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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT04083976




Registration number
NCT04083976
Ethics application status
Date submitted
6/09/2019
Date registered
10/09/2019
Date last updated
21/09/2020

Titles & IDs
Public title
A Study of Erdafitinib in Participants With Advanced Solid Tumors and Fibroblast Growth Factor Receptor (FGFR) Gene Alterations
Scientific title
A Phase 2 Study of Erdafitinib in Subjects With Advanced Solid Tumors and FGFR Gene Alterations
Secondary ID [1] 0 0
2019-002113-19
Secondary ID [2] 0 0
CR108661
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Advanced Solid Tumor 0 0
Condition category
Condition code

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Erdafitinib

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.


Treatment: Drugs: Erdafitinib
Participants will receive erdafitinib oral tablets.

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Overall Response Rate (ORR) as Assessed by Independent Review Committee (IRC) - ORR as assessed by IRC is defined as the percentage of participants who achieve a complete response (CR) or partial response (PR).
Timepoint [1] 0 0
Up to 6 Years
Secondary outcome [1] 0 0
Overall Response Rate as Assessed by Investigator - ORR as assessed by investigator is defined as the percentage of participants who achieve a CR or PR.
Timepoint [1] 0 0
Up to 6 Years
Secondary outcome [2] 0 0
Duration of Response (DOR) - 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.
Timepoint [2] 0 0
Up to 6 Years
Secondary outcome [3] 0 0
Disease Control Rate (DCR) - DCR is defined as the percentage of participants with CR, PR or stable disease (SD).
Timepoint [3] 0 0
Up to 6 Years
Secondary outcome [4] 0 0
Progression Free Survival (PFS) - 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.
Timepoint [4] 0 0
Up to 6 Years
Secondary outcome [5] 0 0
Overall Survival (OS) - OS will be measured from the date of first dose of study drug to the date of the participant's death.
Timepoint [5] 0 0
Up to 6 Years
Secondary outcome [6] 0 0
Plasma Concentrations of Erdafitinib - Plasma concentrations of erdafitinib will be reported.
Timepoint [6] 0 0
Predose and 2-4 hours postdose
Secondary outcome [7] 0 0
Number of Participants with Adverse Events (AEs) - 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.
Timepoint [7] 0 0
Up to 6 Years
Secondary outcome [8] 0 0
Number of Participants with Adverse Events by Severity - 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.
Timepoint [8] 0 0
Up to 6 Years
Secondary outcome [9] 0 0
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 - 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.
Timepoint [9] 0 0
Baseline up to 6 Years
Secondary outcome [10] 0 0
Change from Baseline in Health-Related Quality of Life as Assessed by Patient Global Impression of Symptom Severity (PGIS) Scale Score - 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?
Timepoint [10] 0 0
Baseline up to 6 Years
Secondary outcome [11] 0 0
Change from Baseline in Health-Related Quality of Life as Assessed by Patient Global Impression of Change (PGIC) Scale - 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.
Timepoint [11] 0 0
Baseline up to 6 Years
Secondary outcome [12] 0 0
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 - 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).
Timepoint [12] 0 0
Baseline up to 6 Years

Eligibility
Key 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
Minimum age
12 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Flinders Centre for Innovation in Cancer - Adelaide
Recruitment hospital [2] 0 0
Chris O'Brien Lifehouse - Camperdown
Recruitment hospital [3] 0 0
St. Vincent's Hospital Sydney - Darlinghurst
Recruitment hospital [4] 0 0
St Vincents Hospital Melbourne - Melbourne
Recruitment hospital [5] 0 0
Fiona Stanley Hospital - Murdoch
Recruitment hospital [6] 0 0
Intergrated Clinical Oncology Network Pty Ltd (ICON) - South Brisbane
Recruitment postcode(s) [1] 0 0
5042 - Adelaide
Recruitment postcode(s) [2] 0 0
2050 - Camperdown
Recruitment postcode(s) [3] 0 0
2010 - Darlinghurst
Recruitment postcode(s) [4] 0 0
3065 - Melbourne
Recruitment postcode(s) [5] 0 0
6150 - Murdoch
Recruitment postcode(s) [6] 0 0
4101 - South Brisbane
Recruitment outside Australia
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Bydgoszcz
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Konin
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Barcelona
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Hospitalet de Llobregat, Barcelona
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Madrid
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Sevilla
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Valencia
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Sutton
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Wirral

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Janssen Research & Development, LLC
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/show/NCT04083976
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Janssen Research & Development, LLC Clinical Trial
Address 0 0
Janssen Research & Development, LLC
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Study Contact
Address 0 0
Country 0 0
Phone 0 0
844-434-4210
Fax 0 0
Email 0 0
JNJ.CT@sylogent.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT04083976