Study of Erdafitinib Intravesical Delivery System for Localized Bladder Cancer

NCT05316155 | Active Not Recruiting Phase 1 FDA Drug

• 4 other identifiers: CR10911542756493BLC10032021-004144-222023-506265-65-00
• Study Type: interventional
• Target: 235
• Locations: 8 countries
• Sites: 59

Brief Summary

The purpose of the study in Part 1 (dose escalation) and in Part 2 (dose expansion) is to determine the recommended Phase 2 dose(s) (RP2D\[s\]) and evaluate preliminary clinical efficacy. Part 3 (dose expansion) will confirm safety and preliminary clinical activity at the RP2D. Part 4 (RP2D expansion; MoonRISe-2) will assess the overall complete response (CR) in participants with intermediate-risk-non-muscle invasive bladder cancer (IR-NMIBC; means the cancer cells are only in the bladder's inner lining).

Conditions

Non-Muscle Invasive Bladder Neoplasms

Keywords

Non-Muscle Invasive Bladder Cancer (NMIBC); Muscle Invasive Bladder Cancer (MIBC)

Outcome Measures

Primary Outcomes (4)

• Parts 1 to 3: Number of Participants with Adverse Events (AEs)

  An adverse event (AE) is any untoward medical event that occurs in a participant administered an investigational product, and it does not necessarily indicate only events with clear causal relationship with the relevant investigational product.

  Up to approximately 7 years 4 months

• Parts 1 to 3: Number of Participants with AEs by Severity

  Severity will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 5.0. Severity scale ranges from Grade 1 (Mild) to Grade 5 (Death). Grade 1= Mild, Grade 2= Moderate, Grade 3= Severe, Grade 4= Life-threatening and Grade 5= Death related to adverse event.

  Up to approximately 7 years 4 months

• Part 1: Number of Participants with Dose-limiting Toxicity (DLT)

  Number of participants with DLT will be assessed. The DLTs are specific adverse events and are defined as any of the following: high grade non-hematologic toxicity, or hematologic toxicity.

  Up to 28 days

• Part 4: Overall Complete Response (CR) in Participants with Intermediate Risk-Non-Muscle Invasive Bladder Cancer (IR-NMIBC)

  Overall CR is defined as the negative cystoscopy or positive cystoscopy with centrally reviewed biopsy negative for malignancy.

  Up to approximately 7 years 4 months

Secondary Outcomes (16)

• Parts 1 to 3: Plasma Concentration of Erdafitinib

  Cohorts 1, 3 and 5: up to 6 months; Cohort 2 and 4: up to 8 weeks

• Parts 1 to 3: Urine Concentration of Erdafitinib

  Cohorts 1, 3 and 5: up to 6 months; Cohort 2 and 4: up to 8 weeks

• Parts 1 to 3: Cohorts 1 and 2: Recurrence-Free Survival (RFS)

  Up to approximately 7 years 4 months

• Parts 1 to 3: Cohort 3 and 5: Complete Response (CR) Rate

  At 3 months

• Parts 1 to 3: Cohort 3 and 5: Duration of CR

  Up to approximately 7 years 4 months

Study Arms (4)

Part 1: Dose Escalation

EXPERIMENTAL

Participants with recurrent, bacillus Calmette-Guerin (BCG)-experienced high risk papillary-only Non-Muscle-Invasive Bladder Cancer (NMIBC), refusing or ineligible for radical cystectomy or with recurrent, intermediate-risk NMIBC will receive Erdafitinib Intravesical Delivery System. The dose will be escalated to determine preliminary recommended phase 2 dose(s) (RP2D\[s\]) for Part 2.

Drug: Erdafitinib Intravesical Delivery System

Part 2: Dose Expansion

EXPERIMENTAL

Participants in each of 5 disease-specific NMIBC or MIBC cohorts may be enrolled at one or more dose levels that have been determined to be safe in Part 1.

Drug: Erdafitinib Intravesical Delivery System

Part 3: RP2D Dose Expansion

EXPERIMENTAL

Participants in 2 of the disease-specific NMIBC cohorts (cohorts 1 and 3) may be enrolled at RP2D to determine the safety, evaluate PK and preliminary clinical activity.

Drug: Erdafitinib Intravesical Delivery System

Part 4: Phase 2 Expansion

EXPERIMENTAL

Participants with recurrent IR-NMIBC will be enrolled in this part to further evaluate the safety, efficacy, and PK of the selected RP2D.

Drug: Erdafitinib Intravesical Delivery System

Interventions

Erdafitinib Intravesical Delivery System DRUG

Erdafitinib intravesical delivery system will be administered.

Also known as: JNJ-42756493

Part 1: Dose Escalation; Part 2: Dose Expansion; Part 3: RP2D Dose Expansion; Part 4: Phase 2 Expansion

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Parts 1-3:
• Muscle-invasive or recurrent, non-muscle-invasive urothelial carcinoma of the bladder
• For selected Cohorts: Activating tumor pan-fibroblast growth factor receptor (FGFR) mutation or fusion, as determined by local or central testing, approved by the sponsor prior to the start of study treatment. Local tissue-based results (if already existing) from next-generation sequencing (NGS) or polymerase chain reaction (PCR) tests performed in Clinical Laboratory Improvement Amendments (CLIA) -certified or equivalent laboratories, or results from commercially available PCR or NGS tests
• Cohorts 1 and 2: Bacillus Calmette-Guérin (BCG) experienced, or participants with no BCG experience because BCG was not available as a treatment option in the participant's location within the previous 2 years and is currently unavailable. Participants who received an abbreviated course of BCG due to toxicity are still eligible
• Cohort 1 only: Refuses or is not eligible for radical cystectomy (RC)
• Cohorts 2 and 4: Willing and eligible for RC
• Part 4:
• Have histologically confirmed diagnosis of recurrent Intermediate-risk-non-muscle invasive bladder cancer (IR-NMIBC) Ta LG tumors
• Must not have undergone tumor debulking or selective ablation of visible lesions; partial tumor biopsy to confirm diagnosis and provide tissue for biomarker testing is permitted as long as remaining tumor is at least 5 millimeter (mm) in size
• Must submit tissue and urine for FGFR testing
• Can have a prior or concurrent second malignancy which natural history or treatment is unlikely to interfere with any study endpoints of safety or the efficacy of the study treatment

You may not qualify if:

• Parts 1-3:
• Concurrent extra-vesical (that is, urethra, ureter, renal pelvis) transitional cell carcinoma of the urothelium
• Prior treatment with an pan-fibroblast growth factor receptor (FGFR) inhibitor
• Received pelvic radiotherapy \<=6 months prior to the planned start of study treatment. If received pelvic radiotherapy greater than (\>)6 months prior to the start of study treatment, there must be no cystoscopic evidence of radiation cystitis
• Presence of any bladder or urethral anatomic feature that in the opinion of the investigator may prevent the safe use of Erdafitinib intravesical delivery system
• Indwelling urinary catheter. Intermittent catheterization is acceptable
• Part 4:
• Histologically confirmed diagnosis of T1 NMIBC, HR NMIBC (HG/G2 or HG/G3 or CIS) or MIBC, locally advanced, non-resectable, or metastatic urothelial carcinoma at any time prior to enrollment. Previous high grade (HG) disease is accepted as long as diagnosis date is greater than or equal to (\>=5) years ago and there is documentation of low grade (LG) Ta thereafter
• Known allergies, hypersensitivity, or intolerance to any study component or its excipients
• Has a current diagnosis of newly diagnosed IR-NMIBC
• Received an investigational treatment for bladder cancer after Transurethral Resection of the Bladder Tumor (TURBT) for the current NMIBC diagnosis or within 4 weeks or the agent/therapy washout period, whichever is longer, before the planned first dose of study treatment, or is currently enrolled in an investigational study
• Evidence of current bladder perforation by cystoscopy or imaging

Sponsors & Collaborators

• Janssen Research & Development, LLC: lead

Study Sites (59)

University of Alabama at Birmingham - The Kirklin Clinic

Birmingham, Alabama, 35294, United States

Location

University of Southern California

Los Angeles, California, 90033, United States

Location

Urology Associates of Denver

Lone Tree, Colorado, 80124, United States

Location

Urological Research Network

Hialeah, Florida, 33016, United States

Location

Advanced Urology Institute

Largo, Florida, 33771, United States

Location

Advent Health Orlando

Orlando, Florida, 32804, United States

Location

Advanced Urology Institute 1

Oxford, Florida, 34484, United States

Location

H Lee Moffitt Cancer Center

Tampa, Florida, 33612, United States

Location

Northwestern University

Chicago, Illinois, 60611, United States

Location

Associated Urological Specialists

Chicago Ridge, Illinois, 60415, United States

Location

Urology of Indiana

Greenwood, Indiana, 46143, United States

Location

Urologic Specialists of Northwest Indiana

Merrillville, Indiana, 46410, United States

Location

University of Kentucky

Lexington, Kentucky, 40506, United States

Location

Southern Urology LLC

Lafayette, Louisiana, 70508, United States

Location

Greater Boston Urology

Plymouth, Massachusetts, 02360, United States

Location

Specialty Clinical Research of St Louis

St Louis, Missouri, 63141, United States

Location

Hackensack University Medical Center Urology

Hackensack, New Jersey, 07601, United States

Location

Associated Medical Professionals

Syracuse, New York, 13210, United States

Location

Levine Cancer Institute, Carolinas HealthCare System

Charlotte, North Carolina, 28204, United States

Location

Central Ohio Urology Group

Gahanna, Ohio, 43230, United States

Location

Carolina Urologic Research Center

Myrtle Beach, South Carolina, 29572, United States

Location

Low Country Urology Clinics

North Charleston, South Carolina, 29406, United States

Location

Urology Associates

Nashville, Tennessee, 37209, United States

Location

Urology Austin

Austin, Texas, 78745, United States

Location

Urology San Antonio Research

San Antonio, Texas, 78229, United States

Location

Vancouver Prostate Centre Diamond Health Care Centre

Vancouver, British Columbia, V5Z 1M9, Canada

Location

Nova Scotia Health Authority

Halifax, Nova Scotia, B3H 2Y9, Canada

Location

St Josephs Healthcare Hamilton

Hamilton, Ontario, L8N 4A6, Canada

Location

Princess Margaret Cancer Centre

Toronto, Ontario, M5G 2M9, Canada

Location

Universitatsklinikum Carl Gustav Carus Dresden

Dresden, 01307, Germany

Location

Urologicum Duisburg

Duisburg, 47169, Germany

Location

Universitatsklinikum Frankfurt

Frankfurt am Main, 60590, Germany

Location

Marien hospital Herne

Herne, 44625, Germany

Location

Urologie Neandertal Praxis Mettmann

Mettmann, 40822, Germany

Location

Universitatsklinikum Munster

Münster, 48149, Germany

Location

Universitaetsklinikum Ulm

Ulm, 89081, Germany

Location

Rambam Medical Center

Haifa, 3109601, Israel

Location

Carmel Medical Center

Haifa, 3436212, Israel

Location

Rabin Medical Center

Petah Tikva, 4941492, Israel

Location

Tel Aviv Sourasky Medical Center

Tel Aviv, 64239, Israel

Location

National Hospital Organization Kyushu Medical Center

Fukuoka, 810 8563, Japan

Location

Yamanashi Prefectural Central Hospital

Kofu, 400-8506, Japan

Location

Osaka General Medical Center

Osaka, 558-8558, Japan

Location

Toyama University Hospital

Toyama, 930 0194, Japan

Location

Radboud Umcn

Nijmegen, 6525 GA, Netherlands

Location

UMC Utrecht

Utrecht, 3584 CX, Netherlands

Location

National Cancer Center

Goyang-si, 10408, South Korea

Location

Chonnam National University Hospital

Gwangju, 61469, South Korea

Location

Seoul National University Hospital

Seoul, 03080, South Korea

Location

Severance Hospital Yonsei University Health System

Seoul, 03722, South Korea

Location

The Catholic University of Korea Seoul St Marys Hospital

Seoul, 06591, South Korea

Location

Fund. Puigvert

Barcelona, 08025, Spain

Location

Hosp Univ Vall D Hebron

Barcelona, 08035, Spain

Location

Hosp Clinic de Barcelona

Barcelona, 08036, Spain

Location

Hosp Reina Sofia

Córdoba, 14004, Spain

Location

Hosp Univ Fund Jimenez Diaz

Madrid, 28040, Spain

Location

Hosp. Univ. 12 de Octubre

Madrid, 28041, Spain

Location

Hosp. Univ. La Paz

Madrid, 28046, Spain

Location

Instituto Valenciano de Oncologia

Valencia, 46009, Spain

Location

MeSH Terms

Conditions

Non-Muscle Invasive Bladder Neoplasms

Interventions

erdafitinib

Condition Hierarchy (Ancestors)

Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms; Urinary Bladder Neoplasms; Urologic Neoplasms; Urogenital Neoplasms; Neoplasms by Site; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Urinary Bladder Diseases; Urologic Diseases; Male Urogenital Diseases

Study Officials

• Janssen Research & Development, LLC Clinical Trial

  Janssen Research & Development, LLC

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 30, 2022
• First Posted: April 7, 2022
• Study Start: April 11, 2022
• Primary Completion (Estimated): March 30, 2028
• Study Completion (Estimated): July 24, 2029
• Last Updated: July 31, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will share

Locations

IL (4); ES (8); KR (5); JP (4); CA (4); DE (7); US (25); NL (2)