Open-label Phase II Trial with High Risk NMIBC and FGFR Alterations
Research type
Research Study
Full title
A Randomized Phase 2 Study of Erdafitinib Versus Investigator Choice of Intravesical Chemotherapy in Subjects Who Received Bacillus Calmette-Guérin (BCG) and Recurred With High Risk Non-Muscle-Invasive Bladder Cancer (NMIBC) and FGFR Mutations or Fusions
IRAS ID
276212
Contact name
James Catto
Contact email
Sponsor organisation
Janssen-Cilag International NV
Eudract number
2019-002449-39
Duration of Study in the UK
6 years, 3 months, 18 days
Research summary
This research is an open label phase 2 trial to investigate the efficacy and the safety of erdafitinib compared to investigator's choice of intravesical treatment (Mitomycin C/hyperthermic Mitomycin C) in subjects with high risk Non-Muscle Invasive Bladder Cancer (NMIBC) and FGFR mutations or fusions.
In total 280 patients will be enrolled into 3 cohort in this study.
Cohort 1 will enrol 240 high-risk NMIBC patients presenting as papillary tumour only, with disease recurrence after BCG therapy, and who either refuse or are not eligible for cystectomy. Patients will be assigned randomly 2:1 to either erdafitinib or investigator's choice of intravesical treatment. Treatment will be once weekly for at least 4 doses then monthly for at least 6 months. Additional doses are allowed per local standard of care. Randomisation will be stratified by tumour stage and type of BCG. Patients with high-risk recurrence on Investigator’s Choice may cross over to treatment with erdafitinib.
Exploratory Cohort 2 will enrol 20 high-risk BCG-unresponsive NMIBC patients presenting as CIS and will receive Erdafitinib
Exploratory Cohort 3 will enrol 20 intermediate-risk NMIBC patients presenting with papillary disease only and will receive Erdafitinib.
Patients receiving erdafitinib will have starting doses of 8mg once daily for 28 days in a 28-day cycle, which may be increased to 9mg once daily based on Cycle 1 Day 14 serum phosphate levels.
The study comprises of a Screening Phase (molecular screening and full study screening), a Treatment Phase, and a post-treatment Follow-up Phase. Subjects will be assessed on a regular basis for disease response (positive urine cytology/cystoscopy and CT/MRI Urogram). The end of study is defined as 4 years after the last subject is enrolled or last follow-up survival assessment for the last subject participating in the study. Two interim analyses are planned for Cohort 1.REC name
Yorkshire & The Humber - Leeds East Research Ethics Committee
REC reference
20/YH/0025
Date of REC Opinion
12 Mar 2020
REC opinion
Further Information Favourable Opinion