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

  • End date
    May 15, 2027
  • participants needed
  • sponsor
    enGene, Inc.
Updated on 15 September 2023


This study will evaluate the safety and efficacy of intravesical administration of EG-70 and 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 for whom BCG therapy is unresponsive and are recommended for radical cystectomy, or high-risk NMIBC patients who are BCG-naïve or have received incomplete BCG treatment.


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 patients will be enrolled in Phase 1, and Cohort 1 of Phase 2. Eligible high-risk NMIBC patients who have been incompletely treated or are BCG-naïve will be enrolled starting in Phase 2 in a separate single-arm cohort (Cohort 2).

Patients will be treated for up to four 12-week cycles of study drug instillation doses and assessments with follow up assessments.

Condition Superficial Bladder Cancer
Treatment EG-70
Clinical Study IdentifierNCT04752722
SponsorenGene, Inc.
Last Modified on15 September 2023


Yes No Not Sure

Inclusion Criteria

BCG-unresponsive Patients
BCG-unresponsive NMIBC with carcinoma in situ (CIS) with or without resected papillary tumors who are ineligible for or have elected not to undergo cystectomy
persistent high-grade disease (Ta, T1, or Tis) after receiving intravesical BCG induction (at least 5 of 6 induction doses) plus maintenance (at least 2 of 3 doses) or recurrence of high-grade papillary disease within 6 months or Tis within 12 months of BCG instillation or
T1 high grade disease residual at the first evaluation following induction BCG (at least 5 of 6 doses)
BCG-Naïve or BCG-incompletely treated Patients (Phase 2 Only)
NMIBC with Cis with or without resected papillary tumors who are ineligible for or
have elected not to undergo cystectomy
has had a hysterectomy or bilateral oophorectomy or
persistent high-grade disease (Ta, T1, or Tis)
after incomplete BCG treatment (at least 1 dose) or
who have not yet received any treatment with BCG, but who have previously been
treated with at least 1 dose of intravesical chemotherapy following transurethral
Performance Status: Eastern Cooperative Oncology Group (ECOG) 0, 1, and 2
resection of bladder tumor (TURBT)
Hematologic inclusion within 2 weeks of start of treatment
All Patients
Absolute neutrophil count >1,500/mm3
Hemoglobin >9.0 g/dl
Platelet count >100,000/mm3
Hepatic inclusion within 2 weeks of Day 1
Patients who have previously been treated with an investigational or approved
checkpoint inhibitor (e.g., pembrolizumab) and failed treatment are eligible for
inclusion 30 days post-treatment (Phase 1) or 3 months post-treatment (Phase 2)
Male or non-pregnant, non-lactating female, 18 years or older
Women of childbearing potential must have a negative pregnancy test at Screening. A
female patient is considered to be of child-producing potential unless she
is age ≥ 60 years and is amenorrhoeic or
is age < 60 years and has been amenorrhoeic for ≥ 12 months (including no
irregular menses or spotting) in the absence of any medication which induces a
menopausal state and has documented ovarian failure by serum oestradiol and
follicle-stimulating hormone levels within the institutional laboratory
postmenopausal range)
All patients of childbearing potential must be willing to consent to using effective
In Phase 2, for patients with T1 lesions, Screening biopsy must be considered adequate
double-barrier contraception, i.e., intrauterine device, birth control pills
(contain the muscularis layer)
depo-provera, and condoms while on treatment and for 3 months after their
participation in the study ends
Total bilirubin must be ≤1.5 x the upper limit of normal (ULN)
Adequate renal function with creatinine clearance >30 mL/min
Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤2.5 x ULN
for the institution, alkaline phosphatase ≤2.5 x ULN for the institution, unless
bone metastasis is present in the absence of liver metastasis
Prothrombin time and partial thromboplastin time within the normal limits at
Must have satisfactory bladder function with ability to retain study drug for a
minimum of 60 minutes
Patient or legally authorized representative (LAR) must be willing and able to comply
with all protocol requirements
Patient or LAR must be willing and able to give informed consent and any
authorizations required by local law for participation in the study

Exclusion Criteria

Concurrent treatment with any chemotherapeutic agent
Evidence of metastatic disease
History of interstitial cystitis
Known human immunodeficiency virus (HIV), Hepatitis B, or Hepatitis C infection
History of partial cystectomy
Any other malignancy diagnosed within 1 year of study entry (except basal or squamous
cell skin cancers or noninvasive cancer of the cervix) is excluded
Treatment with last therapeutic agent (including intravesical chemotherapy post-TURBT)
Treatment with pembrolizumab within 30 days (Phase 1) or 3 months (Phase 2) prior to
within 30 days of Screening (Phase 1 and Phase 2) or treatment with an investigational
checkpoint inhibitor within 3 months of Screening (Phase 2 only)
Evidence of persistent or ongoing renal failure
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 1 month prior to Day 1
History of external beam radiation to the pelvis at any time 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
History of difficult catheterization that in the opinion of the Investigator will
prevent administration of EG-70
Active, uncontrolled bacterial, viral, or fungal infection(s) requiring systemic
Significant cardiovascular risk (e.g., coronary stenting within 8 weeks, myocardial
infarction within 6 months)
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