Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285)
Research type
Research Study
Full title
An Open-label, Single Arm, Phase 2 Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, in Patients with Advanced Systemic Mastocytosis
IRAS ID
245587
Contact name
Dr Deepti Radia
Contact email
Sponsor organisation
Blueprint Medicines Corporation
Eudract number
2017-004836-13
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
124159, IND Number
Duration of Study in the UK
4 years, 11 months, 12 days
Research summary
This research study is an open label study designed to investigate the safety and effectiveness of an experimental drug called avapritinib (also known as BLU-285), when given to individuals who have been diagnosed with Advanced Systemic Mastocytosis (ASM).
Mast cells are produced in the bone marrow, the spongy tissue found in the hollow centres of some bones, and live longer than normal cells. They're an important part of the immune system and help fight infection. Mast cells also play a major role in allergic reactions by releasing histamine and other molecules into the bloodstream.
Mastocytosis is a condition that occurs when mast cells accumulate in skin and/or internal organs such as the liver, spleen, bone marrow, and small intestines and cause abnormal organ function. The signs and symptoms vary based on which part(s) of the body are affected.
Systemic mastocytosis (SM) is a fatal disease of the blood with no known cure and, when aggressive, can be fatal within four years of diagnosis. Some people with SM may experience episodes of severe symptoms that last 15-30 minutes, often with specific triggers such as physical exertion or stress. Symptoms can include itching, vomiting, diarrhoea, muscle/ joint pain, mood changes, headaches and tiredness.
This study aims to recruit approximately 60 participants globally. There will be an 8 week screening period during which eligibility will be assessed. Eligible participants will begin treatment with avapritinib at a dose of 200 mg once a day, in 28-day cycles. After at least 8 weeks of treatment, the dose can be escalated to 300mg once daily at the discretion of the investigator.
Dosing will continue unless the individual’s disease worsens, they have side-effects related to the treatment that prevent continuation, or they wish to stop treatment for any reason. Participants are expected to participate for 12 months.REC name
East of England - Cambridge South Research Ethics Committee
REC reference
18/EE/0306
Date of REC Opinion
5 Dec 2018
REC opinion
Further Information Favourable Opinion