LEGEND Study: EG-70 in NMIBC Patients BCG-Unresponsive and High-Risk NMIBC Incompletely Treated With BCG or BCG-Naïve

NCT04752722 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: EG-70-101
• Study Type: interventional
• Target: 350
• Locations: 10 countries
• Sites: 101

Brief Summary

This study will evaluate the safety and efficacy of intravesical administration of EG-70 in the bladder and its effect on bladder tumors in patients with NMIBC. 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.

Conditions

Superficial Bladder Cancer; Non-muscle Invasive Bladder Cancer With Carcinoma in Situ

Keywords

Non-muscle invasive bladder cancer (NMIBC); Bacillus calmette- guerin (BCG) failure; BCG unresponsive; NMIBC; Bladder Cancer; LEGEND Study; EG-70; High-risk NMIBC; BCG-naïve; Incomplete BCG treatment; Carcinoma in situ (Cis); BCG-exposed

Outcome Measures

Primary Outcomes (3)

• Phase 1: Nature, incidence, relatedness, and severity of all AEs and SAEs according to the CTCAE v5.0.

  The type, incidence, relatedness and severity of treatment emergent adverse events of EG-70 as assessed by NCI-CTCAE V5.0 will be monitored.

  Approximately 2 years

• Phase 2: Percentage of patients with cystoscopic CR at 48 weeks, based on exam, urine cytology and appropriate biopsies.

  Complete response rate will be measured by determining the number of patients without recurrence of high-grade disease.

  Approximately 48 weeks

• Phase 2: Nature, incidence, relatedness, and severity of treatment emergent adverse events (as assessed by CTCAE v5.0)

  The type, incidence, relatedness and severity of treatment emergent adverse events of EG-70 as assessed by NCI-CTCAE V5.0 will be monitored.

  Approximately 3 years

Secondary Outcomes (6)

• Phase 1: The number of patients who experience a DLT through the end of Cycle 1

  Approximately 12 Weeks

• Phase 1: CR rate to EG-70 by cystoscopy at approximately 12 weeks.

  Approximately 12 weeks

• Phase 2: Progression-free survival (PFS)

  Approximately 3 years

• Phase 2: CR rate at 12, 24, 36, and 96 weeks

  Approximately 12, 24, 36, and 96 weeks

• Phase 2: Duration of response of the responding patients

  Approximately 3 years

Study Arms (2)

Phase 1

EXPERIMENTAL

Dose escalation phase

Drug: EG-70 (phase 1)

Phase 2

EXPERIMENTAL

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

Drug: EG-70 (phase 2)

Interventions

EG-70 (phase 1) DRUG

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.

Also known as: Phase 1

Phase 1

EG-70 (phase 2) DRUG

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.

Also known as: Phase 2

Phase 2

Eligibility Criteria

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

You may qualify if:

• BCG-unresponsive Patients:
• 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:
• 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:
• Male or non-pregnant, non-lactating female, 18 years or older.
• Women of childbearing potential must have a negative pregnancy test at Screening.
• 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.
• 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.
• Performance Status: Eastern Cooperative Oncology Group 0, 1, and 2.
• Adequate renal function with creatinine clearance \>30 mL/min
• Prothrombin time and partial thromboplastin time ≤1.25 x ULN or within the therapeutic range if on anticoagulation therapy.
• Must have satisfactory bladder function with ability to retain study drug for 60 minutes.

You may not qualify if:

• Active malignancies (i.e., progressing or requiring treatment change in the last 24 months). Exceptions allowed under Sponsor review.
• Concurrent treatment with any chemotherapeutic agent.
• History of partial cystectomy.
• Treatment with last therapeutic agent (including intravesical chemotherapy post-TURBT) within 30 days of Screening (prior to the screening biopsy).
• Patients who have received systemic immunosuppressive medication including high-dose corticosteroids.
• History of severe asthma or other respiratory diseases.
• History of unresolved vesicoureteral reflux or an indwelling urinary stent.
• History of unresolved hydronephrosis due to ureteral obstruction.
• 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.
• History of external beam radiation to the pelvis or prostate brachytherapy within the last 12 months.
• History of interstitial lung disease and/or pneumonitis in patients who have previously received a PD-1 or PD-L1 inhibitor therapy.
• Evidence of metastatic disease.
• History of difficult catheterization that in the opinion of the Investigator will prevent administration of EG-70.
• Active interstitial cystitis on cystoscopy or biopsy.
• Active, uncontrolled bacterial, viral, or fungal infection(s) requiring systemic therapy.

Sponsors & Collaborators

• enGene, Inc.: lead

Study Sites (101)

The University of Alabama at Birmingham Clinical Research Unit (CRU)

Birmingham, Alabama, 35249, United States

RECRUITING

Mayo Clinic

Scottsdale, Arizona, 85259, United States

RECRUITING

Urological Associates of South Arizona

Tucson, Arizona, 85715, United States

COMPLETED

Arkansas Urology

Little Rock, Arkansas, 72211, United States

RECRUITING

University of California - Irvine Medical Center

Irvine, California, 92697, United States

RECRUITING

UC San Diego Moores Cancer Center

La Jolla, California, 92037, United States

RECRUITING

USC/Norris Comprehensive Cancer Center

Los Angeles, California, 90033, United States

RECRUITING

Tower Urology

Los Angeles, California, 90048, United States

RECRUITING

Om Research

San Diego, California, 92123, United States

RECRUITING

Colorado Clinical Research

Lakewood, Colorado, 80228, United States

RECRUITING

The George Washington Medical Faculty Associates

Washington D.C., District of Columbia, 20037, United States

RECRUITING

University of Florida

Jacksonville, Florida, 32209, United States

RECRUITING

Sylvester Comprehensive Cancer Center / University of Miami Hospital and Clinics

Miami, Florida, 33136, United States

RECRUITING

Emory University

Atlanta, Georgia, 30322, United States

RECRUITING

Rush University Medical Center

Chicago, Illinois, 60607, United States

RECRUITING

Urology of Indiana

Greenwood, Indiana, 46143, United States

RECRUITING

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

RECRUITING

John Hopkins Hospital

Baltimore, Maryland, 21287, United States

RECRUITING

Chesapeake Urology Research Associates

Hanover, Maryland, 21076, United States

RECRUITING

Brigham and Women's Hospital

Boston, Massachusetts, 45227, United States

RECRUITING

Henry Ford Health System

Detroit, Michigan, 48202, United States

RECRUITING

Corewell Health Medical Group and Spectrum Health Hospitals

Grand Rapids, Michigan, 49503, United States

RECRUITING

University of Minnesota

Minneapolis, Minnesota, 55455, United States

RECRUITING

Mayo Clinic

Rochester, Minnesota, 55905, United States

RECRUITING

Rutgers Cancer Institute of New Jersey

New Brunswick, New Jersey, 08901, United States

RECRUITING

New Jersey Urology, LLC

Voorhees Township, New Jersey, 08043, United States

RECRUITING

Albany Medical College

Albany, New York, 12208, United States

RECRUITING

Mount Sinai Medical Center

New Haven, New York, 10029, United States

RECRUITING

Laura & Isaac Perlmutter Cancer Center at NYU Langone Health

New York, New York, 10016, United States

COMPLETED

Associated Medical Professionals of NY,

Syracuse, New York, 13210, United States

RECRUITING

UNC Chapel Hill Hospital

Chapel Hill, North Carolina, 27514, United States

RECRUITING

Duke Health - Duke Cancer Center

Durham, North Carolina, 27710, United States

RECRUITING

Associated Urologists of North Carolina

Raleigh, North Carolina, 27612, United States

RECRUITING

University of Cincinnati Medical Center

Cincinnati, Ohio, 45219, United States

RECRUITING

Central Ohio Urology Group

Gahanna, Ohio, 43230, United States

RECRUITING

Clinical Research Solutions - Helios Clinical Research

Middleburg Heights, Ohio, 44130, United States

RECRUITING

Oregon Health & Science University (OHSU)

Portland, Oregon, 97239, United States

RECRUITING

Thomas Jefferson University, Sidney Kimmel Cancer Center

Philadelphia, Pennsylvania, 19107, United States

RECRUITING

Carolina Urologic Research Center, LLC

Myrtle Beach, South Carolina, 29572, United States

RECRUITING

Urology Associates, P.C.

Nashville, Tennessee, 37209, United States

RECRUITING

Vanderbilt Univerity Medical Center

Nashville, Tennessee, 37232, United States

RECRUITING

Urology Austin

Austin, Texas, 78745, United States

RECRUITING

UT Southwestern Medical Center

Dallas, Texas, 75390, United States

RECRUITING

Houston Metro Urology

Houston, Texas, 77027, United States

RECRUITING

Houston Methodist Hospital - Department of Urology

Houston, Texas, 77030, United States

RECRUITING

University of Texas - MD Anderson Cancer Center

Houston, Texas, 77030, United States

RECRUITING

Huntsman Cancer Institute, University of Utah

Salt Lake City, Utah, 84112, United States

RECRUITING

University of Virginia Comprehensive Cancer Center

Charlottesville, Virginia, 22903, United States

RECRUITING

Froedtert Hospital / Medical College of Wisconsin

Milwaukee, Wisconsin, 52336, United States

RECRUITING

Sydney Adventist Hospital

Wahroonga, New South Wales, 2076, Australia

RECRUITING

Gold Coast University Hospital

Southport, Queensland, 4215, Australia

RECRUITING

Icon Cancer Center Windsor Gardens

Windsor Gardens, South Australia, 5087, Australia

RECRUITING

Prostate Cancer Centre

Calgary, Alberta, T2V1P9, Canada

RECRUITING

Vancouver Prostate Centre

Vancouver, British Columbia, V5Z1M9, Canada

RECRUITING

Nova Scotia Health Authority

Halifax, Nova Scotia, B3H2Y9, Canada

RECRUITING

University Health Network, Princess Margaret Cancer Centre

Toronto, Ontario, M5G 2M9, Canada

RECRUITING

McGill University Health Center - Glen site

Montreal, Quebec, H4A3J1, Canada

RECRUITING

CHUM Centre Hospitalier de l Universite de Montreal

Montreal, Quebec, Canada

RECRUITING

CHU d'Angers

Angers, 49933, France

RECRUITING

CHU Bordeaux Pellegrin

Bordeaux, 33076, France

RECRUITING

lnstitut Bergonie 229 Cour de l'Argonne

Bordeaux, 33076, France

RECRUITING

CHU de Lille

Lille, 59000, France

RECRUITING

Hospices Civils de Lyon

Lyon, 69002, France

RECRUITING

Hopital Bichat Claude-Bernard

Paris, 75018, France

RECRUITING

CHU de Rauen- Hopital Charles Nicolle

Rouen, 76031, France

RECRUITING

Klinik fur Urologie, Uro-Onkologie, spezielle urologische und Roboter-assistierte Chirurgie Universitatsklinikum Koln (AOR)

Cologne, 50937, Germany

RECRUITING

Urologicum Duisburg

Duisburg, 47169, Germany

RECRUITING

Universitaetsklinikum Erlangen

Erlangen, 91054, Germany

RECRUITING

Urologie Neandertal Mettmann

Mettmann, 40822, Germany

RECRUITING

Studienpraxis Urologie

Nürtingen, 72622, Germany

RECRUITING

Universitaetsklinikum Tubingen

Tübingen, 72076, Germany

RECRUITING

Clinic Unit of Urology, IRCCS Ospedale San Raffaele

Milan, MI, 20132, Italy

RECRUITING

Istituto Europeo di Oncologia

Milan, 20141, Italy

RECRUITING

Istituto Nazionale Tumori IRCCS Fondazione G. Pascale

Naples, 80131, Italy

RECRUITING

Fondazione Policlinico Universitario "A.Gemelli" IRCCS, Largo

Rome, 00168, Italy

RECRUITING

Azienda Ospedaliero-Universitaria Sant'Andrea

Rome, 00189, Italy

RECRUITING

UOC Urologia, IFO- lstituto Tumori "Regina Elena"

Rome, 53 00144, Italy

RECRUITING

Ospedelae Molinette San Giovanni Battista Di Torino

Torino, 10126, Italy

RECRUITING

Seoul National University Bundang Hospital

Seongnam, Gyunggi-do, 13620, South Korea

RECRUITING

Chonnam National University Hwasun Hospital

Hwasun, Jeollanam-do, 58128, South Korea

RECRUITING

Seoul National University Hospital

Seoul, 03080, South Korea

RECRUITING

Severance Hospital, Yonsei University Health System

Seoul, 03722, South Korea

RECRUITING

The Catholic University of Korea, Soul St. Mary's Hospital

Seoul, 06591, South Korea

RECRUITING

IOB - Hospital Quironsalud Barcelona

Barbera Del Valles, 5-7, Spain

RECRUITING

Fundacio Puigvert

Barcelona, 08025, Spain

RECRUITING

Hospital Clinic Barcelona

Barcelona, 08036, Spain

RECRUITING

Hospital Germans Trias i Pujol

Barcelona, 08916, Spain

RECRUITING

Hospital Universitari de Bellvitge

L'Hospitalet de Llobregat, 08907, Spain

RECRUITING

Hospital General Universitario Gregorio Maranon

Madrid, 28007, Spain

RECRUITING

Hospital Universitario 12 de Octubre

Madrid, 28041, Spain

RECRUITING

Hospital Universitario La Paz

Madrid, 28046, Spain

RECRUITING

Hospital Quironsalud Madrid (Next Oncology)

Madrid, 28223, Spain

RECRUITING

Hospital Universitario Infanta Sofia

Madrid, 28702, Spain

RECRUITING

Hospital General Universitario Morales Meseguer

Murcia, 30008, Spain

RECRUITING

Hospital Universitario Marqués de Valdecilla,

Santander, 39008, Spain

RECRUITING

Kaohsiung Medical University Chung-Ho Memorial Hospital

Kaohsiung City, 807, Taiwan

RECRUITING

China Medical University Hospital

Taichung, 40447, Taiwan

RECRUITING

Taichung Veterans General Hospital

Taichung, 40705, Taiwan

RECRUITING

Chi Mei Medical Center

Tainan, 71004, Taiwan

RECRUITING

National Taiwan University Hospital

Taipei, 100, Taiwan

RECRUITING

The Royal Marsden NHS Foundation Trust

London, SW3 6JJ, United Kingdom

RECRUITING

MeSH Terms

Conditions

Non-Muscle Invasive Bladder Neoplasms; Urinary Bladder Neoplasms; Carcinoma in Situ

Interventions

Clinical Trials, Phase I as Topic; Clinical Trials, Phase II as Topic

Condition Hierarchy (Ancestors)

Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms; Urologic Neoplasms; Urogenital Neoplasms; Neoplasms by Site; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Urinary Bladder Diseases; Urologic Diseases; Male Urogenital Diseases

Intervention Hierarchy (Ancestors)

Clinical Trials as Topic; Clinical Studies as Topic; Epidemiologic Study Characteristics; Epidemiologic Methods; Investigative Techniques; Health Care Evaluation Mechanisms; Quality of Health Care; Health Care Quality, Access, and Evaluation; Public Health; Environment and Public Health

Study Officials

• Christine Tosone, Ms, RAC

  enGene, Inc.

  STUDY DIRECTOR

Central Study Contacts

enGene clinical trials

CONTACT

+18572991097; clinicaltrials@engene.com

Chris Tosone

CONTACT

Study Design

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

Study Record Dates

• First Submitted: February 4, 2021
• First Posted: February 12, 2021
• Study Start: April 22, 2021
• Primary Completion (Estimated): June 30, 2026
• Study Completion (Estimated): November 30, 2028
• Last Updated: April 30, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

CA (6); FR (7); ES (12); TW (5); US (49); IT (7); DE (6); GB (1); AU (3); KR (5)