107 Participants Needed

Erdafitinib vs Chemotherapy for Bladder Cancer

Recruiting at 188 trial locations
SC
Overseen ByStudy Contact
Age: 18+
Sex: Any
Trial Phase: Phase 2
Sponsor: Janssen Research & Development, LLC
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial
Breakthrough TherapyThis drug has been fast-tracked for approval by the FDA given its high promise
Approved in 2 JurisdictionsThis treatment is already approved in other countries

Trial Summary

What is the purpose of this trial?

This trial tests erdafitinib, a pill that targets proteins to stop cancer growth, in patients with a type of bladder cancer that has returned after standard treatment. The drug blocks signals that help cancer cells grow. Erdafitinib is approved for treating a specific type of advanced bladder cancer.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What data supports the effectiveness of the drug erdafitinib for bladder cancer?

Erdafitinib has been shown to be effective in treating advanced bladder cancer, particularly in patients with specific genetic changes (FGFR2 and FGFR3 alterations), by stopping or reducing tumor growth. It is approved for use in patients whose cancer has progressed after other treatments, like chemotherapy.12345

Is erdafitinib safe for humans?

Erdafitinib has been shown to be generally tolerable in patients with advanced bladder cancer, but common side effects include high phosphate levels in the blood and eye problems, which require regular monitoring.12367

How is the drug erdafitinib different from other treatments for bladder cancer?

Erdafitinib is unique because it is an oral drug that specifically targets and inhibits fibroblast growth factor receptors (FGFRs), which are often altered in certain bladder cancers. This makes it particularly effective for patients with FGFR3 or FGFR2 alterations, offering a targeted approach compared to traditional chemotherapy.12389

Research Team

JR

Janssen Research & Development, LLC Clinical Trial

Principal Investigator

Janssen Research & Development, LLC

Eligibility Criteria

This trial is for adults with high-risk non-muscle-invasive bladder cancer that has returned after BCG therapy. Participants must have specific FGFR mutations or fusions, be unable to undergo cystectomy, and have good performance status (ECOG 0-1). They need proper organ function and a negative pregnancy test for women of childbearing potential.

Inclusion Criteria

I am not undergoing or cannot undergo bladder removal surgery.
Must sign an informed consent form (ICF) (or their legally acceptable representative must sign) indicating that he or she understands the purpose of, and procedures required for, the study and is willing to participate in the study
I am fully active or can carry out light work.
See 5 more

Exclusion Criteria

I have no active cancers except possibly treated skin cancer, certain breast or prostate cancers.
My bladder cancer is a specific type, like small cell or squamous.
I have been treated with an FGFR inhibitor before.
See 2 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either erdafitinib or investigator's choice of intravesical chemotherapy

Up to 4 years
Cycle 1 Day 14, Cycle 2 Day 1 (each cycle is of 28 days)

Follow-up

Participants are monitored for recurrence-free survival and adverse events

Up to 4 years

Long-term extension

Participants may continue treatment with erdafitinib if they demonstrate recurrence

Treatment Details

Interventions

  • Erdafitinib
  • Gemcitabine
  • Mitomycin C
Trial OverviewThe study compares the effectiveness of Erdafitinib against standard intravesical chemotherapy drugs like Gemcitabine or Mitomycin C in preventing cancer recurrence. It targets patients whose bladder cancer harbors certain genetic changes and who didn't respond well to previous treatments.
Participant Groups
4Treatment groups
Experimental Treatment
Active Control
Group I: Cohort 3Experimental Treatment1 Intervention
Marker lesion study in intermediate-risk NMIBC presenting as papillary disease only. All enrolled participants will receive treatment with erdafitinib.
Group II: Cohort 2Experimental Treatment1 Intervention
Participants with high-risk, BCG- unresponsive NMIBC presenting as CIS with or without concurrent papillary tumor will receive treatment with erdafitinib.
Group III: Cohort 1: ErdafitinibExperimental Treatment1 Intervention
Participants with high-risk non-muscle-invasive bladder cancer (NMIBC) presenting as papillary tumor only (carcinoma in situ \[CIS\], absent), with disease recurrence after bacillus Calmette- Guerin (BCG) therapy will receive treatment with erdafitinib.
Group IV: Cohort 1: Investigators ChoiceActive Control2 Interventions
Participants with high-risk NMIBC presenting as papillary tumor only (CIS, absent), with disease recurrence after BCG therapy will receive the investigator's choice of either intravesical gemcitabine or intravesical mitomycin C (MMC) or hyperthermic MMC. Participants who are randomized to gemcitabine or MMC or hyperthermic MMC in Cohort 1 and demonstrate a recurrence via investigator disease assessment will have the opportunity to cross over to treatment with erdafitinib.

Erdafitinib is already approved in United States, European Union for the following indications:

🇺🇸
Approved in United States as Balversa for:
  • Locally advanced or metastatic urothelial carcinoma with susceptible FGFR3 genetic alterations
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Approved in European Union as Balversa for:
  • Locally advanced or metastatic urothelial carcinoma with susceptible FGFR3 genetic alterations

Find a Clinic Near You

Who Is Running the Clinical Trial?

Janssen Research & Development, LLC

Lead Sponsor

Trials
1,022
Recruited
6,408,000+
Giacomo Salvadore profile image

Giacomo Salvadore

Janssen Research & Development, LLC

Chief Medical Officer since 2023

MD from the University of Rome, Tor Vergata

Ricardo Attar profile image

Ricardo Attar

Janssen Research & Development, LLC

Chief Executive Officer since 2008

PhD in Molecular Biology from the University of Buenos Aires

Findings from Research

Erdafitinib, a tyrosine kinase inhibitor for advanced urothelial carcinoma, was studied using a pharmacokinetic model in mice and rats to determine effective dosing for phase 1 trials, showing that doses above 4 mg/day resulted in significant tumor growth inhibition.
The modeling predicted that efficacious unbound concentrations of erdafitinib were identified, aligning with early clinical activity observed at a dose of 6 mg in the first-in-human trial, demonstrating the potential of translational modeling in optimizing drug dosing.
A translational model-based approach to inform the choice of the dose in phase 1 oncology trials: the case study of erdafitinib.Tosca, EM., Terranova, N., Stuyckens, K., et al.[2022]
Erdafitinib is a newly approved pan-FGFR inhibitor specifically for treating locally advanced or metastatic urothelial carcinoma associated with FGFR3 or FGFR2 mutations.
The drug is also being explored for its potential effectiveness against various other cancers, including cholangiocarcinoma and non-small cell lung cancer, indicating its broad therapeutic applications.
Erdafitinib: First Global Approval.Markham, A.[2020]
In a long-term follow-up of the BLC2001 study involving 101 patients with advanced urothelial carcinoma, erdafitinib demonstrated a 40% objective response rate, indicating its efficacy in this patient population with specific FGFR alterations.
The safety profile of erdafitinib remained consistent over time, with 71% of patients experiencing grade 3-4 adverse events, but no new safety concerns were identified, suggesting it is a manageable treatment option.
Efficacy and safety of erdafitinib in patients with locally advanced or metastatic urothelial carcinoma: long-term follow-up of a phase 2 study.Siefker-Radtke, AO., Necchi, A., Park, SH., et al.[2022]

References

A translational model-based approach to inform the choice of the dose in phase 1 oncology trials: the case study of erdafitinib. [2022]
Erdafitinib: First Global Approval. [2020]
Efficacy and safety of erdafitinib in patients with locally advanced or metastatic urothelial carcinoma: long-term follow-up of a phase 2 study. [2022]
Erdafitinib or Chemotherapy in Advanced or Metastatic Urothelial Carcinoma. [2023]
Plain language summary of erdafitinib in locally advanced or metastatic urothelial carcinoma: a phase 2 study with long-term follow-up. [2023]
Erdafitinib-Induced Secondary Maculopathy. [2023]
Clinical Evidence and Selecting Patients for Treatment with Erdafitinib in Advanced Urothelial Carcinoma. [2022]
Erdafitinib in BCG-treated high-risk non-muscle invasive bladder cancer. [2023]
Erdafitinib versus pembrolizumab in pretreated patients with advanced or metastatic urothelial cancer with select FGFR alterations: cohort 2 of the randomized phase III THOR trial. [2023]