Clinical trial NCT05704244: Safety and Efficacy of FE 999326 Administered Intravesically to Japanese Subjects with High-grade, BCG Unresponsive, Non-muscle Invasive Bladder Cancer (NMIBC)
Official title: 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)
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Bladder Cancer
Lead sponsor: Ferring Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2023-01-30
Enrollment: 25
Sex: ALL
Minimum age (years): 20
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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


Eligibility criteria: 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

Primary outcomes:
measure: 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
timeFrame: At Month 12 assessment
Secondary outcomes:
measure: 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
timeFrame: Up to 5 years
measure: 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
timeFrame: Up to 4 years
measure: 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)
timeFrame: Up to 5 years
measure: Incidence of cystectomy
timeFrame: Up to 4 years
measure: Time to cystectomy
timeFrame: Up to 4 years
measure: Overall survival
timeFrame: Up to 5 years
measure: Concentration of FE 999326-specific DNA levels in urine
timeFrame: 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)
measure: Type, incidence, relatedness and severity of treatment emergent adverse events
timeFrame: Up to 5 years
Other outcomes:
Locations:
city: Nagakute-shi
country: Japan
facility: Ferring Investigational Site
state: Aichi
city: Nagoya-shi
country: Japan
facility: Ferring Investigational Site
state: Aichi
city: Narita-shi
country: Japan
facility: Ferring Investigational Site
state: Chiba
city: Matsuyama-Shi
country: Japan
facility: Ferring Investigational Site
state: Ehime
city: Fukuoka-shi
country: Japan
facility: Ferring Investigational Site
state: Fukuoka
city: Hiroshima-shi
country: Japan
facility: Ferring Investigational Site
state: Hiroshima
city: Hakodate-shi
country: Japan
facility: Ferring Investigational Site
state: Hokkaido
city: Sapporo-shi
country: Japan
facility: Ferring Investigational Site
state: Hokkaido
city: Hitachi-shi
country: Japan
facility: Ferring Investigational Site
state: Ibaraki
city: Tsukuba-shi
country: Japan
facility: Ferring Investigational Site
state: Ibaraki
city: Kawasaki-shi
country: Japan
facility: Ferring Investigational Site
state: Kanagawa
city: Yokohama-shi
country: Japan
facility: Ferring Investigational Site
state: Kanagawa
city: Nankoku-shi
country: Japan
facility: Ferring Investigational Site
state: Kochi
city: Tsu-shi
country: Japan
facility: Ferring Investigational Site
state: Mie
city: Sendai-shi
country: Japan
facility: Ferring Investigational Site
state: Miyagi
city: Matsumoto-shi
country: Japan
facility: Ferring Investigational Site
state: Nagano
city: Kashihara-shi
country: Japan
facility: Ferring Investigational Site
state: Nara
city: Okayama-shi
country: Japan
facility: Ferring Investigational Site
state: Okayama
city: Takatsuki-shi
country: Japan
facility: Ferring Investigational Site
state: Osaka
city: Bunkyo-ku
country: Japan
facility: Ferring Investigational Site
state: Tokyo
city: Minato-ku
country: Japan
facility: Ferring Investigational Site
state: Tokyo
city: Mitaka-shi
country: Japan
facility: Ferring Investigational Site
state: Tokyo
city: Toyama-shi
country: Japan
facility: Ferring Investigational Site
state: Toyama
city: Wakayama-shi
country: Japan
facility: Ferring Investigational Site
state: Wakayama
Study officials:
affiliation: Ferring Pharmaceuticals
name: Global Clinical Compliance
role: STUDY_DIRECTOR
References: