LEGEND Study: EG-70 in NMIBC Patients BCG-Unresponsive and High-Risk NMIBC Incompletely Treated With BCG or BCG-Naïve
NCT ID: NCT04752722
Last Updated: 2025-11-12
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1/PHASE2
350 participants
INTERVENTIONAL
2021-04-22
2028-11-30
Brief Summary
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This study study consists of two phases; a Phase 1 dose-escalation to establish safety and recommended the phase 2 dose, followed by a Phase 2 study to establish how effective the treatment is.
The Study will include patients with NMIBC with Cis for whom BCG therapy is unresponsive and patients with NMIBC with Cis who are BCG-naïve or inadequately treated.
Detailed Description
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Eligible BCG-unresponsive NMIBC patients will be enrolled in Phase 1, and Cohort 1 of Phase 2.
Eligible high-risk NMIBC patients will be enrolled starting in Phase 2 in separate single are cohorts include: BCG-naïve patients or BCG-exposed (incompletely treated) patients with Carcinoma in situ (CIS), and BCG-unresponsive HG Ta/T1 papillary disease without CIS.
Patients will be treated for up to four 12-week cycles of study drug instillation doses and assessments with follow up assessments.
Patients with complete response following the four 12-week cycles will enter up to 4 maintenance treatment cycles, and those remaining in complete response will enter another 4 maintenance treatment cycles or follow up assessments.
Conditions
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Keywords
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Study Design
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NON_RANDOMIZED
SEQUENTIAL
TREATMENT
NONE
Study Groups
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Phase 1
Dose escalation phase
EG-70 (phase 1)
Patients will receive up to four cycles of EG-70 administered as a bladder instillation of a 50 mL volume of study drug via catheter with a targeted retention time of 60 minutes.One cycle lasts approximately 12 weeks and consists of either a 2-dose (Day 1 and Day 8) or 4-dose (Day 1, Day 8, Day 29 and Day 36) regimen.
Phase 2
Cohort 1: Recommended Phase 2 dose (RP2D) with eligible BCG-unresponsive NMIBC patients with CIS, up to 4 cycles of treatment with EG-70
Cohorts 2A, 2B and 3: RP2D with eligible high-risk NMIBC patients with CIS who are BCG-naïve, BCG-exposed (incompletely treated with BCG) or BCG-unresponsive HG Ta/T1 papillary disease without CIS
EG-70 (phase 2)
Patients will receive during Treatment Period, up to 4 cycles of EG-70 at the RP2D defined in Phase 1 administered as a bladder instillation of a 50 mL volume of study drug via catheter with a targeted retention time of 60 minutes. One cycle lasts approximately 12 weeks. For Maintenance treatment 2 doses of EG-70 will be administered as a bladder instillation per 12-week cycle.
Interventions
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EG-70 (phase 1)
Patients will receive up to four cycles of EG-70 administered as a bladder instillation of a 50 mL volume of study drug via catheter with a targeted retention time of 60 minutes.One cycle lasts approximately 12 weeks and consists of either a 2-dose (Day 1 and Day 8) or 4-dose (Day 1, Day 8, Day 29 and Day 36) regimen.
EG-70 (phase 2)
Patients will receive during Treatment Period, up to 4 cycles of EG-70 at the RP2D defined in Phase 1 administered as a bladder instillation of a 50 mL volume of study drug via catheter with a targeted retention time of 60 minutes. One cycle lasts approximately 12 weeks. For Maintenance treatment 2 doses of EG-70 will be administered as a bladder instillation per 12-week cycle.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
1. BCG-unresponsive NMIBC with carcinoma in situ (CIS) with or without coexisting papillary Ta/T1 tumors who are ineligible for or have elected not to undergo cystectomy, and have experienced CIS disease within 12 months of treatment where: adequate BCG regimen consists of at least 2 courses of BCG where the first course (induction) must have included at least 5 or 6 doses and the second course may have included a re-induction (at least 2 treatments) or maintenance (at least 2 doses), and Cis must be documented or indicated by pathology
Phase 2 Only:
2. BCG-Naïve or BCG-incompletely treated Patients with CIS or BCG-unresponsive, HG Ta/T1 papillary disease without CIS:
-NMIBC with current Cis of the bladder, with or without coexisting papillary Ta/T1 NMIBC tumor(s), who are ineligible for or have elected not to undergo cystectomy, where: either: cohort 2a) no treatment with BCG but may have previously been treated with at least 1 dose of intravesical chemotherapy following transurethral resection of bladder tumor (TURBT) and Cis must be documented or cohort 2b) indicated by pathology incomplete BCG treatment (at least 1 dose and less than the 5+2 doses required for adequate dosing per Cohort 1) or cohort 3) patients who are BCG-unresponsive following adequate treatment, with HG Ta/T1 papillary disease without CIS.
All Patients:
3. Patients who have previously been treated with a checkpoint inhibitor and failed treatment are eligible for inclusion 30 days post-treatment (Phase 1) or 3 months post-treatment (Phase 2).
4. Male or non-pregnant, non-lactating female, 18 years or older.
5. Women of childbearing potential must have a negative pregnancy test at Screening.
6. Female patients of childbearing potential must be willing to consent to using highly effective birth control methods; Male patients are required to utilize a condom for the duration of the study treatment through 3 months post-dose.
7. In Phase 2, for patients with T1 lesions may be eligible after repeat TURBT if pathology shows non-invasive (Ta or less) or no disease.
8. Performance Status: Eastern Cooperative Oncology Group 0, 1, and 2.
9. Hematologic inclusion: a. Absolute neutrophil count \>1,500/mm3. b. Hemoglobin \>9.0 g/dL. c. Platelet count \>100,000/mm3.
10. Hepatic inclusion: a. Total bilirubin must be ≤1.5 x the upper limit of normal (ULN). b. Aspartate aminotransferase (AST), alanine aminotransferase (ALT) and alkaline phosphatase ≤2.5 x ULN.
11. Adequate renal function with creatinine clearance \>30 mL/min
12. Prothrombin time and partial thromboplastin time ≤1.25 x ULN or within the therapeutic range if on anticoagulation therapy.
13. Must have satisfactory bladder function with ability to retain study drug for 60 minutes.
Exclusion Criteria
2. Concurrent treatment with any chemotherapeutic agent.
3. History of partial cystectomy.
4. Treatment with last therapeutic agent (including intravesical chemotherapy post-TURBT) within 30 days of Screening (prior to the screening biopsy).
5. Patients who have received systemic immunosuppressive medication including high-dose corticosteroids.
6. History of severe asthma or other respiratory diseases.
7. History of unresolved vesicoureteral reflux or an indwelling urinary stent.
8. History of unresolved hydronephrosis due to ureteral obstruction.
9. Participation in any other research protocol involving administration of an investigational agent within 30 Days prior to screening or any prior treatment of NMIBC with any investigational gene or immunotherapy agent.
10. History of external beam radiation to the pelvis or prostate brachytherapy within the last 12 months.
11. History of interstitial lung disease and/or pneumonitis in patients who have previously received a PD-1 or PD-L1 inhibitor therapy.
12. Evidence of metastatic disease.
13. History of difficult catheterization that in the opinion of the Investigator will prevent administration of EG-70.
14. Active interstitial cystitis on cystoscopy or biopsy.
15. Active, uncontrolled bacterial, viral, or fungal infection(s) requiring systemic therapy.
16. Known human immunodeficiency virus, Hepatitis B, or Hepatitis C infection.
17. Significant cardiovascular risk (e.g., coronary stenting within 8 weeks, myocardial infarction within 6 months).
18. Hypersensitivity to any of the excipients of the study drug.
18 Years
ALL
No
Sponsors
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enGene, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Christine Tosone, Ms, RAC
Role: STUDY_DIRECTOR
enGene, Inc.
Locations
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The University of Alabama at Birmingham Clinical Research Unit (CRU)
Birmingham, Alabama, United States
Mayo Clinic
Scottsdale, Arizona, United States
Urological Associates of South Arizona
Tucson, Arizona, United States
Arkansas Urology
Little Rock, Arkansas, United States
University of California - Irvine Medical Center
Irvine, California, United States
UC San Diego Moores Cancer Center
La Jolla, California, United States
Urology Group of Southern California / American Institute of Research
Los Angeles, California, United States
USC/Norris Comprehensive Cancer Center
Los Angeles, California, United States
Tower Urology
Los Angeles, California, United States
Genesis Research
San Diego, California, United States
Colorado Clinical Research
Lakewood, Colorado, United States
The George Washington Medical Faculty Associates
Washington D.C., District of Columbia, United States
University of Florida
Jacksonville, Florida, United States
Sylvester Comprehensive Cancer Center / University of Miami Hospital and Clinics
Miami, Florida, United States
Emory University
Atlanta, Georgia, United States
Rush University Medical Center
Chicago, Illinois, United States
Urology of Indiana
Greenwood, Indiana, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
John Hopkins Hospital
Baltimore, Maryland, United States
Chesapeake Urology Research Associates
Hanover, Maryland, United States
Brigham and Women's Hospital
Boston, Massachusetts, United States
Karmanos Cancer Institute
Detroit, Michigan, United States
Henry Ford Health System
Detroit, Michigan, United States
Corewell Health Medical Group and Spectrum Health Hospitals
Grand Rapids, Michigan, United States
University of Minnesota
Minneapolis, Minnesota, United States
Mayo Clinic
Rochester, Minnesota, United States
Rutgers Cancer Institute of New Jersey
New Brunswick, New Jersey, United States
New Jersey Urology, LLC
Voorhees Township, New Jersey, United States
Albany Medical College
Albany, New York, United States
Mount Sinai Medical Center
New Haven, New York, United States
Laura & Isaac Perlmutter Cancer Center at NYU Langone Health
New York, New York, United States
Associated Medical Professionals of NY,
Syracuse, New York, United States
UNC Chapel Hill Hospital
Chapel Hill, North Carolina, United States
Associated Urologists of North Carolina
Raleigh, North Carolina, United States
University of Cincinnati Medical Center
Cincinnati, Ohio, United States
Central Ohio Urology Group
Gahanna, Ohio, United States
Clinical Research Solutions - Helios Clinical Research
Middleburg Heights, Ohio, United States
The University of Toledo Medical Center
Toledo, Ohio, United States
Oregon Health & Science University (OHSU)
Portland, Oregon, United States
Carolina Urologic Research Center, LLC
Myrtle Beach, South Carolina, United States
Urology Associates, P.C.
Nashville, Tennessee, United States
Vanderbilt Univerity Medical Center
Nashville, Tennessee, United States
Urology Austin
Austin, Texas, United States
UT Southwestern Medical Center
Dallas, Texas, United States
Houston Metro Urology
Houston, Texas, United States
Houston Methodist Hospital - Department of Urology
Houston, Texas, United States
University of Texas - MD Anderson Cancer Center
Houston, Texas, United States
Huntsman Cancer Institute, University of Utah
Salt Lake City, Utah, United States
University of Virginia Comprehensive Cancer Center
Charlottesville, Virginia, United States
Froedtert Hospital / Medical College of Wisconsin
Milwaukee, Wisconsin, United States
Sydney Adventist Hospital
Wahroonga, New South Wales, Australia
Gold Coast University Hospital
Southport, Queensland, Australia
Icon Cancer Center Windsor Gardens
Windsor Gardens, South Australia, Australia
Prostate Cancer Centre
Calgary, Alberta, Canada
Vancouver Prostate Centre
Vancouver, British Columbia, Canada
Nova Scotia Health Authority
Halifax, Nova Scotia, Canada
University Health Network, Princess Margaret Cancer Centre
Toronto, Ontario, Canada
McGill University Health Center - Glen site
Montreal, Quebec, Canada
CHUM Centre Hospitalier de l Universite de Montreal
Montreal, Quebec, Canada
CHU d'Angers
Angers, , France
CHU Bordeaux Pellegrin
Bordeaux, , France
lnstitut Bergonie 229 Cour de l'Argonne
Bordeaux, , France
CHU de Lille
Lille, , France
Hospices Civils de Lyon
Lyon, , France
Hopital Bichat Claude-Bernard
Paris, , France
CHU de Rauen- Hopital Charles Nicolle
Rouen, , France
Klinik fur Urologie, Uro-Onkologie, spezielle urologische und Roboter-assistierte Chirurgie Universitatsklinikum Koln (AOR)
Cologne, , Germany
Urologicum Duisburg
Duisburg, , Germany
Universitaetsklinikum Erlangen
Erlangen, , Germany
Urologie Neandertal Mettmann
Mettmann, , Germany
Studienpraxis Urologie
Nürtingen, , Germany
Universitaetsklinikum Tubingen
Tübingen, , Germany
Clinic Unit of Urology, IRCCS Ospedale San Raffaele
Milan, MI, Italy
Istituto Europeo di Oncologia
Milan, , Italy
Istituto Nazionale Tumori IRCCS Fondazione G. Pascale
Napoli, , Italy
Fondazione Policlinico Universitario "A.Gemelli" IRCCS, Largo
Rome, , Italy
Azienda Ospedaliero-Universitaria Sant'Andrea
Rome, , Italy
UOC Urologia, IFO- lstituto Tumori "Regina Elena"
Rome, , Italy
Seoul National University Bundang Hospital
Seongnam, Gyunggi-do, South Korea
Chonnam National University Hwasun Hospital
Hwasun, Jeollanam-do, South Korea
Seoul National University Hospital
Seoul, , South Korea
Severance Hospital, Yonsei University Health System
Seoul, , South Korea
The Catholic University of Korea, Soul St. Mary's Hospital
Seoul, , South Korea
IOB - Hospital Quironsalud Barcelona
Barbera Del Valles, , Spain
Fundacio Puigvert
Barcelona, , Spain
Hospital Clinic Barcelona
Barcelona, , Spain
Hospital Germans Trias i Pujol
Barcelona, , Spain
Hospital Universitari de Bellvitge
L'Hospitalet de Llobregat, , Spain
Hospital General Universitario Gregorio Maranon
Madrid, , Spain
Hospital Universitario 12 de Octubre
Madrid, , Spain
Hospital Universitario La Paz
Madrid, , Spain
Hospital Quironsalud Madrid (Next Oncology)
Madrid, , Spain
Hospital Universitario Infanta Sofia
Madrid, , Spain
Hospital General Universitario Morales Meseguer
Murcia, , Spain
Hospital Universitario Marqués de Valdecilla,
Santander, , Spain
Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, , Taiwan
China Medical University Hospital
Taichung, , Taiwan
Taichung Veterans General Hospital
Taichung, , Taiwan
Chi Mei Medical Center
Tainan, , Taiwan
National Taiwan University Hospital
Taipei, , Taiwan
The Royal Marsden NHS Foundation Trust
London, , United Kingdom
Countries
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Central Contacts
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Chris Tosone
Role: CONTACT
Facility Contacts
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Susan Kalota
Role: primary
Edward Uchio
Role: primary
Salmasi
Role: primary
Anne Schuckman
Role: primary
Terry Williams
Role: primary
Alanna Gavriushina
Role: primary
Katayune Golshan
Role: backup
Michael Whalen
Role: primary
Kethandapatti Balaji
Role: primary
Shreyas Joshi
Role: primary
Faith Rahman
Role: primary
Laura Mitchell
Role: backup
Rian Dickstein
Role: primary
Mark Preston
Role: primary
Saachi Pawa
Role: backup
Kevin Ginsburg
Role: primary
Johar Raza, MD
Role: primary
Conrad Tobert
Role: primary
Marissa Twedt
Role: primary
Joseph Zabell
Role: backup
Shah
Role: primary
Packiam
Role: primary
Gordon Brown
Role: primary
John Sfakianos
Role: primary
Marc Bjurlin
Role: primary
Mark Jalkut
Role: primary
Mohammed Kamel
Role: primary
Kara Lasorella
Role: primary
J Liu
Role: primary
Neal Shore
Role: primary
Jayram
Role: primary
Chang
Role: primary
Jose Santoyo
Role: primary
Yair Lotan
Role: backup
Gelpi-Hammerschmidt
Role: primary
Taliah Muhammad
Role: primary
Ashish Kamat, MD
Role: primary
Christine Ibilibor
Role: primary
Scott Johnson
Role: primary
Ricardo Rendon
Role: primary
Robin Simpson
Role: backup
W Kassouf
Role: primary
K Jaureguisanchez
Role: backup
Other Identifiers
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EG-70-101
Identifier Type: -
Identifier Source: org_study_id