Safety and Efficacy of FE 999326 Administered Intravesically to Japanese Subjects With High-grade, BCG Unresponsive, Non-muscle Invasive Bladder Cancer (NMIBC)

A Phase 3, Open Label Trial to Evaluate the Safety and Efficacy of FE 999326 Administered Intravesically to Japanese Subjects With High-grade, BCG Unresponsive, Non-muscle Invasive Bladder Cancer (NMIBC)

To evaluate the safety and efficacy of FE 999326 in Japanese subjects with high-grade, BCG unresponsive NMIBC.

Study Overview

• Status: Recruiting
• Conditions: Bladder Cancer
• Intervention / Treatment: Drug: FE 999326

• Study Type: Interventional
• Enrollment (Estimated): 24
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Global Clinical Compliance; Phone Number: +1 833-548-1402 (US/Canada); Email: DK0-Disclosure@ferring.com
• Study Contact Backup: Name: Global Clinical Compliance; Phone Number: +1 862-286-5200 (outside US); Email: DK0-Disclosure@ferring.com

Study Locations

• Japan
  • Aichi
    • Nagakute-shi, Aichi, Japan
      • Recruiting
      • Ferring Investigational Site
    • Nagoya-shi, Aichi, Japan
      • Recruiting
      • Ferring Investigational Site
  • Chiba
    • Narita-shi, Chiba, Japan
      • Recruiting
      • Ferring Investigational Site
  • Ehime
    • Matsuyama-Shi, Ehime, Japan
      • Recruiting
      • Ferring Investigational Site
  • Fukuoka
    • Fukuoka-shi, Fukuoka, Japan
      • Recruiting
      • Ferring Investigational Site
  • Hiroshima
    • Hiroshima-shi, Hiroshima, Japan
      • Recruiting
      • Ferring Investigational Site
  • Hokkaido
    • Hakodate-shi, Hokkaido, Japan
      • Recruiting
      • Ferring Investigational Site
    • Sapporo-shi, Hokkaido, Japan
      • Recruiting
      • Ferring Investigational Site
  • Ibaraki
    • Hitachi-shi, Ibaraki, Japan
      • Recruiting
      • Ferring Investigational Site
    • Tsukuba-shi, Ibaraki, Japan
      • Recruiting
      • Ferring Investigational Site
  • Kanagawa
    • Kawasaki-shi, Kanagawa, Japan
      • Recruiting
      • Ferring Investigational Site
    • Yokohama-shi, Kanagawa, Japan
      • Recruiting
      • Ferring Investigational Site
  • Kochi
    • Nankoku-shi, Kochi, Japan
      • Recruiting
      • Ferring Investigational Site
  • Mie
    • Tsu-shi, Mie, Japan
      • Recruiting
      • Ferring Investigational Site
  • Miyagi
    • Sendai-shi, Miyagi, Japan
      • Recruiting
      • Ferring Investigational Site
  • Nagano
    • Matsumoto-shi, Nagano, Japan
      • Recruiting
      • Ferring Investigational Site
  • Nara
    • Kashihara-shi, Nara, Japan
      • Recruiting
      • Ferring Investigational Site
  • Okayama
    • Okayama-shi, Okayama, Japan
      • Recruiting
      • Ferring Investigational Site
  • Osaka
    • Takatsuki-shi, Osaka, Japan
      • Recruiting
      • Ferring Investigational Site
  • Tokyo
    • Bunkyo-ku, Tokyo, Japan
      • Recruiting
      • Ferring Investigational Site
    • Minato-ku, Tokyo, Japan
      • Recruiting
      • Ferring Investigational Site
    • Mitaka-shi, Tokyo, Japan
      • Recruiting
      • Ferring Investigational Site
  • Toyama
    • Toyama-shi, Toyama, Japan
      • Recruiting
      • Ferring Investigational Site
  • Wakayama
    • Wakayama-shi, Wakayama, Japan
      • Recruiting
      • Ferring Investigational Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 20 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Have at entry, confirmed by a pathology report:

  1. Carcinoma in situ (CIS) only
  2. Ta/T1 high-grade disease with concomitant CIS or
  3. Ta/T1 high-grade disease without concomitant CIS
• Subjects who did not respond to BCG treatment and have a persistent high-grade recurrence within 12 months after BCG was initiated, and those who despite an initial complete response to BCG, relapse with CIS within 12 months of their last intravesical treatment with BCG or relapse with high-grade Ta/T1 NMIBC within 6 months of their last intravesical treatment with BCG.
• Life expectancy >2 years, in the opinion of the investigator
• Eastern Cooperative Oncology Group (ECOG) status 2 or less
• Absence of concomitant upper tract urothelial carcinoma or urothelial carcinoma within the prostatic urethra
• Subjects with prostate cancer on active surveillance at low risk for progression, defined as prostate-specific antigen (PSA) <10 ng/dL

Exclusion Criteria:

• Current or previous evidence of muscle invasive (muscularis propria) or metastatic disease presented at the screening visit.
• Current systemic therapy for bladder cancer
• Current or prior investigational treatment for BCG unresponsive NMIBC or any other investigational drug within 1 month prior to screening
• Current or prior pelvic external beam radiotherapy within 5 years of entry
• Use of other adenovirus vector medications, including COVID-19 vaccines, within 2 weeks before and after instillation
• History of malignancy of other organ system within past 5 years, except treated basal cell carcinoma or squamous cell carcinoma of the skin and ≤pT2 upper tract urothelial carcinoma at least 24 months after nephroureterectomy.
• Subjects who cannot hold instillation for 1 hour
• Subjects who cannot tolerate intravesical dosing or intravesical surgical manipulation

• Intravesical therapy within 8 weeks prior to beginning trial treatment, with the exception of:

  • Cytotoxic agents when administered as a single instillation immediately following a TURBT procedure
  • Previous intravesical BCG therapy
• Systemic immunosuppressive therapy within 3 months prior to screening

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: FE 999326	Drug: FE 999326; 75 mL FE 999326 (3 x 10^11 vp/mL) of sterile suspension instilled once every 3 months into the bladder via a urinary catheter; Other Names: nadofaragene firadenovec

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Whether or not a subject with CIS (with or without concomitant high-grade Ta or T1 papillary disease) responds to treatment, defined as complete response at any time after first administration of FE 999326	A subject with CIS (with or without concomitant high-grade Ta or T1 papillary disease) has achieved a complete response at an efficacy assessment if urine cytology is negative and there are no lesions on cystoscopy. If biopsy of the bladder is performed, this must be negative.	At Month 12 assessment

Secondary Outcome Measures

Outcome Measure	Time Frame
Overall survival	Up to 5 years
Duration of complete response in subjects with CIS (with or without concomitant high-grade Ta or T1 papillary disease) who show a complete response at any time after first administration of FE 999326	Up to 5 years
Whether or not a subject with high-grade Ta or T1 papillary disease (without concomitant CIS) responds to treatment, defined as absence of recurrence of high-grade disease	Up to 4 years
Duration of event-free survival in subjects with high-grade Ta or T1 papillary disease (without concomitant CIS) and in subjects with CIS (with or without papillary disease)	Up to 5 years
Incidence of cystectomy	Up to 4 years
Time to cystectomy	Up to 4 years
Concentration of FE 999326-specific DNA levels in urine	Up to day 1 of month 4 after start of treatment; and 3, 6, 9 and 12 months after last dose (up to 5 years after start of treatment)
Type, incidence, relatedness and severity of treatment emergent adverse events	Up to 5 years

Collaborators and Investigators

• Sponsor: Ferring Pharmaceuticals
• Investigators: Study Director: Global Clinical Compliance, Ferring Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): December 21, 2022
• Primary Completion (Estimated): December 27, 2024
• Study Completion (Estimated): December 27, 2028

Study Registration Dates

• First Submitted: December 21, 2022
• First Submitted That Met QC Criteria: January 19, 2023
• First Posted (Actual): January 30, 2023

Study Record Updates

• Last Update Posted (Actual): March 22, 2024
• Last Update Submitted That Met QC Criteria: March 21, 2024
• Last Verified: October 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Urologic Neoplasms; Urogenital Neoplasms; Neoplasms by Site; Urologic Diseases; Carcinoma; Neoplasms, Glandular and Epithelial; Urinary Bladder Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Urinary Bladder Neoplasms; Non-Muscle Invasive Bladder Neoplasms
• Other Study ID Numbers: 000381

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No