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BRIEF TITLE: EG-70 as an Intravesical Administration to Patients With BCG Unresponsive Non-Muscle Invasive Bladder Cancer (NMIBC)

A Phase 1/2 Study of EG-70 as an Intravesical Administration to Patients With BCG Unresponsive Non-Muscle Invasive Bladder Cancer (NMIBC)


  • Org Study ID: EG-70-101
  • Secondary ID:
  • NCT ID: NCT04752722
  • NCT Alias:
  • Sponsor: enGene, Inc. - Industry
  • Source: enGene, Inc.

Brief Summary

This open-label, multicenter study will evaluate the safety and efficacy of intravesical administration of EG-70 and its effect on bladder tumors in patients with NMIBC who have failed BCG therapy and are recommended for radical cystectomy. This study study consists of two parts, a dose-escalation Phase (Phase 1) to establish safety and recommended phase 2 dose (RP2D), followed by a Phase 2 efficacy study at the RP2D to establish efficacy.

Detailed Description


EG-70 is a novel non-viral gene therapy. EG-70 is designed to elicit a local immune response
following delivery of gene therapies to mucosal tissues, such as 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.

Overal Status Start Date Phase Study Type
Recruiting March 2021 Phase 1/Phase 2 Interventional

Primary Outcomes:

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

Primary Outcome 1 - Time Frame: Approximately 2 years

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

Primary Outcome 2 - Time Frame: Approximately 48 weeks

Primary Outcome 3 - Measure: Phase 2: Nature, incidence, relatedness, and severity of treatment emergent adverse events (as assessed by CTCAE v5.0)

Primary Outcome 3 - Time Frame: Approximately 3 years

Condition:

  • Superficial Bladder Cancer

Eligibility

Criteria:
Inclusion Criteria:

1. BCG-unresponsive NMIBC with carcinoma in situ (CIS) with or without resected papillary
tumors who are indicated for, ineligible for, or have elected not to undergo
cystectomy:

1. persistent high-grade disease (Ta, T1, or Tis) or recurrence within 6 months of
receiving at least 2 courses of intravesical BCG (at least 5 of 6 induction doses
and at least 2 of 3 maintenance doses).

2. T1 high grade disease residual at the first evaluation following induction BCG
(at least 5 of 6 doses).

2. 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).

3. Male or non-pregnant, non-lactating female, 18 years or older.

4. 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:

1. has had a hysterectomy or bilateral oophorectomy or

2. is age ≥ 60 years and is amenorrhoeic or

3. 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).

5. All patients of childbearing potential must be willing to consent to using effective
double-barrier contraception, i.e., intrauterine device, birth control pills,
depo-provera, and condoms while on treatment and for 3 months after their
participation in the study ends.

6. Performance Status: Eastern Cooperative Oncology Group (ECOG) 0, 1, and 2.

7. Hematologic inclusion within 2 weeks of start of treatment:

1. Absolute neutrophil count >1,500/mm3.

2. Hemoglobin >9.0 g/dl.

3. Platelet count >100,000/mm3.

8. Hepatic inclusion within 2 weeks of Day 1:

1. Total bilirubin must be ≤1.5 x the upper limit of normal (ULN).

2. Adequate renal function with creatinine clearance >30 mL/min (measured using
Cockcroft-Gault equation or the estimated glomerular filtration rate from the
Modification of Diet in Renal Disease Study).

3. 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.

9. Prothrombin time and partial thromboplastin time within the normal limits at
Screening.

10. Must have satisfactory bladder function with ability to retain study drug for a
minimum of 60 minutes.

11. Must be willing and able to comply with all protocol requirements.

12. Must be willing and able to give informed consent and any authorizations required by
local law for participation in the study.

Exclusion Criteria:

1. 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.

2. Concurrent treatment with any chemotherapeutic agent.

3. Treatment with pembrolizumab within 30 days (Phase 1) or 3 months (Phase 2) prior to
Screening.

4. Treatment with last therapeutic agent 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).

5. Women who are pregnant or lactating.

6. History of vesicoureteral reflux or an indwelling urinary stent.

7. Participation in any other research protocol involving administration of an
investigational agent within 1 month prior to Day 1.

8. History of radiation to the pelvis.

9. History of interstitial lung disease and/or pneumonitis in patients who have
previously received a PD-1 or PD-L1 inhibitor therapy.

10. Evidence of metastatic disease.

11. History of difficult catheterization that in the opinion of the Investigator will
prevent administration of EG-70.

12. History of interstitial cystitis.

13. Active, uncontrolled bacterial, viral, or fungal infection(s) requiring systemic
therapy.

14. Known human immunodeficiency virus (HIV), Hepatitis B, or Hepatitis C infection.

15. Significant cardiovascular risk (e.g., coronary stenting within 8 weeks, myocardial
infarction within 6 months).
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Gender: All

Minimum Age: 18 Years

Maximum Age: N/A

Healthy Volunteers: No

Overall Contact

Name: Bao Le

Phone: 5143324888

Email: clinicaltrials@engene.com

Location

Facility Status Contact
New Jersey Urology, LLC
Voorhees, New Jersey 08043
United States
Recruiting