DZB-CS-301: Oral Derazantinib in iCCA with genomic aberration
Research type
Research Study
Full title
A pivotal study of derazantinib in patients with inoperable or advanced intrahepatic cholangiocarcinoma and FGFR2 gene fusions or FGFR2 gene mutations or amplifications
IRAS ID
256189
Contact name
Jeff Evans
Contact email
Sponsor organisation
Basilea Pharmaceutica International Ltd
Eudract number
2016-004448-12
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
N/A, N/A
Duration of Study in the UK
1 years, 5 months, 27 days
Research summary
The purpose of this single-arm, open label, research study is to test how well the study medication, derazantinib, works in patients with inoperable or advanced intrahepatic cholangiocarcinoma (iCCA) and FGFR2 genetic aberrations.
iCCA is asymptomatic in the early stages and by the time it is diagnosed, most patients present with an advanced stage of the disease. At this point approximately 90% of patients are not eligible for surgery. Even combined multi-agent chemotherapy does not offer a lasting response and only 5-10% of patients survive 5 years after diagnosis. FGFR dysregulations such as fusions, amplification, and mutations are frequently observed in human cancers, including iCCA.
The study consists of 2 Sub-studies. Sub-Study 1 will enrol approx. 100 patients with FGFR2 gene fusions to determine objective response rate (ORR) and Sub-Study 2 will enrol patients with FGFR2 gene mutations or amplifications and will be conducted in two stages with approximately 15 patients in stage 1 and 28 patients if the study proceeds to stage 2. In each sub-study, a sub-set of up to 20 patients will be asked to participate in biomarker assessments, rich PK blood sampling and PK urine sampling.
Patients will receive treatment in cycles, continuously. Each treatment cycle will be 28 days long. Subjects will receive treatment with derazantinib capsules until withdrawal of consent, death, radiographic disease progression, or unacceptable toxicity occurs. Procedures will include, but are not limited to, blood tests, vital signs, a test to measure heart activity (electrocardiogram), Radiological or MRI Scans to measure their tumour and answering questions about quality of life.
The study is expected to last approx. 4 years and will take place within over 50 centres globally.
Basilea Pharmaceutica International Ltd. is funding the research.
REC name
West of Scotland REC 1
REC reference
19/WS/0107
Date of REC Opinion
2 Sep 2019
REC opinion
Further Information Favourable Opinion