Neoplasms

All Conditions

Urinary Tract, Sexual Organs, and Pregnancy Conditions

Carcinoma

Urinary Bladder Neoplasms

Carcinoma in Situ

Non-Muscle Invasive Bladder Neoplasms

Neoplasms, Glandular and Epithelial

Neoplasms by Histologic Type

Urologic Neoplasms

Urogenital Neoplasms

Urogenital Diseases

Female Urogenital Diseases

Pregnancy Complications

Female Urogenital Diseases and Pregnancy Complications

Urinary Bladder Diseases

Urologic Diseases

Male Urogenital Diseases

All Drugs and Chemicals

Hyaluronic Acid

BCG Vaccine

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

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 1)

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.

EG-70 (phase 2)

enGene clinical trials

Chris Tosone

Susan Kalota

Edward Uchio

Anne Schuckman

Terry Williams

Alanna Gavriushina

Katayune Golshan

Michael Whalen

Kethandapatti Balaji

Shreyas Joshi

Faith Rahman

Laura Mitchell

Rian Dickstein

Johar Raza, MD

Conrad Tobert

Marissa Twedt

Joseph Zabell

Gordon Brown

John Sfakianos

Marc Bjurlin

Mark Jalkut

Mohammed Kamel

Kara Lasorella

J Liu

Neal Shore

Jose Santoyo

Yair Lotan

Taliah Muhammad

Ashish Kamat, MD

Scott Johnson

Christine Tosone, Ms, RAC

This study will evaluate the safety and efficacy of intravesical administration of 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 patients with NMIBC with Cis who are BCG-naïve or inadequately treated.

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.

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

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

The type, incidence, relatedness and severity of treatment emergent adverse events of EG-70 as assessed by NCI-CTCAE V5.0 will be monitored.

Complete response rate will be measured by determining the number of patients without recurrence of high-grade disease.

To identify the number of patients who experience a DLT through the end of Cycle 1

To evaluate preliminary efficacy of EG-70 by 12 weeks via cystoscopy

To further evaluate CR at the efficacy analysis following each cycle.

Durability will be measured by determining the number of patients without recurrence of high-grade disease.

enGene, Inc.

Rare Diseases

Carcinoma, Transitional Cell

Transitional Cell Carcinoma

Antineoplastic Agents

Vaccines

Durvalumab

Antineoplastic Agents, Immunological

Durvalumab: Fixed dose level IV infusion every 4 weeks for 13 study treatment cycles/infusions over 12 months/1 year. Cystoscopy with biopsy will be performed every 3 months to monitor the treatment response during this one year of treatment phase. It will be performed every 6 months during year 2 of the surveillance phase.

Durvalumab will be given every 4 weeks at 1500 mg/kg IV for total of 12 months/13 doses.

Cystoscopy with biopsy and transurethral resection of the bladder tumor (TURBT) (if indicated) will be performed at baseline, month 3, 6, 9, 12, 18, and 24. The month 6 and 24 cystoscopy will be done in the operating room with mapping biopsy. Rest of the cystoscopic exam with biopsy will be performed in the out-patient office setting and if clinically indicated will be repeated in the operating room. Participants will be off study if any of the biopsies document muscle invasive (T2 or above) urothelial carcinoma. Participants will also be off study if their month 6, 9, 12, 18 biopsies show persistent (month 6) or recurrent CIS or invasive (T1 or above) urothelial carcinoma. Otherwise, participants will remain on study until after the month 24 mapping biopsy.

Cystoscopy with Biopsy

Jingsong Zhang, M.D., Ph.D.

The purpose of this study is to test if an experimental drug called Durvalumab (Medi4736) given by intravenous (IV) infusion is effective in treating carcinoma in situ (CIS) of the bladder that no longer responds to Bacillus Calmette-Guérin (BCG) and to collect information on the safety of these drugs and whether they cause any side effects.

Phase 2 Durvalumab (Medi4736) for Bacillus Calmette-Guérin (BCG) Refactory Urothelial Carcinoma in Situ of the Bladder

A Phase 2 Study of Check Point Inhibitor, Durvalumab (Medi4736) for Bacillus Calmette-Guérin (BCG) Refractory Urothelial Carcinoma in Situ (CIS) of the Bladder

Complete Response Rate at month six based on the week 26 mapping biopsy in BCG refractory CIS urothelial bladder cancer. The absence of CIS of bladder on the mapping biopsies after pathological review would be considered complete response to treatment.

Complete Response Rate at month 24 based on the week 104 mapping biopsy in BCG refractory CIS urothelial bladder cancer. The absence of CIS of bladder on the mapping biopsies after pathological review would be considered complete response to treatment.

AstraZeneca

H. Lee Moffitt Cancer Center and Research Institute

Durvalumab: Fixed dose level IV infusion every 4 weeks for 13 study treatment cycles/infusions over 12 months/1 year. Cystoscopy with biopsy will be performed every 3 months to monitor the treatment response during this one year of treatment phase. It will be performed every 6 months during year 2 of the surveillance phase.

Durvalumab: Durvalumab will be given every 4 weeks at 1500 mg/kg IV for total of 12 months/13 doses.

Cystoscopy with Biopsy: Cystoscopy with biopsy and transurethral resection of the bladder tumor (TURBT) (if indicated) will be performed at baseline, month 3, 6, 9, 12, 18, and 24. The month 6 and 24 cystoscopy will be done in the operating room with mapping biopsy. Rest of the cystoscopic exam with biopsy will be performed in the out-patient office setting and if clinically indicated will be repeated in the operating room. Participants will be off study if any of the biopsies document muscle invasive (T2 or above) urothelial carcinoma. Participants will also be off study if their month 6, 9, 12, 18 biopsies show persistent (month 6) or recurrent CIS or invasive (T1 or above) urothelial carcinoma. Otherwise, participants will remain on study until after the month 24 mapping biopsy.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
LEGEND Study: EG-70 in NMIBC Patients BCG-Unresponsive and High-Risk NMIBC Incompletely Treated With BCG or BCG-Naïve	NCT04752722	Superficial Bla... Non-muscle Inva...	EG-70 (phase 1) EG-70 (phase 2)	18 Years -	enGene, Inc.
LEGEND Study: EG-70 in NMIBC Patients BCG-Unresponsive and High-Risk NMIBC Incompletely Treated With BCG or BCG-Naïve	NCT04752722	Superficial Bla... Non-muscle Inva...	EG-70 (phase 1) EG-70 (phase 2)	18 Years -	enGene, Inc.
Phase 2 Durvalumab (Medi4736) for Bacillus Calmette-Guérin (BCG) Refactory Urothelial Carcinoma in Situ of the Bladder	NCT02901548	Carcinoma in Si... Bladder Cancer	Durvalumab Cystoscopy with...	18 Years -	H. Lee Moffitt Cancer Center and Research Institute
LEGEND Study: EG-70 in NMIBC Patients BCG-Unresponsive and High-Risk NMIBC Incompletely Treated With BCG or BCG-Naïve	NCT04752722	Superficial Bla... Non-muscle Inva...	EG-70 (phase 1) EG-70 (phase 2)	18 Years -	enGene, Inc.

Spots Global Cancer Trial Database for carcinoma in situ (cis)