HARBOR: Study of BLU-263 for ISM
Research type
Research Study
Full title
A Randomized, Double-Blind, Placebo-Controlled Phase 2/3 Study of BLU-263 in Indolent Systemic Mastocytosis
IRAS ID
1003872
Contact name
Tanya Green
Contact email
Sponsor organisation
Blueprint Medicines Corporation
Eudract number
2020-005173-28
Clinicaltrials.gov Identifier
Research summary
Systemic mastocytosis (SM) is a rare disease which results in accumulation of mast cells (MC) in internal tissues and organs, such as the skin, the liver, spleen, bone marrow, small intestines. SM can be associated with weakening and potentially life-threatening symptoms including anaphylaxis (allergic reaction), skin lesions, itchy skin, diarrhoea, cognitive impairments (trouble remembering, learning new things, concentrating etc). These symptoms have a severely negative impact on the quality of life (QoL) of patients physically, emotionally, and psychosocially.
Typical treatments of the symptoms include the use of multiple medications at the same time which aim to ease the symptoms but do not address the basic cause of the disease. Example medications include antihistamines, H2-blockers, proton-pump inhibitors, sodium cromoglycate, steroids and anti-IgE antibodies.
SM is estimated to occur in 1 in 100,000 people. Ninety-five percent of patients with SM are considered to have non-advanced SM (non-AdvSM), which includes the World Health Organization (WHO) variant of indolent SM (ISM) as well as a small number of patients with smouldering SM (SSM). Patients with non-AdvSM suffer long term and may worsen over time with no approved treatments to reduce their burden of disease or impact their disease course. Approximately 5% of patients with ISM show disease progression to severe forms of SM associated with poor overall survival. Currently there are no available targeted therapies for patients with ISM.
The study will test the drug BLU-263. BLU-263 is a new drug, that is being developed for treating people with ISM and/or mMCAS. BLU-263 is the first drug in a group of drugs that inhibits the DNA mutation that causes SM called KIT D816V.
Approximately 403 adults will take part in this worldwide study. This study will be conducted at approximately 50 sites globally.REC name
North West - Haydock Research Ethics Committee
REC reference
22/NW/0070
Date of REC Opinion
27 May 2022
REC opinion
Further Information Favourable Opinion