disorder is being adequately treated in the opinion of the investigator.
  • Patients with uncontrolled diabetes. Uncontrolled diabetes is defined as hemoglobin A1c (HbA1c) ≥8% or HbA1c 7% to <8% with associated diabetes symptoms (polyuria or polydipsia) that are not otherwise explained.
  • Unstable angina.
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  • New York Heart Association (NYHA) Grade II or greater congestive heart failure.
  • History of myocardial infarction within 6 months.
  • History of stroke within 6 months.
  • Evidence of bleeding diathesis or coagulopathy. Therapeutic anticoagulation is permitted, but patients must be on a stable dose.
  • Major surgical procedure within 28 days prior to the study. (Transurethral resection of bladder tumor is permitted)
  • Serious, non-healing wound, ulcer, or bone fracture.
  • Patients with active tuberculosis.
  • Other prior malignancy active within the previous 2 years except for local or organ confined early stage cancer that has been definitively treated with curative intent or does not require treatment, does not require ongoing treatment, has no evidence of active disease, and has a negligible risk of recurrence and is therefore unlikely to interfere with the endpoints of the study.
  • Prior treatment with enfortumab vedotin or other MMAE-based antibody-drug conjugates (ADCs).
  • No known active Hepatitis B (e.g., HBsAg reactive) or Hepatitis C (e.g., HCV RNA [qualitative] is detected).
  • Women who are breastfeeding or pregnant as evidenced by a positive pregnancy test within 14 days of first dose.
  • Known severe (≥ Grade 3) hypersensitivity to enfortumab vedotin or to any excipient contained in the drug formulation of enfortumab vedotin (including histidine, trehalose dihydrate, and polysorbate 20).
  • Male subjects who are unwilling to use contraception during the treatment and for at least 31 weeks after the last dose of study treatment (5 half-lives of study drug plus 90 days duration of sperm turnover).
  • Women of childbearing potential (WOCBP) not using a medically acceptable means of contraception throughout the study treatment and for at least 23 weeks following the last dose of study treatment (5 half-lives of study drug plus 30 days duration of ovulatory cycle).
  • °WOCBP are defined as those who have experienced menarche and who have not undergone successful surgical sterilization (hysterectomy, bilateral tubal ligation, or bilateral oophorectomy) or are not postmenopausal. Postmenopausal is defined as:
  • Amenorrhea ≥ 12 consecutive months without another cause, or
  • For women with irregular menstrual periods and on hormone replacement therapy (HRT), a documented serum follicle stimulating hormone (FSH) level > 35 mIU/mL
  • Subjects who are compulsorily detained for treatment of either a psychiatric or physical (e.g., infectious disease) illness.
  • Inability to comply with study and/or follow-up procedures.

Trial locations and contacts

8 sites in 1 country.

8 sites in 1 country

United States8 sites

  • Memorial Sloan Kettering Basking Ridge (Limited Protocol Activities)

    Basking Ridge, New Jersey, 07920

  • Memorial Sloan Kettering Suffolk - Commack (Limited protocol activities)

    Commack, New York, 11725

  • Memorial Sloan Kettering Westchester (Limited Protocol Activities)

    Harrison, New York, 10604

  • Memorial Sloan Kettering Monmouth (Limited Protocol Activities)

    Middletown, New Jersey, 07748

  • Memorial Sloan Kettering Bergen (Limited Protocol Activities)

    Montvale, New Jersey, 07645

  • Memorial Sloan Kettering Cancer Center (All Protocol Activities)

    New York, New York, 10065

  • University of Rochester Medical Center (Data Collection Only)

    Rochester, New York, 14642

  • Memorial Sloan Kettering Nassau (Limited protocol activities)

    Uniondale, New York, 11553

Other trials in United States

About this study

Official title: Neoadjuvant Enfortumab Vedotin in High-Grade Urothelial Carcinoma of the Upper Urinary Tract (Including Ureter and Renal Pelvis)

The purpose of this study is to find out whether the study drug, enfortumab vedotin, is an effective and safe treatment for people who have urothelial carcinoma of the upper urinary tract. Study participants will be people who are not eligible to receive or have chosen not to receive the chemotherapy drug cisplatin for treatment of their cancer. In addition, all participants will be planning on having standard surgery to remove their tumor.

Being tested
Enfortumab Vedotin, Radical surgery
Keywords
Enfortumab Vedotin, Upper Urinary Tract, High Grade, Radical surgery + pelvic lymph node dissection, 21-393
Medical subject headings
Carcinoma, Transitional Cell

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Enfortumab VedotinDrug

    All patients will receive enfortumab vedotin at 1.25 mg/kg on days 1 and 8 of 21 days cycle, for a total of three cycles.

  • Radical surgeryProcedure or surgery

    Radical surgery, such as radical nephroureterectomy, nephrectomy or ureterectomy is to take place within 60 days from the last dose of treatment end of cycle 3 (i.e., cycle 3 day 22).

Study groups in NCT05868265
Study groupRoleWhat they receive
Enfortumab VedotinExperimentalAll patients will receive enfortumab vedotin at 1.25 mg/kg on days 1 and 8 of 21 days cycle, for a total of three cycles, followed by radical nephroureterectomy, ureterectomy, or nephrectomy, depending on the site of the tumor and per the clinical decision of the treating urologist.
Receives: Drug: Enfortumab Vedotin; Procedure: Radical surgery

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Proportion of patients who achieve pathologic complete response

    The absence of residual disease on pathologic evaluation on surgical specimen following neoadjuvant enfortumab vedotin for UTUC. Pathologists will assess surgical specimens systematically using criteria agreed upon for all conventional neoadjuvant treatment based on the AJCC TNM staging system.

    Measured: up to 2 years

Secondary outcomes (1)

  1. The rate of grade 3 - 4 toxicity

    according to CTCAE v5.0.

    Measured: up to 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted22 May 2023
  2. Study start2 Jun 2023
  3. Primary completion2 Jun 2027 (estimated)
  4. Study completion2 Jun 2027 (estimated)

Resources

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 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Model description
This is a single arm phase II trial.
Masking
None (open label)
Enrolment
9 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05868265
Sponsor's protocol ID
21-393
Status last verified
Aug 2026
First submitted
11 May 2023
Last updated
6 Aug 2026
Results posted
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: Memorial Sloan Kettering Cancer Center supports the international committee of medical journal editors (ICMJE) and the ethical obligation of responsible sharing of data from clinical trials. The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made beginning 12 months after publication and for up to 36 months post publication. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals. Requests may be made to: crdatashare@mskcc.org.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Enfortumab Vedotin and Radical surgery in people with Urothelial Carcinoma, run by Memorial Sloan Kettering Cancer Center at 8 sites 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 16 inclusion and 25 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: under way, not looking for participants, as of the sponsor's last update on 6 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 8 sites 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Proportion of patients who achieve pathologic complete response (time frame: up to 2 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05868265.

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 6 Aug 2026.

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