LEGEND Study: EG-70 in NMIBC Patients BCG-Unresponsive and High-Risk NMIBC Incompletely Treated With BCG or BCG-Naïve
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|ClinicalTrials.gov Identifier: NCT04752722|
Recruitment Status : Recruiting
First Posted : February 12, 2021
Last Update Posted : October 3, 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.
|Condition or disease||Intervention/treatment||Phase|
|Superficial Bladder Cancer||Drug: EG-70||Phase 1 Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||222 participants|
|Intervention Model:||Sequential Assignment|
|Masking:||None (Open Label)|
|Official 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|
|Actual Study Start Date :||April 22, 2021|
|Estimated Primary Completion Date :||June 2025|
|Estimated Study Completion Date :||May 2027|
Experimental: Phase 1
Dose escalation phase
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.
Other Name: Phase 1
Experimental: Phase 2
Cohort 1: Recommended Phase 2 dose (RP2D) with eligible BCG-unresponsive NMIBC patients, up to 4 cycles of treatment with EG-70
Cohort 2: RP2D with eligible high-risk NMIBC patients who have been incompletely treated with BCG or are BCG-naïve
Cohort 1 and Cohort 2: Patients will receive 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.
Other Name: Phase 2
- Phase 1: Nature, incidence, relatedness, and severity of all AEs and SAEs according to the CTCAE v5.0. [ Time Frame: Approximately 2 years ]The type, incidence, relatedness and severity of treatment emergent adverse events of EG-70 as assessed by NCI-CTCAE V5.0 will be monitored.
- Phase 2: Percentage of patients with cystoscopic CR at 48 weeks, based on exam, urine cytology and appropriate biopsies. [ Time Frame: Approximately 48 weeks ]Complete response rate will be measured by determining the number of patients without recurrence of high-grade disease.
- Phase 2: Nature, incidence, relatedness, and severity of treatment emergent adverse events (as assessed by CTCAE v5.0) [ Time Frame: Approximately 3 years ]The type, incidence, relatedness and severity of treatment emergent adverse events of EG-70 as assessed by NCI-CTCAE V5.0 will be monitored.
- Phase 1: The number of patients who experience a DLT through the end of Cycle 1 [ Time Frame: Approximately 12 Weeks ]To identify the number of patients who experience a DLT through the end of Cycle 1
- Phase 1: CR rate to EG-70 by cystoscopy at approximately 12 weeks. [ Time Frame: Approximately 12 weeks ]To evaluate preliminary efficacy of EG-70 by 12 weeks via cystoscopy
- Phase 2: Progression-free survival (PFS) [ Time Frame: Approximately 3 years ]To evaluate disease-free survival rate
- Phase 2: CR rate at 12, 24, 36, and 96 weeks [ Time Frame: Approximately 12, 24, 36, and 96 weeks ]To further evaluate CR at the efficacy analysis following each cycle.
- Phase 2: Duration of response of the responding patients [ Time Frame: Approximately 3 years ]Durability will be measured by determining the number of patients without recurrence of high-grade disease.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04752722
|Contact: Chris Tosoneemail@example.com|
|Study Director:||Christine Tosone, Ms||enGene, Inc.|