Pioneer
Research type
Research Study
Full title
A 3 Part, Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Evaluate Safety and Efficacy of Avapritinib (BLU-285), a Selective KIT Mutation-Targeted Tyrosine Kinase Inhibitor, in Indolent and Smoldering Systemic Mastocytosis with Symptoms Inadequately Controlled with Standard Therapy
IRAS ID
254054
Contact name
Anthony Boral
Contact email
Sponsor organisation
Blueprint Medicines Corporation
Eudract number
2018-000588-99
ISRCTN Number
ISRCTN00000000
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
124159, IND Number
Duration of Study in the UK
5 years, 0 months, 2 days
Research summary
This is a 3 part, randomised, double blind study to learn more about the safety and efficacy of an experimental drug called avapritinib (formerly called BLU-285) when given to individuals who have been diagnosed with Indolent Systemic Mastocytosis (ISM) or Smoldering Systemic Mastocytosis (SSM). Indolent Systemic Mastocytosis (ISM) and Smoldering Systemic Mastocytosis (SSM) are subtypes of Systemic Mastocytosis which is a disorder where there are too many mast cells in bone marrow and/or in other organs and these mast cells release their contents abnormally. Mast cells are a type of white blood cell that is part of the immune system. When increased in number or abnormal in function they can accumulate in organs and release their contents resulting in allergic reactions and other related symptoms such as itching, redness of the skin, pain, fatigue, mood changes.
The research study will be conducted at approximately 22 medical centres worldwide and is divided into 3 parts. Approximately 40 participants will participate in Part 1 of the research study, and approximately 72 participants will participate in Part 2. After participants finish Part 1 or Part 2, they will all transition to Part 3 for continued treatment.
Recruitment is for participants 18 years or older and in the UK will be at secondary care NHS sites by specialists experienced in diagnosing and treating the condition.
The study will last approximately 5 years.
REC name
London - City & East Research Ethics Committee
REC reference
19/LO/0142
Date of REC Opinion
14 Mar 2019
REC opinion
Further Information Favourable Opinion