A Study to Evaluate Enfortumab Vedotin Versus (vs) Chemotherapy in Subjects With Previously Treated Locally Advanced or Metastatic Urothelial Cancer (EV-301)

NCT ID: NCT03474107

Last Updated: 2026-01-09

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

608 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-06-27

Study Completion Date

2025-11-27

Brief Summary

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The purpose of this study was to compare the overall survival (OS) of participants with locally advanced or metastatic urothelial cancer treated with enfortumab vedotin (EV) to the OS of participants treated with chemotherapy.

This study compared progression-free survival on study therapy (PFS1); the overall response rate (ORR) and the disease control rate (DCR) per Response Evaluation Criteria in Solid Tumors (RECIST) V1.1 of participants treated with EV to participants treated with chemotherapy.

In addition, this study evaluated the duration of response (DOR) per RECIST V1.1 of EV and chemotherapy and assessed the safety and tolerability of EV, as well as, the quality of life (QOL) and Patient Reported Outcomes (PRO) parameters.

Detailed Description

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Japan PMDA has approved enfortumab vedotin (Padcev) for the treatment of advanced urothelial cancer. The study will continue as a post marketing study in Japan.

Participants considered an adult according to local regulation at the time of obtaining informed consent participated in the study.

Conditions

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Ureteral Cancer Urothelial Cancer Bladder Cancer

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Arm A: Enfortumab Vedotin 1.25 mg/kg

Participants received 1.25 milligrams per kilogram (mg/kg) of body weight enfortumab vedotin by intravenous infusion over approximately 30 minutes on days 1, 8 and 15 of every 28-day cycle. Participants received study treatment until radiological disease progression as determined per investigator assessment or other discontinuation criteria were met or upon study termination, or study completion, whichever occurred first.

Group Type EXPERIMENTAL

Enfortumab Vedotin

Intervention Type DRUG

Intravenous infusion

Arm B: Chemotherapy

Participants received either 75 milligrams per square meter (mg/m\^2) docetaxel by IV infusion over approximately 1 hour or 320 mg/m\^2 vinflunine by IV infusion over approximately 20 minutes or 175 mg/m\^2 paclitaxel by IV infusion over approximately 1 hour on day 1 of every 21-day cycle. Participants received study treatment until radiological disease progression as determined per investigator assessment or other discontinuation criteria were met or upon study termination, or study completion, whichever occurred first.

Group Type ACTIVE_COMPARATOR

Docetaxel

Intervention Type DRUG

Intravenous infusion

Vinflunine

Intervention Type DRUG

Intravenous infusion

Paclitaxel

Intervention Type DRUG

Intravenous infusion

Cross-over Extension (COE)

Eligible participants from chemotherapy arm who met the criteria for COE will receive 1.25 mg/kg of body weight enfortumab vedotin by intravenous infusion over approximately 30 minutes on days 1, 8 and 15 of every 28-day cycle until discontinuation criteria is met.

Group Type EXPERIMENTAL

Enfortumab Vedotin

Intervention Type DRUG

Intravenous infusion

Interventions

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Enfortumab Vedotin

Intravenous infusion

Intervention Type DRUG

Docetaxel

Intravenous infusion

Intervention Type DRUG

Vinflunine

Intravenous infusion

Intervention Type DRUG

Paclitaxel

Intravenous infusion

Intervention Type DRUG

Other Intervention Names

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ASG-22ME ASG-22CE

Eligibility Criteria

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Inclusion Criteria

* Subject is legally an adult according to local regulation at the time of signing informed consent.
* Subject has histologically or cytologically confirmed urothelial carcinoma (i.e., cancer of the bladder, renal pelvis, ureter, or urethra). Subjects with urothelial carcinoma (transitional cell) with squamous differentiation or mixed cell types are eligible.
* Subject must have experienced radiographic progression or relapse during or after a checkpoint inhibitor (CPI) (anti-programmed cell death protein 1 (PD1) or anti-programmed death-ligand 1 (PD-L1)) for locally advanced or metastatic disease. Subjects who discontinued CPI treatment due to toxicity are eligible provided that the subjects have evidence of disease progression following discontinuation. The CPI need not be the most recent therapy. Subjects for whom the most recent therapy has been a non-CPI based regimen are eligible if the subjects have progressed/relapsed during or after the subjects most recent therapy. Locally advanced disease must not be amenable to resection with curative intent per the treating physician.
* Subject must have received a platinum containing regimen (cisplatin or carboplatin) in the metastatic/locally advanced, neoadjuvant or adjuvant setting. If platinum was administered in the adjuvant/neoadjuvant setting subject must have progressed within 12 months of completion.
* Subject has radiologically documented metastatic or locally advanced disease at baseline.
* An archival tumor tissue sample should be available for submission to central laboratory prior to study treatment. If an archival tumor tissue sample is not available, a fresh tissue sample should be provided. If a fresh tissue sample cannot be provided due to safety concerns, enrollment into the study must be discussed with the medical monitor.
* Subject has ECOG PS of 0 or 1
* The subject has the following baseline laboratory data:

* absolute neutrophil count (ANC) ≥ 1500/mm3
* platelet count ≥ 100 × 10\^9/L
* hemoglobin ≥ 9 g/dL
* serum total bilirubin ≤ 1.5 × upper limit of normal (ULN) or ≤ 3 × ULN for subjects with Gilbert's disease
* creatinine clearance (CrCl) ≥ 30 mL/min as estimated per institutional standards or as measured by 24 hour urine collection (glomerular filtration rate \[GFR\] can also be used instead of CrCl)
* alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 2.5 × ULN or ≤ 3 x ULN for subjects with liver metastases
* Female subject must either:

* Be of nonchildbearing potential: Postmenopausal (defined as at least 1 year without any menses for which there is no other obvious pathological or physiological cause) prior to screening, or documented surgically sterile (e.g., hysterectomy, bilateral salpingectomy, bilateral oophorectomy).
* Or, if of childbearing potential: Agree not to try to become pregnant during the study and for at least 6 months after the final study drug administration, and have a negative urine or serum pregnancy test within 7 days prior to Day 1 (Females with false positive results and documented verification of negative pregnancy status are eligible for participation), and if heterosexually active, agree to consistently use a condom plus 1 form of highly effective birth control per locally accepted standards starting at screening and throughout the study period and for at least 6 months after the final study drug administration.
* Female subject must agree not to breastfeed or donate ova starting at screening and throughout the study period, and for at least 6 months after the final study drug administration.
* A sexually active male subject with female partner(s) who is of childbearing potential is eligible if:

* Agrees to use a male condom starting at screening and continue throughout the study treatment and for at least 6 months after final study drug administration. If the male subject has not had a vasectomy or is not sterile as defined below the subjects female partner(s) is utilizing 1 form of highly effective birth control per locally accepted standards starting at screening and continue throughout study treatment and for at least 6 months after the male subject receives final study drug administration.
* Male subject must not donate sperm starting at screening and throughout the study period, and for at least 6 months after the final study drug administration.
* Male subject with a pregnant or breastfeeding partner(s) must agree to abstinence or use a condom for the duration of the pregnancy or time partner is breastfeeding throughout the study period and for at least 6 months after the final study drug administration.
* Subject agrees not to participate in another interventional study while on treatment in present study.


* Institutional review board (IRB)/ independent ethics committee (IEC) approved written COE informed consent and privacy language as per national regulations (e.g., health insurance portability and accountability act \[HIPAA\] Authorization for US sites) must be obtained from the subject prior to any study-related procedures (including withdrawal of prohibited medication, if applicable).
* Subject was randomized to Arm B and is either currently on study treatment or has discontinued study treatment due to intolerance, AE or progression of disease and has not started a new systemic anticancer treatment.

Exclusion Criteria

* Subject has preexisting sensory or motor neuropathy Grade ≥ 2.
* Subject has active central nervous system (CNS) metastases. Subjects with treated CNS metastases are permitted on study if all the following are true:

* CNS metastases have been clinically stable for at least 6 weeks prior to screening
* If requiring steroid treatment for CNS metastases, the subject is on a stable dose ≤ 20 mg/day of prednisone or equivalent for at least 2 weeks
* Baseline scans show no evidence of new or enlarged brain metastasis
* Subject does not have leptomeningeal disease
* Subject has ongoing clinically significant toxicity (Grade 2 or higher with the exception of alopecia) associated with prior treatment (including systemic therapy, radiotherapy or surgery). Subject with ≤ Grade 2 immunotherapy-related hypothyroidism or panhypopituitarism may be enrolled when well-maintained/controlled on a stable dose of hormone replacement therapy (if indicated). Subjects with ongoing ≥ Grade 3 immunotherapy-related hypothyroidism or panhypopituitarism are excluded. Subjects with ongoing immunotherapy related colitis, uveitis, or pneumonitis or subjects with other immunotherapy related AEs requiring high doses of steroids (\> 20 mg/day of prednisone or equivalent) are excluded.
* Subject has prior treatment with EV or other monomethyl auristatin E (MMAE)-based Antibody drug conjugates (ADCs).
* Subject has received prior chemotherapy for urothelial cancer with all available study therapies in the control arm (i.e., both prior paclitaxel and docetaxel in regions where vinflunine is not an approved therapy, or prior paclitaxel, docetaxel and vinflunine in regions where vinflunine is an approved therapy).
* Subject has received more than 1 prior chemotherapy regimen for locally advanced or metastatic urothelial cancer, including chemotherapy for adjuvant or neo-adjuvant disease if recurrence occurred within 12 months of completing therapy. The substitution of carboplatin for cisplatin does not constitute a new regimen provided no new chemotherapeutic agents were added to the regimen.
* Subject has history of another malignancy within 3 years before the first dose of study drug, or any evidence of residual disease from a previously diagnosed malignancy. Subjects with nonmelanoma skin cancer, localized prostate cancer treated with curative intent with no evidence of progression, low-risk or very low-risk (per standard guidelines) localized prostate cancer under active surveillance/watchful waiting without intent to treat, or carcinoma in situ of any type (if complete resection was performed) are allowed.
* Subject is currently receiving systemic antimicrobial treatment for viral, bacterial, or fungal infection at the time of first dose of EV. Routine antimicrobial prophylaxis is permitted.
* Subject has known active Hepatitis B (e.g., hepatitis B surface antigen (HBsAg) reactive) or active hepatitis C (e.g., hepatitis C virus (HCV) Ribonucleic Acid (RNA) \[qualitative\] is detected).
* Subject has known history of human immunodeficiency virus (HIV) infection (HIV 1 or 2).
* Subject has documented history of a cerebral vascular event (stroke or transient ischemic attack), unstable angina, myocardial infarction, or cardiac symptoms (including congestive heart failure) consistent with New York Heart Association Class III-IV within 6 months prior to the first dose of study drug.
* Subject has radiotherapy or major surgery within 4 weeks prior to first dose of study drug.
* Subject has had chemotherapy, biologics, investigational agents, and/or antitumor treatment with immunotherapy that is not completed 2 weeks prior to first dose of study drug.
* Subject has known hypersensitivity to EV or to any excipient contained in the drug formulation of EV; OR subject has known hypersensitivity to biopharmaceuticals produced in Chinese hamster ovary (CHO) cells.
* Subject has known hypersensitivity to the following: docetaxel or to any of the other excipients listed in product label, including polysorbate 80, paclitaxel or to any of the other excipients listed in product label, such as macrogolglycerol ricinoleate 35 (Ph.Eur.); and vinflunine or to any of the other excipients listed in product label such as other vinca alkaloids (vinblastine,vincristine, vindesine, vinorelbine).
* Subject has known active keratitis or corneal ulcerations.
* Subject has other underlying medical condition that would impair the ability of the subject to receive or tolerate the planned treatment and follow-up.
* History of uncontrolled diabetes mellitus within 3 months of the first dose of study drug. Uncontrolled diabetes is defined as hemoglobin A1C (HbA1c) ≥ 8% or HbA1c between 7 and \< 8% with associated diabetes symptoms (polyuria or polydipsia) that are not otherwise explained.


* Subject has been diagnosed with a new malignancy while on Arm B in the EV-301 study. Subjects with nonmelanoma skin cancer, localized prostate cancer treated with curative intent with no evidence of progression, low-risk or very low-risk (per standard guidelines) localized prostate cancer under active surveillance/watchful waiting without intent to treat, or carcinoma in situ of any type (if complete resection was performed) are allowed.
* Subject has already started commercial EV or arrangements have been made for subject to start commercial EV which is reimbursed in their country. Additionally, if EV is commercially available with reimbursement in the potential subject's country, the subject can consider transitioning to the commercial product unless otherwise discussed with sponsor.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Seagen Inc.

INDUSTRY

Sponsor Role collaborator

Astellas Pharma Global Development, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Director

Role: STUDY_DIRECTOR

Astellas Pharma Global Development, Inc.

Locations

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UCI Chao Family Comprehensive Cancer Center

Orange, California, United States

Site Status

University of California

Sacramento, California, United States

Site Status

Innovative Clinical Research

Whittier, California, United States

Site Status

University of Colorado

Denver, Colorado, United States

Site Status

Smilow Cancer Hospital at Yale-New Haven

New Haven, Connecticut, United States

Site Status

Sylvester Comprehensive Cancer Center

Miami, Florida, United States

Site Status

Florida Hospital

Orlando, Florida, United States

Site Status

Rush University Medical Center

Chicago, Illinois, United States

Site Status

Norton Cancer Institute

Louisville, Kentucky, United States

Site Status

Dana-Farber Cancer Institute

Boston, Massachusetts, United States

Site Status

Nebraska Cancer Specialists

Omaha, Nebraska, United States

Site Status

Roswell Park Cancer Institute

Buffalo, New York, United States

Site Status

Long Island Jewish Medical Center

Lake Success, New York, United States

Site Status

Sidney Kimmel Center for Prostate and Urologic Cancers

New York, New York, United States

Site Status

Montefiore Medical Center

The Bronx, New York, United States

Site Status

White Plains Hospital Center for Cancer Care - Oncology Site

White Plains, New York, United States

Site Status

Toledo Clinic Cancer Center

Toledo, Ohio, United States

Site Status

Providence Portland Med Center

Portland, Oregon, United States

Site Status

University of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status

Fox Chase Cancer Center

Philadelphia, Pennsylvania, United States

Site Status

Lifespan Rhode Island Hospital

Providence, Rhode Island, United States

Site Status

Saint Francis Hospital

Greenville, South Carolina, United States

Site Status

HOPE Cancer Center of East Texas

Tyler, Texas, United States

Site Status

Benaroya Research Institute at Virginia Mason

Seattle, Washington, United States

Site Status

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Site Status

Site AR54001

Buenos Aires, , Argentina

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Site AU61006

Adelaide, , Australia

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Site AU61001

Miranda, , Australia

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Site AU61004

St Leonards, , Australia

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Site AU61002

Sydney, , Australia

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Site AT43005

Linz, , Austria

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Site AT43001

Salzburg, , Austria

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Site AT43004

Vienna, , Austria

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Site BE32011

Aalst, , Belgium

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Site BE32007

Brussels, , Belgium

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Site BE32013

Brussels, , Belgium

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Site BE32010

Charleroi, , Belgium

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Site BE32001

Ghent, , Belgium

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Site BE32008

Ghent, , Belgium

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Site BE32005

Hasselt, , Belgium

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Site BE32003

Leuven, , Belgium

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Site BE32009

Liège, , Belgium

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Site CA15015

Calgary, , Canada

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Site CA15012

Edmonton, , Canada

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Site CA15014

London, , Canada

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Site CA15002

Montreal, , Canada

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Site CA15007

Montreal, , Canada

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Site CA15011

Oshawa, , Canada

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Site CA15004

Québec, , Canada

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Site CA15008

Saskatoon, , Canada

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Site CA15001

Sherbrooke, , Canada

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Site CA15005

Toronto, , Canada

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Site CA15013

Vancouver, , Canada

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Site DK45003

Aalborg, , Denmark

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Site DK45004

Copenhagen, , Denmark

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Site DK45001

Herlev, , Denmark

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Site FR33021

Besançon, , France

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Site FR33009

Bordeaux, , France

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Site FR33018

Bordeaux, , France

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Site FR33001

Brest, , France

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Site FR33016

Caen, , France

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Site FR33015

Lyon, , France

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Site FR33014

Marseille, , France

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Site FR33003

Nice, , France

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Site FR33022

Paris, , France

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Site FR33005

Pierre-Bénite, , France

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Site FR33004

Saint-Mandé, , France

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Site FR33002

Strasbourg, , France

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Site FR33019

Toulouse, , France

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Site FR33006

Villejuif, , France

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Site DE49011

Essen, , Germany

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Site DE49008

Heidelberg, , Germany

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Site DE49010

Münster, , Germany

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Site DE49003

Tübingen, , Germany

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Site DE49009

Würzburg, , Germany

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Site IT39008

Arezzo, , Italy

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Site IT39019

Cremona, , Italy

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Site IT39010

Milan, , Italy

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Site IT39025

Modena, , Italy

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Site IT39013

Pisa, , Italy

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Site IT39014

Reggio Emilia, , Italy

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Site IT39004

Terni, , Italy

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Site JP81010

Hirosaki, Aomori, Japan

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Site JP81014

Kashiwa, Chiba, Japan

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Site JP81007

Sapporo, Hokkaido, Japan

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Site JP81026

Sapporo, Hokkaido, Japan

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Site JP81020

Tsukuba, Ibaraki, Japan

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Site JP81018

Morioka, Iwate, Japan

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Site JP81009

Kita-gun, Kagawa-ken, Japan

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Site JP81002

Yokohama, Kanagawa, Japan

Site Status

Site JP81005

Sendai, Miyagi, Japan

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Site JP81016

Sayama, Osaka, Japan

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Site JP81024

Takatsuki, Osaka, Japan

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Site JP81008

Bunkyo-ku, Tokyo, Japan

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Site JP81012

Koto-ku, Tokyo, Japan

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Site JP81013

Shinjuku-ku, Tokyo, Japan

Site Status

Site JP81011

Ube, Yamaguchi, Japan

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Site JP81015

Chiba, , Japan

Site Status

Site JP81019

Fukuoka, , Japan

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Site JP81023

Fukuoka, , Japan

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Site JP81004

Hiroshima, , Japan

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Site JP81001

Kyoto, , Japan

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Site JP81017

Niigata, , Japan

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Site JP81003

Okayama, , Japan

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Site JP81022

Osaka, , Japan

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Site JP81021

Tokushima, , Japan

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Site JP81006

Toyama, , Japan

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Site NL31002

Amsterdam, , Netherlands

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Site NL31003

Amsterdam, , Netherlands

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Site NL31009

Nijmegen, , Netherlands

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Site NL31001

Tilburg, , Netherlands

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Site PT35102

Lisbon, , Portugal

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Site PT35105

Lisbon, , Portugal

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Site PT35106

Porto, , Portugal

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Site RU70002

Ivanovo, , Russia

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Site RU70009

Obninsk, , Russia

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Site RU70005

Omsk, , Russia

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Site RU70015

Vologda, , Russia

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Site KR82006

Daejeon, , South Korea

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Site KR82007

Goyang-si, , South Korea

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Site KR82012

Hwasun-gun, , South Korea

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Site KR82002

Incheon, , South Korea

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Site KR82001

Seongnam-si, , South Korea

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Site KR82003

Seoul, , South Korea

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Site KR82004

Seoul, , South Korea

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Site KR82008

Seoul, , South Korea

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Site KR82009

Seoul, , South Korea

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Site KR82010

Seoul, , South Korea

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Site KR82005

Shin, , South Korea

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Site ES34010

Badajoz, , Spain

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Site ES34002

Badalona, , Spain

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Site ES34001

Barcelona, , Spain

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Site ES34012

Barcelona, , Spain

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Site ES34023

Barcelona, , Spain

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Site ES34014

Córdoba, , Spain

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Site ES34003

Madrid, , Spain

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Site ES34013

Madrid, , Spain

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Site ES34015

Madrid, , Spain

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Site ES34017

Madrid, , Spain

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Site ES34011

Manresa, , Spain

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Site ES34019

Pamplona, , Spain

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Site ES34005

Seville, , Spain

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Site ES34007

Valencia, , Spain

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Site ES34008

Valencia, , Spain

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Site CH41002

Bern, , Switzerland

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Site CH41001

Chur, , Switzerland

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Site TW88602

Kaohsiung City, , Taiwan

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Site TW88605

Kaohsiung City, , Taiwan

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Site TW88606

Taichung, , Taiwan

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Site TW88601

Tainan, , Taiwan

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Site TW88604

Taipei, , Taiwan

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Site TW88607

Taoyuan District, , Taiwan

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Site GB44005

London, , United Kingdom

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Site GB44006

London, , United Kingdom

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Site GB44004

Metropolitan Borough of Wirral, , United Kingdom

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Site GB44002

Sheffield, , United Kingdom

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Site GB44011

Southampton, , United Kingdom

Site Status

Site GB44013

Sutton, , United Kingdom

Site Status

Countries

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United States Argentina Australia Austria Belgium Canada Denmark France Germany Italy Japan Netherlands Portugal Russia South Korea Spain Switzerland Taiwan United Kingdom

References

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Rosenberg JE, Mamtani R, Sonpavde GP, Loriot Y, Duran I, Lee JL, Matsubara N, Vulsteke C, Castellano D, Sridhar SS, Pappot H, Gurney H, Bedke J, van der Heijden MS, Galli L, Keam B, Masumori N, Meran J, O'Donnell PH, Park SH, Grande E, Sengelov L, Uemura H, Skaltsa K, Campbell M, Matsangou M, Wu C, Hepp Z, McKay C, Powles T, Petrylak DP. Health-related Quality of Life in Patients with Previously Treated Advanced Urothelial Carcinoma from EV-301: A Phase 3 Trial of Enfortumab Vedotin Versus Chemotherapy. Eur Urol. 2024 Jun;85(6):574-585. doi: 10.1016/j.eururo.2024.01.007. Epub 2024 Feb 28.

Reference Type DERIVED
PMID: 38418343 (View on PubMed)

Powles T, Rosenberg JE, Sonpavde GP, Loriot Y, Duran I, Lee JL, Matsubara N, Vulsteke C, Castellano D, Wu C, Campbell M, Matsangou M, Petrylak DP. Enfortumab Vedotin in Previously Treated Advanced Urothelial Carcinoma. N Engl J Med. 2021 Mar 25;384(12):1125-1135. doi: 10.1056/NEJMoa2035807. Epub 2021 Feb 12.

Reference Type DERIVED
PMID: 33577729 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2017-003344-21

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

jRCT2080224027

Identifier Type: REGISTRY

Identifier Source: secondary_id

2024-517571-20-00

Identifier Type: REGISTRY

Identifier Source: secondary_id

7465-CL-0301

Identifier Type: -

Identifier Source: org_study_id

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