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Looking for participantsPhase1/Phase2

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

Sponsor: enGene, Inc.

NCT ID: NCT04752722

View on ClinicalTrials.gov ↗

At a glance

What the study gets you
Access to the study treatment being tested
Type of study
Interventional (receives a drug or procedure)
Time in hospital
In-person visits at study sites — visit count not specified by the sponsor
Drug or intervention
EG-70 (phase 1) (drug), EG-70 (phase 2) Master Protocol (drug), Surfactant Bladder Pre-Rinse and EG-70 (Substudy) (drug)
How long the study runs
Study runs about 114 months (dates as stated)
About the drug or intervention
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. · EG-70 (phase 2) Master Protocol — drug: Under the Treatment Period, patients will receive 4 instillations per cycle for 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. · Surfactant Bladder Pre-Rinse and EG-70 (Substudy) — drug: Patients will receive via catheter a 50 mL volume of a 5-minute bladder rinse immediately prior to administration of 50 mL of detalimogene voraplasmid (EG-70) at the RP2D for 30 minutes.
Patient visit burden
Not specified by the sponsor

In plain English

The LEGEND Study is testing a new treatment called EG-70 for people with non-muscle invasive bladder cancer (NMIBC), which is cancer in the inner layers of the bladder that has not spread into the muscle. It includes people whose cancer did not respond to BCG (a standard bladder cancer treatment given directly into the bladder), as well as some who have never had BCG or did not finish a full course. EG-70 is given through a tube (catheter) into the bladder, and patients need to be able to hold it in the bladder for 60 minutes.

Who can take part

  • Adults aged 18 or over, male or female (women must not be pregnant or breastfeeding, and a pregnancy test is needed at screening for women who can become pregnant).
  • People with high-grade non-muscle invasive bladder cancer, including a type called carcinoma in situ (CIS), which may be with or without papillary tumours (Ta/T1).
  • People whose cancer did not respond to BCG treatment (called BCG-unresponsive) after at least two proper courses of BCG, who cannot have or have chosen not to have surgery to remove the bladder (cystectomy).
  • In Phase 2 only: people who have never had BCG, or who had an incomplete course of BCG, with CIS; and people who are BCG-unresponsive with high-grade Ta/T1 papillary disease without CIS.
  • People who have already tried a checkpoint inhibitor (a type of immunotherapy) that did not work, as long as enough time has passed since that treatment (30 days in Phase 1, 3 months in Phase 2).
  • Reasonably fit and well day-to-day (performance status score of 0, 1 or 2), with healthy enough blood, liver, kidney and blood-clotting test results.
  • Able to hold the study drug in the bladder for 60 minutes.
  • In Phase 2, people with T1 tumours may join after a repeat TURBT (an operation to remove bladder tumours) if follow-up tests show the disease is non-invasive or gone.
  • Agreement to use highly effective contraception: women who can become pregnant must use effective birth control; men must use condoms during treatment and for 3 months after the last dose.

Who may not be able to

  • Another cancer that is active or needed a change of treatment in the last 24 months (exceptions may be allowed).
  • Currently having chemotherapy.
  • Previous surgery to remove part of the bladder.
  • Cancer treatment (including chemotherapy placed in the bladder after tumour removal) within 30 days before screening.
  • Taking medicines that suppress the immune system, including high-dose steroids.
  • History of severe asthma or other serious lung disease.
  • Ongoing urine flowing backwards from the bladder to the kidneys (vesicoureteral reflux), or a stent left in the urinary system.
  • Ongoing kidney swelling caused by a blocked ureter (the tube from kidney to bladder).
  • Taking part in another study drug trial within the last 30 days, or having had a previous experimental gene or immunotherapy for this type of bladder cancer.
  • Radiotherapy to the pelvis or prostate brachytherapy (internal radiation) in the last 12 months.
  • History of interstitial lung disease or lung inflammation, if previously treated with a PD-1 or PD-L1 inhibitor (a type of immunotherapy).
  • Evidence the cancer has spread to other parts of the body.
  • A history of problems having a catheter fitted that would stop EG-70 being given.
  • Active interstitial cystitis (long-term bladder inflammation) seen on examination or biopsy.
  • An active infection needing treatment.
  • Known HIV, hepatitis B or hepatitis C infection.
  • Serious heart problems (for example, a stent fitted in the last 8 weeks, or a heart attack in the last 6 months).
  • Allergy to any of the other ingredients in the study drug. For the bladder rinse groups, a known allergy to polidocanol.

What taking part involves

  • • EG-70 is given directly into the bladder through a catheter (a thin tube).
  • • You need to keep the medicine in your bladder for 60 minutes after it is given.
  • • The trial has two phases, and which phase you join affects some of the entry rules.

Time commitment: Not stated — ask the trial team, as the registry data does not give the number of visits, how long the study lasts, or how often treatment is given.

Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.

Type of study
Testing a treatment
Ages
18 Years and over
Who
All
Number of participants
350
Started
2021-04-22
Last checked
2026-08

Plain English Summary

What is this study?

  • • Testing a new treatment for superficial bladder cancer
  • • Phase1/Phase2 - 350 participants
  • • This study will evaluate the safety and efficacy of intravesical administration of detalimogene (EG-70) in the bladder and its effect on bladder tumors in patients with NMIBC

Who can take part?

  • • Ages 18 Years and over
  • • Diagnosed with superficial bladder cancer

Where?

  • • London - The Royal Marsden NHS Foundation Trust

This is a simplified summary. Always discuss with your doctor before making any decisions.

About This Trial

This study will evaluate the safety and efficacy of intravesical administration of detalimogene (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 other high risk patients with NMIBC. A Substudy will include a surfactant bladder rinse prior to the instillation of detalimogene in patients with NMIBC with CIS for whom BCG therapy is unresponsive.

More detail

EG-70 is a novel non-viral gene therapy. EG-70 is designed to elicit a local immune response following delivery of the study gene therapy to the bladder urothelium. This approach of local administration through bladder instillation has the potential to induce a potent immune response exclusively at the site of the tumor, resulting in greater therapeutic benefit while reducing undesirable systemic toxicity. Eligible BCG-unresponsive NMIBC with CIS patients will be enrolled in Phase 1, and Cohort 1 of Phase 2. Eligible high-risk NMIBC patients will be enrolled in Phase 2 into separate 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 four treatment cycles will enter up to 8 maintenance treatment cycles.

Superficial Bladder CancerNon-muscle Invasive Bladder Cancer With Carcinoma in SituNon-muscle Invasive Bladder Cancer

How this trial compares with your answers

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What we know so far

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Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.

Eligibility at a Glance

Key info

  • Age: 18 Years and over
  • Who can join: All genders

Biomarkers mentioned

have a negativePD-L1

Treatment history

Treatments you must have had:

  • ✓ a negative pregnancy test at Screening
  • ✓ to utilize a condom for the duration of the study treatment through 3 months post-dose
  • ✓ satisfactory bladder function with ability to retain study drug for 60 minutes

What the study is looking for

  • ✓BCG-unresponsive Patients:
  • ✓Phase 2 Only:
  • ✓BCG-Naïve or BCG-incompletely treated Patients with CIS or BCG-unresponsive, HG Ta/T1 papillary disease without CIS:
  • ✓All Patients with High Grade NMIBC:
  • ✓Patients who have previously been treated with a checkpoint inhibitor and failed treatment are eligible for...

Who cannot take part

  • ✗Active malignancies (i.e., progressing or requiring treatment change in the last 24 months). Exceptions allowed...
  • ✗Concurrent treatment with any chemotherapeutic agent.
  • ✗History of partial cystectomy.
  • ✗Treatment with last therapeutic agent (including intravesical drug treatment post-TURBT) within 30 days of Screening...
  • ✗Patients who have received systemic immunosuppressive medication including high-dose corticosteroids.
See the full criteria
Inclusion Criteria: BCG-unresponsive Patients: 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 with High Grade NMIBC: 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: 1. Active malignancies (i.e., progressing or requiring treatment change in the last 24 months). Exceptions allowed under Sponsor review. 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. Exclusionary for Bladder Rinse cohorts: known allergy to polidocanol

Where Is This Study? (1 UK site)

The Royal Marsden NHS Foundation Trust

London SW3 6JJ, United Kingdom

Recruiting
Hospital R&D contact (matched)

Mark Brandon-Grove

research.development@rmh.nhs.uk020 3186 5416

How to Get in Touch

enGene clinical trials

Sponsor contact

CONTACT

+18572991097 clinicaltrials@engene.com

Chris Tosone

Sponsor contact

CONTACT

Data sourced from ClinicalTrials.gov · Last verified: 2026-08