AZURE

• Research type

  Research Study

• Full title

  A Phase 1/2, open-label, 2-arm study evaluating BLU-263 as monotherapy and in combination with azacitidine, in patients with KIT altered hematologic malignancies

• IRAS ID

  1005803

• Contact name

  Tanya Green

• Contact email

  tgreen@blueprintmedicines.com

• Sponsor organisation

  Blueprint Medicines Corporation

• Eudract number

  2022-001535-87

• Clinicaltrials.gov Identifier

  NCT05609942

• Research summary

  Systemic Mastocytosis (SM) is a rare disease caused by too many mast cells (MC), that build up inside of your body in organs, such as the skin, liver, spleen, bone marrow and intestines. The disease is often caused by a change in a cell’s DNA, which is called a mutation. In SM, the most frequent mutation is called KIT D816V.

  SM may cause health problems that can deeply affect a person’s quality of life and how they feel physically, emotionally, and socially.
  This includes:
  - skin lesions
  - itchy skin
  - diarrhoea
  - brain fog (trouble remembering, learning new things, concentrating, etc.)
  - fatigue
  - bone pain

  There is also a chance of anaphylaxis, which is a severe allergic reaction that can cause death. People with a type of SM called Advanced SM (AdvSM) may not live as long as others who do not have SM. Some people with AdvSM may also develop a type of blood cancer called an Associated Haematological Neoplasm (AHN).

  SM is thought to occur in about 1 in every 10,000 people. Out of the people who have SM, about 1 in every 20 have AdvSM. In the past, patients with AdvSM did not have many treatment options. There is a medical need for patients with AdvSM to be treated with drugs that are selective and targeted to the KIT mutation. BLU-263 was created to target the mutation that often causes SM. This study will first test the potential benefits and safety of BLU-263 in people with AdvSM. After initial testing, BLU-263 will be tested along with a second drug created to treat AHN. Around 108 adults with AdvSM will take part in this worldwide study. Patients will take part for an average of 4 years, although this time could be longer or shorter.

• REC name

  London - Brent Research Ethics Committee

• REC reference

  22/LO/0865

• Date of REC Opinion

  15 May 2023

• REC opinion

  Further Information Favourable Opinion