Avapritinib Rollover Study
Research type
Research Study
Full title
An Open-label, Multicenter, Rollover Study in Patients Who Participated in an Avapritinib Clinical Study
IRAS ID
1010233
Contact name
Tanya Green
Contact email
Sponsor organisation
Blueprint Medicines Corporation
Research summary
This is an open-label rollover study funded by Blueprint Medicines Corporation to provide continued access to avapritinib and evaluate long-term safety and tolerability in patients with Systemic Mastocytosis (SM) with a subtype of Advanced SM or Indolent SM (ISM) who have participated in a Blueprint Medicines sponsored study and continue to clinically benefit from avapritinib. ISM and Advanced SM are disorders where abnormal mast cells (a type of white blood cell) accumulate in organs and bone marrow and cause abnormal organ function. The study will be run in approximately 13 sites in 7 countries. Approximately 60 patients will participate.
Avapritinib will be administered orally at the dose and frequency received on the parent study. Study visits will be conducted on an outpatient basis but may be performed on an inpatient basis as needed. No maximum treatment duration has been set. Study visits will be done at least every 6 months. Patients will remain on the study until any discontinuation criteria are met. Patients will be monitored at regular intervals for safety. All patients will have an end of treatment (EOT) evaluation with reason for study drug discontinuation recorded.
The following assessments will be done:
- Screening visit: informed consent, inclusion/exclusion criteria, demographics, serum pregnancy test (if applicable).
- On-treatment visits: safety assessments and study drug compliance. Participants with ISM who increase in dose of avapritinib over 25mg will also have blood tests to assess complete blood count at the time of dose increase, 3 months and 6 months post dose increase, and the continue according to local standard of care/requirements.
- EOT visit (within 14 days of last-dose): safety assessments, study drug compliance, serum pregnancy test (if applicable).
- Safety follow-up visit (30 days after last dose), this may be via phone call.REC name
London - Surrey Borders Research Ethics Committee
REC reference
24/LO/0697
Date of REC Opinion
18 Nov 2024
REC opinion
Further Information Favourable Opinion