Phase 1 Study of BLU-554 in Patients with Hepatocellular Carcinoma
Research type
Research Study
Full title
A Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of BLU-554 in Patients with Hepatocellular Carcinoma and Cholangiocarcinoma.
IRAS ID
181060
Contact name
Patrick McNamara
Contact email
Sponsor organisation
Blueprint Medicines
Eudract number
2015-001662-26
Duration of Study in the UK
4 years, 0 months, 1 days
Research summary
This is a Phase 1, first-in-human study of BLU-554 in patients with Hepatocellular Carcinoma (HCC) and Cholangiocarcinoma.
HCC is a leading cause of cancer-related deaths and represents a worldwide unmet clinical need. Given the poor prognosis of patients with advanced HCC and cholangiocarcinoma and the potential risk/benefit of BLU-554 in the treatment of patients with these conditions, further development of this agent is warranted.
The study consists of a dose-escalation (Part 1) and a dose-expansion (Part 2). Part 1 will include patients with HCC who have previously been treated with sorafenib, have declined sorafenib, or who have not had access to sorafenib. The primary aims of Part 1 are to define the safety and tolerability of BLU-554, to identify the maximum tolerated dose (MTD) or recommended Phase 2 dose and to characterise BLU-554 pharmacokinetics and pharmacodynamics.
The Part 2 dose expansion will start once an MTD has been determined in part 1. Part 2 will enrol patients with HCC and patients with cholangiocarcinoma.
Approximately 60 patients will be enrolled in this study, including approx. 25 patients in Part 1 and approx. 35 patients in Part 2. Up to 40 centres in the US, EU and Asia will participate in this study.
The minimum duration of treatment participation is approx. 3 months, including a screening period to assess study eligibility, of up to 4 weeks, a treatment period of at least 1 cycle (28 days) an End of Treatment visit and a Follow-up telephone contact.
Eligible patients will undergo PK and PD sample collection, vital signs measurement, ECG monitoring, safety laboratory tests, disease-response assessment by magnetic resonance imaging (MRI) or computed tomography (CT) and adverse event collection. Continuous Holter monitoring will be performed in patients in Part 2 of the study at selected centres only.
The study is sponsored by Blueprint Medicines.REC name
HSC REC B
REC reference
15/NI/0137
Date of REC Opinion
30 Jul 2015
REC opinion
Favourable Opinion