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An Efficacy and Safety Study of Erdafitinib (JNJ-42756493) in Participants With Urothelial Cancer

NCT ID: NCT02365597

Last Updated: 2025-11-12

Study Results

Results available

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Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

239 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-04-22

Study Completion Date

2026-12-31

Brief Summary

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The purpose of this study is to evaluate the objective response rate (complete response \[CR\]+ partial response \[PR\]) of the selected dose regimen in participants with metastatic or surgically unresectable urothelial cancers that harbor specific FGFR genomic alterations.

Detailed Description

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This is a multicenter, open-label study (participants will know the identity of study drugs administered) to evaluate the efficacy and safety of erdafitinib in participants with urothelial cancer. The study comprises a 30-days Screening Phase, a Treatment Phase comprised of 28-day treatment cycles that will continue until disease progression or unacceptable toxicity occurs in a long-term extension (LTE) phase, and a post-treatment Follow-up Phase that will extend from the End-of-Treatment Visit until the participant has died, withdraws consent, is lost to follow-up, or the end of the study, whichever comes first. The end of study is defined as the date when all participants have completed the study treatment (Regimens 1 to 3) and all participants enrolled under the drug-drug interaction (DDI) substudy are no longer receiving treatment with erdafitinib. The purpose of DDI sub-study is to evaluate the interaction of repeated doses of erdafitinib with a sensitive cytochrome 450 (CYP) 3A substrate (midazolam) and with an organic cation transporter 2 (OCT2) probe substrate (metformin). Safety will be monitored throughout the study.

Conditions

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Urothelial Cancer

Keywords

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Urothelial Cancer JNJ-42756493 Erdafitinib Tyrosine Kinase Inhibitor

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Erdafitinib (8 milligram)

Prior to interim analysis 1 (IA1), there were 2 treatment regimens: Regimen 1 (10 milligram \[mg\] once daily, 7 days on/7 days off); and Regimen 2 (6 mg once daily for 28 days). Following IA1, Regimen 1 is closed for further enrollment and starting dose of Regimen 2 is increased to 8 mg once daily for 28 days on a 28-day cycle (referred to as Regimen 3). Participants who enrolled in DDI substudy will receive pretreatment with single doses of midazolam (Day -2) and metformin (Day -1). Participants enrolled in DDI substudy will receive 8 mg erdafitinib treatment from Day 1 to Day 15, single doses of midazolam 2.5 mg (Day 13) and metformin 1000 mg (Day 14) and erdafitinib treatment will continued until disease progression. Participants who completed the DDI substudy and continue to benefit from erdafitinib treatment, will continue to receive erdafitinib in long-term extension (LTE) phase.

Group Type EXPERIMENTAL

Erdafitinib

Intervention Type DRUG

8 mg orally once daily for 28 days on a 28 day cycle.

Midazolam

Intervention Type DRUG

Participants who enrolled in DDI substudy will receive pretreatment with single dose of midazolam on Day -2 and single dose of midazolam on Day 13.

Metformin

Intervention Type DRUG

Participants who enrolled in DDI substudy will receive pretreatment with single dose of metformin on Day -1 and single dose of metformin on Day 14.

Interventions

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Erdafitinib

8 mg orally once daily for 28 days on a 28 day cycle.

Intervention Type DRUG

Midazolam

Participants who enrolled in DDI substudy will receive pretreatment with single dose of midazolam on Day -2 and single dose of midazolam on Day 13.

Intervention Type DRUG

Metformin

Participants who enrolled in DDI substudy will receive pretreatment with single dose of metformin on Day -1 and single dose of metformin on Day 14.

Intervention Type DRUG

Other Intervention Names

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JNJ-42756493

Eligibility Criteria

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Inclusion Criteria

* Must have histologic demonstration of metastatic or surgically unresectable urothelial cancer. Minor components of variant histology such as glandular or squamous differentiation, or evolution to more aggressive phenotypes such as sarcomatoid or micropapillary change are acceptable
* Must have measurable disease according to the Response Evaluation Criteria in Solid Tumors (RECIST, version 1.1) at baseline
* Must have an Eastern Cooperative Oncology Group (ECOG) performance status score 0, 1, or 2
* Must have adequate bone marrow, liver, and renal function as described in protocol
* Negative pregnancy test (urine or serum beta human chorionic gonadotropin \[b-hCG\]) at Screening for women of child bearing potential who are sexually active
* Must have shown disease progression according to RECIST, version 1.1, following prior chemotherapy for metastatic or surgically unresectable urothelial cancer. Participants who received neoadjuvant or adjuvant chemotherapy and showed disease recurrence or progression according to RECIST, version 1.1, within 12 months of the last dose are considered to have received chemotherapy in the metastatic setting. These participants will be referred to as chemo-refractory participants. (Participants who have shown disease progression according to RECIST, version 1.1 following prior treatment with anti-Programmed death-ligand 1 (anti PDL1/PD1) antibodies are also eligible) For DDI substudy
* Disease progression following prior chemotherapy for metastatic or surgically unresectable urothelial cancer. Participants who received neoadjuvant or adjuvant chemotherapy and showed disease recurrence or progression within 12 months of the last dose are considered to have received chemotherapy in the metastatic setting

Exclusion Criteria

* Received chemotherapy, targeted therapies, definitive radiotherapy, or treatment with an investigational anticancer agent within 2 weeks (in the case of nitrosoureas and mitomycin C, within 6 weeks; in the case of immunotherapy, within 4 weeks) before the first administration of study drug. Localized palliative radiation therapy (but should not include radiation to target lesions) and ongoing bisphosphonates and denosumab, are permitted
* Has persistent phosphate level greater than upper limit of normal (ULN) during screening (within 14 days of treatment and prior to Cycle 1 Day 1) and despite medical management
* Has a history of or current uncontrolled cardiovascular disease
* Females who are pregnant, breast-feeding, or planning to become pregnant within 3 months after the last dose of study drug and males ho plan to father a child while enrolled in this study or within 5 months after the last dose of study drug
* Has not recovered from reversible toxicity of prior anticancer therapy (except toxicities which are not clinically significant such as alopecia, skin discoloration, or Grade 1 neuropathy)
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Janssen Research & Development, LLC

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Janssen Research & Development, LLC Clinical Trial

Role: STUDY_DIRECTOR

Janssen Research & Development, LLC

Locations

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Sedona, Arizona, United States

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Los Angeles, California, United States

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Orange, California, United States

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Sacramento, California, United States

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Stanford, California, United States

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Aurora, Colorado, United States

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Washington D.C., District of Columbia, United States

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Chicago, Illinois, United States

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Iowa City, Iowa, United States

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Louisville, Kentucky, United States

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Minneapolis, Minnesota, United States

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Omaha, Nebraska, United States

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Las Vegas, Nevada, United States

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New York, New York, United States

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Charlotte, North Carolina, United States

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Medford, Oregon, United States

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Tualatin, Oregon, United States

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Hershey, Pennsylvania, United States

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Pittsburgh, Pennsylvania, United States

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Myrtle Beach, South Carolina, United States

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Nashville, Tennessee, United States

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Dallas, Texas, United States

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Denton, Texas, United States

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Houston, Texas, United States

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Hampton, Virginia, United States

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Graz, , Austria

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Linz, , Austria

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Vienna, , Austria

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Aalst, , Belgium

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Brussels, , Belgium

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Charleroi, , Belgium

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Ghent, , Belgium

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Wilrijk, , Belgium

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Angers, , France

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Bordeaux, , France

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Caen Cédex 05, , France

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Dijon, , France

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Lyon, , France

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Nice, , France

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Nîmes, , France

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Paris, , France

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Saint-Herblain, , France

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Suresnes, , France

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Villejuif, , France

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Berlin, , Germany

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Erlangen, , Germany

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Essen, , Germany

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Freiburg im Breisgau, , Germany

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Göttingen, , Germany

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Greifswald, , Germany

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Hamburg, , Germany

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Hanover, , Germany

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Heidelberg, , Germany

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München, , Germany

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Münster, , Germany

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Regensburg, , Germany

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Straubing, , Germany

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Weiden/Opf, , Germany

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Bangalore, , India

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Kolkata, , India

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Mira Road (East), , India

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Nadiād, , India

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Beer Yaakov, , Israel

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Beersheba, , Israel

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Haifa, , Israel

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Kfar Saba, , Israel

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Petah Tikva, , Israel

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Tel Aviv, , Israel

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Chisinau, , Moldova

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Bucharest, , Romania

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Cluj-Napoca, , Romania

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Craiova, , Romania

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Iași, , Romania

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Barnaul, , Russia

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Moscow, , Russia

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Omsk, , Russia

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Pyatigorsk, , Russia

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Saint Petersburg, , Russia

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Ufa, , Russia

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Daejeon, , South Korea

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Goyang-si, , South Korea

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Incheon, , South Korea

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Seoul, , South Korea

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Badalona, , Spain

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Barcelona, , Spain

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Madrid, , Spain

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Málaga, , Spain

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Pamplona, , Spain

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Sabadell, , Spain

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Santander, , Spain

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Santiago de Compostela, , Spain

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Seville, , Spain

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Valencia, , Spain

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Taichung, , Taiwan

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Tainan, , Taiwan

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Taipei, , Taiwan

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Taoyuan District, , Taiwan

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Istanbul, , Turkey (Türkiye)

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Blackburn, , United Kingdom

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Dundee, , United Kingdom

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Essex, , United Kingdom

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London, , United Kingdom

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Metropolitan Borough of Wirral, , United Kingdom

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Plymouth, , United Kingdom

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Sutton, , United Kingdom

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Countries

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Italy United States Austria Belgium France Germany India Israel Moldova Romania Russia South Korea Spain Taiwan Turkey (Türkiye) United Kingdom

References

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Siefker-Radtke AO, Necchi A, Park SH, Garcia-Donas J, Huddart RA, Burgess EF, Fleming MT, Rezazadeh Kalebasty A, Mellado B, Varlamov S, Joshi M, Duran I, Tagawa ST, Zakharia Y, Akapame S, Santiago-Walker AE, Monga M, O'Hagan A, Loriot Y; BLC2001 Study Group. Efficacy and safety of erdafitinib in patients with locally advanced or metastatic urothelial carcinoma: long-term follow-up of a phase 2 study. Lancet Oncol. 2022 Feb;23(2):248-258. doi: 10.1016/S1470-2045(21)00660-4. Epub 2022 Jan 11.

Reference Type DERIVED
PMID: 35030333 (View on PubMed)

Dosne AG, Valade E, Goeyvaerts N, De Porre P, Avadhani A, O'Hagan A, Li LY, Ouellet D, Perez Ruixo JJ. Exposure-response analyses of erdafitinib in patients with locally advanced or metastatic urothelial carcinoma. Cancer Chemother Pharmacol. 2022 Feb;89(2):151-164. doi: 10.1007/s00280-021-04381-4. Epub 2022 Jan 3.

Reference Type DERIVED
PMID: 34977972 (View on PubMed)

Loriot Y, Necchi A, Park SH, Garcia-Donas J, Huddart R, Burgess E, Fleming M, Rezazadeh A, Mellado B, Varlamov S, Joshi M, Duran I, Tagawa ST, Zakharia Y, Zhong B, Stuyckens K, Santiago-Walker A, De Porre P, O'Hagan A, Avadhani A, Siefker-Radtke AO; BLC2001 Study Group. Erdafitinib in Locally Advanced or Metastatic Urothelial Carcinoma. N Engl J Med. 2019 Jul 25;381(4):338-348. doi: 10.1056/NEJMoa1817323.

Reference Type DERIVED
PMID: 31340094 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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42756493BLC2001

Identifier Type: OTHER

Identifier Source: secondary_id

2014-002408-26

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

2023-510273-34-00

Identifier Type: REGISTRY

Identifier Source: secondary_id

CR105065

Identifier Type: -

Identifier Source: org_study_id