A new clinical study sponsored by Blueprint Medicines Corp. will examine the long-term safety profile of avapritinib to treat patients with systemic mastocytosis (SM). Blueprint markets avapritinib under the trade name Aykavit.
The new clinical trial is currently recruiting participants who previously took part in a clinical trial sponsored by Blueprint Medicines for avapritinib. Investigators aim to enroll 60 participants and have three study locations, in Belgium, the Netherlands and Norway.
What is SM?
Systemic mastocytosis (SM) is a rare hematological disease characterized by mast cells that are overactive and accumulate in different parts of the body such as the bone marrow, liver, spleen, gastrointestinal tract and lymph nodes.
The clinical trial is an open-label rollover study, meaning that participants who received the treatment in the parent study can continue to receive it. Neither they nor the researchers will be blinded to their treatment status.
Read more about SM therapies
The primary endpoint of the follow-up trial is the onset of a serious adverse event, an adverse event of special interest or any adverse event that prompts discontinuation of the drug. The team expects to follow participants for up to four years.
The new study is open to children, adults and older adults who complied with the protocol of the parent study, who demonstrated clinical benefit from avapritinib treatment and who are not currently participating in any other interventional studies.
The trial began on Nov. 28 and has an expected completion date of Dec. 31, 2027.
The U.S. Food and Drug Administration previously approved avapritinib following results of Blueprint Medicine’s Phase 2 PIONEER study, which demonstrated the medication’s efficacy in treating patients with SM. The European Medicines Agency also approved the medication.
Avapritinib works to inhibit a faulty protein coded by a mutation in the KIT gene, thereby decreasing mast cell proliferation. The KIT gene codes for the protein CD117, and the mutation occurs often in patients with SM. CD117 belongs to a family of proteins known as receptor tyrosine kinases that play a role in the development of mast cells.
