Study of erdafitinib versus chemotherapy (gemcitabine or mitomycin) for patients with high-risk non-muscle-invasive bladder cancer with FGFR mutations who failed BCG therapy

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What is this study about?

This study focuses on Non-Muscle-Invasive Bladder Cancer (NMIBC) that has specific genetic changes called FGFR mutations or fusions. The cancer being studied has returned after previous treatment with Bacillus Calmette-Guérin (BCG). The study compares two different treatments: an oral medication called erdafitinib (also known as JNJ-42756493) and standard chemotherapy medications (gemcitabine or mitomycin) that are placed directly into the bladder.

The main purpose of this research is to determine how well erdafitinib works compared to standard chemotherapy in preventing cancer from returning in patients with high-risk bladder cancer. The study will track how long patients remain free from cancer recurrence after starting treatment.

During the study, patients will be randomly assigned to receive either erdafitinib tablets taken by mouth, or chemotherapy treatment delivered directly into the bladder. The erdafitinib group will take the medication daily for up to 24 months. Patients receiving bladder chemotherapy will get treatment over a period of up to 7 days. The study will continue until early 2025.

1 Initial assessment and medication group assignment

Your bladder cancer type and genetic markers will be checked to confirm eligibility for the study

You will be randomly assigned to receive either erdafitinib tablets or intravesical chemotherapy (medication delivered directly into the bladder)

2 Treatment pathway A – Erdafitinib tablets

If assigned to erdafitinib, you will take tablets by mouth daily

Regular blood tests will monitor your liver and kidney function

Your phosphate levels will be checked regularly

3 Treatment pathway B – Intravesical chemotherapy

If assigned to intravesical therapy, you will receive either gemcitabine or mitomycin

The medication will be delivered directly into your bladder through a small tube

Treatment sessions will be scheduled according to your specific care plan

4 Regular monitoring

Regular bladder examinations (cystoscopy) will check for cancer recurrence

Blood tests will monitor your overall health

Your doctor will assess how well the treatment is working

5 Study completion

The study continues until February 2025

Your participation may end earlier if your cancer returns or if you experience significant side effects

Final assessment will evaluate how long you remained free of cancer recurrence

Who Can Join the Study?

Must be at least 18 years of age or meet the legal age of consent
Must have confirmed recurring bladder cancer that has not invaded the muscle layer (called non-muscle-invasive bladder cancer)
Must have specific genetic changes (mutations or fusions) in the FGFR gene confirmed by testing
Must have previously received treatment with BCG therapy (a type of bladder cancer treatment) and either:
– Had cancer return within 12 months after treatment, or
– Had cancer return within 6 months after treatment, or
– Had signs of cancer after the first treatment course
Must either refuse or not be eligible for surgery to remove the bladder (called cystectomy)
Must be able to perform daily activities with minimal or no assistance (ECOG performance status of 0 or 1)
Must have adequate blood test results showing proper function of:
– Bone marrow
– Liver
– Kidneys
Must sign an informed consent form showing understanding of the study
For women who can become pregnant:
– Must have a negative pregnancy test within 7 days before starting the study
– Must use effective birth control methods
For men with partners who can become pregnant:
– Must use effective birth control methods during the study

Who Cannot Join the Study?

Age below 18 years old
Previous treatment with FGFR inhibitors (medications that block specific proteins involved in cancer growth)
Known allergies or hypersensitivity to the study medication or its components
Presence of muscle-invasive bladder cancer (cancer that has grown into the muscle wall of the bladder)
Significant heart problems, including uncontrolled high blood pressure or recent heart attack
Severe kidney or liver problems
Active or untreated infections
Pregnancy or breastfeeding
Participation in another clinical trial within the past 30 days
Mental conditions that could interfere with following study procedures
History of other cancers in the past 2 years (except for successfully treated non-melanoma skin cancer)
Absence of FGFR mutations or fusions (specific genetic changes in the tumor)
Unable to provide informed consent
Medical conditions that could interfere with study procedures or interpretation of results

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Centre Hospitalier Universitaire De Bordeaux	Bordeaux	France

Other Sites

Site Name	City	Country
Pratia S.A.	Skorzewo	Poland
Centre Hospitalier Universitaire De Rennes	Rennes	France
St. Elisabeth Gruppe GmbH Katholische Kliniken Rhein-Ruhr	Herne	Germany

Trial status

Country	Status	Recruitment Start
France	Not recruiting	18.05.2020
Germany	Not recruiting	18.05.2020
Poland	Not recruiting	18.05.2020

Trial locations

Investigated drugs:

Erdafitinib is a targeted therapy medication that works by blocking specific proteins called FGFR (fibroblast growth factor receptors) in cancer cells. It is used to treat bladder cancer that has specific genetic mutations or fusions in the FGFR genes. This medication is taken orally and works by helping to stop or slow down the growth of cancer cells.

Bacillus Calmette-Guérin (BCG) is a type of immunotherapy used to treat bladder cancer. It is made from a weakened form of bacteria and is delivered directly into the bladder through a catheter. BCG works by stimulating the immune system to fight cancer cells in the bladder.

Intravesical chemotherapy refers to chemotherapy drugs that are placed directly into the bladder through a catheter. This type of treatment delivers medication directly to the tumor site while minimizing exposure to the rest of the body, which helps reduce side effects.

Investigated diseases:

Bladder transitional cell carcinoma

Non-Muscle-Invasive Bladder Cancer (NMIBC) – A type of bladder cancer that affects the inner lining of the bladder without invading the muscle layer. The cancer cells are found only in the bladder’s inner lining and connective tissue layer. NMIBC can occur with genetic changes called FGFR mutations or fusions, which affect how cells grow and divide. The condition can be classified into different risk levels (high-risk and intermediate-risk) based on specific characteristics of the tumor. The cancer may recur in the bladder after initial treatment.

FGFR-Mutated Bladder Cancer – A form of bladder cancer characterized by specific genetic alterations in the Fibroblast Growth Factor Receptor (FGFR) genes. These mutations affect the way cells receive growth signals and can influence how the cancer develops. The presence of FGFR mutations or fusions represents a distinct molecular subtype of bladder cancer. This genetic profile can occur in both high-risk and intermediate-risk cases of bladder cancer.

Trial ID:

2023-510306-40-00

Protocol code:

42756493BLC2003

Trial Phase:

Therapeutic exploratory (Phase II)

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Study of erdafitinib versus chemotherapy (gemcitabine or mitomycin) for patients with high-risk non-muscle-invasive bladder cancer with FGFR mutations who failed BCG therapy

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?