A Study of Erdafitinib in Participants With Advanced Solid Tumors and Fibroblast Growth Factor Receptor (FGFR) Gene Alterations

• ClinicalTrials.gov Identifier: NCT04083976
• Recruitment Status: Recruiting
• First Posted: September 10, 2019
• Last Update Posted: April 8, 2021
• Sponsor: Janssen Research & Development, LLC
• Information provided by (Responsible Party): Janssen Research & Development, LLC

Tracking Information

• First Submitted Date: September 6, 2019
• First Posted Date: September 10, 2019
• Last Update Posted Date: April 8, 2021
• Actual Study Start Date: November 20, 2019
• Estimated Primary Completion Date: June 14, 2022 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: September 6, 2019)

Overall Response Rate (ORR) as Assessed by Independent Review Committee (IRC) [ Time Frame: Up to 6 Years ]

ORR as assessed by IRC is defined as the percentage of participants who achieve a complete response (CR) or partial response (PR).

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: September 6, 2019)

• Overall Response Rate as Assessed by Investigator [ Time Frame: Up to 6 Years ]
  ORR as assessed by investigator is defined as the percentage of participants who achieve a CR or PR.
• Duration of Response (DOR) [ Time Frame: Up to 6 Years ]
  DOR is the duration from the date of initial documentation of a response to the date of first documented evidence of progressive disease (or relapse for participants who experience CR during the study), or death, whichever comes first.
• Disease Control Rate (DCR) [ Time Frame: Up to 6 Years ]
  DCR is defined as the percentage of participants with CR, PR or stable disease (SD).
• Progression Free Survival (PFS) [ Time Frame: Up to 6 Years ]
  PFS is the duration from the date of the first dose of study drug until the date of first documented evidence of progressive disease (or relapse for participants who experience CR during the study) or death, whichever comes first.
• Overall Survival (OS) [ Time Frame: Up to 6 Years ]
  OS will be measured from the date of first dose of study drug to the date of the participant's death.
• Plasma Concentrations of Erdafitinib [ Time Frame: Predose and 2-4 hours postdose ]
  Plasma concentrations of erdafitinib will be reported.
• Number of Participants with Adverse Events (AEs) [ Time Frame: Up to 6 Years ]
  An AE is any untoward medical occurrence in a clinical study participant administered a medicinal (investigational or non-investigational) product. An adverse event does not necessarily have a causal relationship with the relevant investigational product.
• Number of Participants with Adverse Events by Severity [ Time Frame: Up to 6 Years ]
  An AE is any untoward medical occurrence in a clinical study participant administered a medicinal (investigational or non-investigational) product. Adverse event severity is a clinical determination of the intensity of an adverse event.
• Change from Baseline in Health-Related Quality of Life (HRQoL) as Assessed by European Organisation for Research and Treatment of Cancer Quality-of-life Questionnaire Core 30 (EORTC-QLQ-C30) Scale Score [ Time Frame: Baseline up to 6 Years ]
  The EORTC QLQ-C30 includes 30 items in 5 functional scales, 1 global health status scale, 3 symptom scales, and 6 single symptom items. The responses are reported using a verbal rating scale. The item and scale scores are transformed to a 0 to 100 scale. A higher score represents greater HRQoL, better functioning, and more (worse) symptoms.
• Change from Baseline in Health-Related Quality of Life as Assessed by Patient Global Impression of Symptom Severity (PGIS) Scale Score [ Time Frame: Baseline up to 6 Years ]
  The PGIS is a single question regarding the patient report of disease severity: considering all aspects of your cancer symptoms right now would you say your cancer symptoms are none, mild, moderate, severe, or very severe?
• Change from Baseline in Health-Related Quality of Life as Assessed by Patient Global Impression of Change (PGIC) Scale [ Time Frame: Baseline up to 6 Years ]
  The PGIC is the patient-reported outcome (PRO) counterpart to the clinical global impressions (CGI) scale. The PGIC is a single verbal rating scale ranging from 1 = a lot better now to 7 = a lot worse now.
• Change from Baseline in Health-Related Quality of Life as Assessed by European Quality of Life - 5 Dimensions-5 Levels (EQ-5D-5L) Scale Score [ Time Frame: Baseline up to 6 Years ]
  The EQ-5D-5L is a generic measure of health status. The EQ-5D-5L is a 5-item questionnaire that assesses 5 domains including mobility, self-care, usual activities, pain/discomfort and anxiety/depression plus a visual analog scale rating "health today" with anchors ranging from 0 (worst imaginable health state) to 100 (best imaginable health state).

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: A Study of Erdafitinib in Participants With Advanced Solid Tumors and Fibroblast Growth Factor Receptor (FGFR) Gene Alterations
• Official Title: A Phase 2 Study of Erdafitinib in Subjects With Advanced Solid Tumors and FGFR Gene Alterations
• Brief Summary: The purpose of this study is to evaluate the efficacy of erdafitinib in terms of overall response rate (ORR) in participants with advanced solid tumors with fibroblast growth factor receptor (FGFR) mutations and gene fusions.
• Detailed Description: Erdafitinib is a selective and potent pan FGFR 1-4 inhibitor with demonstrated clinical activity in participants with metastatic urothelial cancer and cholangiocarcinoma identified to have alterations in the FGFR pathway. This study targets the underlying altered biology of FGFR-driven tumors irrespective of solid tumor histology subtype. The study consists of screening phase, treatment phase and the post treatment follow-up phase (from the end of treatment visit until the participants has died, withdraws consent, is lost to follow-up, or the end of study, whichever comes first). End of study is defined as 4 years from enrollment of last participant into study or last follow-up visit of last patient, whichever occurs first.
• Study Type: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Advanced Solid Tumor

Intervention

Drug: Erdafitinib

Participants will receive erdafitinib oral tablets.

Other Name: JNJ-42756493

Study Arms

Experimental: Erdafitinib

Participants with fibroblast growth factor receptor (FGFR) mutations and FGFR gene fusions will receive a dose of erdafitinib oral tablets until disease progression, intolerable toxicity, withdrawal of consent, or decision by the investigator to discontinue treatment.

Intervention: Drug: Erdafitinib

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: September 6, 2019): 280
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: September 11, 2026
• Estimated Primary Completion Date: June 14, 2022 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Histologic demonstration of an unresectable, locally advanced, or metastatic solid tumor malignancy with an fibroblast growth factor receptor (FGFR) mutation or FGFR gene fusion
• Measurable disease
• Participant must have received at least one prior line of systemic therapy in the advanced, unresectable, or metastatic setting
• Documented progression of disease, defined as any progression that requires a change in treatment, prior to full study screening

Exclusion Criteria:

• Has had prior chemotherapy, targeted therapy, or treatment with an investigational anticancer agent within 15 days or greater than or equal to (>=) 5 half-lives of the agent (whichever is longer) and up to a maximum of 30 days before the first dose of erdafitinib
• The presence of FGFR gatekeeper and resistance mutations
• Histologic demonstration of urothelial carcinoma
• Hematologic malignancy (i.e., myeloid and lymphoid neoplasms
• For non-small cell lung cancer participants only: pathogenic somatic mutations or gene fusions in the following genes: EGFR, ALK, ROS1, NTRK, and BRAF V600E
• Active malignancies other than for disease requiring therapy

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 12 Years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Contacts

Contact: Study Contact; 844-434-4210; JNJ.CT@sylogent.com

• Listed Location Countries: Argentina, Australia, Belgium, Brazil, China, France, Germany, Italy, Japan, Korea, Republic of, Poland, Spain, Taiwan, United Kingdom, United States

Administrative Information

• NCT Number: NCT04083976
• Other Study ID Numbers: CR108661; 2019-002113-19 ( EudraCT Number ); 42756493CAN2002 ( Other Identifier: Janssen Research & Development, LLC )
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: Yes
• Plan Description: The data sharing policy of the Janssen Pharmaceutical Companies of Johnson & Johnson is available at www.janssen.com/clinical-trials/transparency. As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at yoda.yale.edu
• URL: https://www.janssen.com/clinical-trials/transparency

• Responsible Party: Janssen Research & Development, LLC
• Study Sponsor: Janssen Research & Development, LLC
• Collaborators: Not Provided

Investigators

• Study Director: Janssen Research & Development, LLC Clinical Trial; Janssen Research & Development, LLC

• PRS Account: Janssen Research & Development, LLC
• Verification Date: April 2021