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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04752722




Registration number
NCT04752722
Ethics application status
Date submitted
4/02/2021
Date registered
12/02/2021

Titles & IDs
Public title
LEGEND Study: EG-70 in NMIBC Patients BCG-Unresponsive and High-Risk NMIBC Incompletely Treated With BCG or BCG-Naïve
Scientific title
A Phase 1/2 Study of EG-70 as an Intravesical Administration to Patients With BCG Unresponsive Non-Muscle Invasive Bladder Cancer (NMIBC) and High-Risk NMIBC Patients Who Are BCG Naïve or Received Incomplete BCG Treatment
Secondary ID [1] 0 0
EG-70-101
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Superficial Bladder Cancer 0 0
Non-muscle Invasive Bladder Cancer With Carcinoma in Situ 0 0
Condition category
Condition code
Cancer 0 0 0 0
Bladder - transitional cell cancer
Cancer 0 0 0 0
Cervical (cervix)
Cancer 0 0 0 0
Breast
Renal and Urogenital 0 0 0 0
Other renal and urogenital disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - EG-70 (phase 1)
Treatment: Drugs - EG-70 (phase 2)

Experimental: Phase 1 - Dose escalation phase

Experimental: 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


Treatment: Drugs: 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.

Treatment: Drugs: 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.

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Phase 1: Nature, incidence, relatedness, and severity of all AEs and SAEs according to the CTCAE v5.0.
Assessment method [1] 0 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.
Timepoint [1] 0 0
Approximately 2 years
Primary outcome [2] 0 0
Phase 2: Percentage of patients with cystoscopic CR at 48 weeks, based on exam, urine cytology and appropriate biopsies.
Assessment method [2] 0 0
Complete response rate will be measured by determining the number of patients without recurrence of high-grade disease.
Timepoint [2] 0 0
Approximately 48 weeks
Primary outcome [3] 0 0
Phase 2: Nature, incidence, relatedness, and severity of treatment emergent adverse events (as assessed by CTCAE v5.0)
Assessment method [3] 0 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.
Timepoint [3] 0 0
Approximately 3 years
Secondary outcome [1] 0 0
Phase 1: The number of patients who experience a DLT through the end of Cycle 1
Assessment method [1] 0 0
To identify the number of patients who experience a DLT through the end of Cycle 1
Timepoint [1] 0 0
Approximately 12 Weeks
Secondary outcome [2] 0 0
Phase 1: CR rate to EG-70 by cystoscopy at approximately 12 weeks.
Assessment method [2] 0 0
To evaluate preliminary efficacy of EG-70 by 12 weeks via cystoscopy
Timepoint [2] 0 0
Approximately 12 weeks
Secondary outcome [3] 0 0
Phase 2: Progression-free survival (PFS)
Assessment method [3] 0 0
To evaluate disease-free survival rate
Timepoint [3] 0 0
Approximately 3 years
Secondary outcome [4] 0 0
Phase 2: CR rate at 12, 24, 36, and 96 weeks
Assessment method [4] 0 0
To further evaluate CR at the efficacy analysis following each cycle.
Timepoint [4] 0 0
Approximately 12, 24, 36, and 96 weeks
Secondary outcome [5] 0 0
Phase 2: Duration of response of the responding patients
Assessment method [5] 0 0
Durability will be measured by determining the number of patients without recurrence of high-grade disease.
Timepoint [5] 0 0
Approximately 3 years
Secondary outcome [6] 0 0
Phase 2: Quality of Life Assessment
Assessment method [6] 0 0
Health-related quality of life
Timepoint [6] 0 0
24 weeks

Eligibility
Key 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:
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.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
SA
Recruitment hospital [1] 0 0
Icon Cancer Center Windsor Gardens - Windsor Gardens
Recruitment postcode(s) [1] 0 0
5087 - Windsor Gardens
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
District of Columbia
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Georgia
Country [6] 0 0
United States of America
State/province [6] 0 0
Illinois
Country [7] 0 0
United States of America
State/province [7] 0 0
Kansas
Country [8] 0 0
United States of America
State/province [8] 0 0
Maryland
Country [9] 0 0
United States of America
State/province [9] 0 0
Massachusetts
Country [10] 0 0
United States of America
State/province [10] 0 0
Michigan
Country [11] 0 0
United States of America
State/province [11] 0 0
Minnesota
Country [12] 0 0
United States of America
State/province [12] 0 0
New Jersey
Country [13] 0 0
United States of America
State/province [13] 0 0
New York
Country [14] 0 0
United States of America
State/province [14] 0 0
North Carolina
Country [15] 0 0
United States of America
State/province [15] 0 0
Ohio
Country [16] 0 0
United States of America
State/province [16] 0 0
Oregon
Country [17] 0 0
United States of America
State/province [17] 0 0
South Carolina
Country [18] 0 0
United States of America
State/province [18] 0 0
Tennessee
Country [19] 0 0
United States of America
State/province [19] 0 0
Texas
Country [20] 0 0
United States of America
State/province [20] 0 0
Virginia
Country [21] 0 0
United States of America
State/province [21] 0 0
Wisconsin
Country [22] 0 0
Canada
State/province [22] 0 0
Nova Scotia
Country [23] 0 0
Canada
State/province [23] 0 0
Quebec
Country [24] 0 0
France
State/province [24] 0 0
Bordeaux
Country [25] 0 0
France
State/province [25] 0 0
Lille
Country [26] 0 0
Germany
State/province [26] 0 0
Duisburg
Country [27] 0 0
Spain
State/province [27] 0 0
Badalona
Country [28] 0 0
Spain
State/province [28] 0 0
Barcelona
Country [29] 0 0
Spain
State/province [29] 0 0
Madrid
Country [30] 0 0
Spain
State/province [30] 0 0
Santander
Country [31] 0 0
Taiwan
State/province [31] 0 0
Kaohsiung

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
enGene, Inc.
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Public notes

Contacts
Principal investigator
Name 0 0
Christine Tosone, Ms, RAC
Address 0 0
enGene, Inc.
Country 0 0
Phone 0 0
Email 0 0
Contact person for public queries
Name 0 0
enGene clinical trials
Address 0 0
Country 0 0
Phone 0 0
+18572991097
Email 0 0
clinicaltrials@engene.com
Contact person for scientific queries

Data sharing statement


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.