Avapritinib for Systemic Mastocytosis
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial focuses on evaluating the long-term safety of avapritinib, a drug for individuals with systemic mastocytosis. Systemic mastocytosis causes an excess of mast cells, leading to issues like skin rashes, digestive problems, and bone pain. The trial targets participants who previously took part in a study with avapritinib and experienced positive outcomes. Ideal participants completed the initial study, adhered to its guidelines, and continue to benefit from avapritinib treatment. As a Phase 4 trial, this study involves an FDA-approved treatment and aims to understand how avapritinib can benefit more patients.
What is the safety track record for avapritinib?
Research has shown that avapritinib is generally easy for patients to handle. In a previous study, serious side effects occurred in only about 0.7% of patients. Another study found that avapritinib has a well-understood safety record, with no new safety issues emerging over time. Patients taking this treatment experienced improvements in their symptoms and overall quality of life. These findings suggest that avapritinib is safe for long-term use in individuals who continue to benefit from it.12345
Why are researchers enthusiastic about this study treatment?
Avapritinib is unique because it specifically targets the KIT D816V mutation, which is a common driver in systemic mastocytosis. Unlike standard treatments, such as midostaurin, which broadly inhibit multiple kinases, avapritinib is more selective, potentially leading to fewer off-target effects. Researchers are excited about avapritinib because its precision in targeting the mutation could offer improved efficacy and a better side effect profile, making it a promising option for patients with systemic mastocytosis.
What is the effectiveness track record for avapritinib in treating systemic mastocytosis?
Research shows that avapritinib, the treatment under study in this trial, effectively treats systemic mastocytosis. Studies have found that avapritinib reduces symptoms and improves patients' quality of life over time. Specifically, for patients with advanced systemic mastocytosis, 76% survived after 24 months, highlighting a significant benefit. Additionally, patients experienced lasting improvements in symptoms and quality of life during treatment, even with lower doses. Overall, avapritinib has proven to be a safe and effective option for different types of systemic mastocytosis.678910
Are You a Good Fit for This Trial?
This trial is for individuals who have systemic mastocytosis and are currently benefiting from avapritinib after participating in a previous Blueprint Medicines study. They must be willing to use effective contraception, have followed the earlier study's rules, can consent in writing, and women of childbearing age need a recent negative pregnancy test.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants continue to receive avapritinib to evaluate long-term safety
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Avapritinib
Find a Clinic Near You
Who Is Running the Clinical Trial?
Blueprint Medicines Corporation
Lead Sponsor