BLU-263 for Systemic Mastocytosis
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial examines a new treatment called elenestinib (BLU-263) for individuals with indolent systemic mastocytosis (ISM), a condition where certain immune cells accumulate and cause symptoms like skin rashes and abdominal pain. The goal is to determine if elenestinib can better control these symptoms compared to a placebo, especially for those whose current treatments are insufficient. Participants will receive either elenestinib or a placebo, alongside their usual symptom treatments. This trial may suit individuals with ISM whose symptoms are not well-managed by current therapies like antihistamines or corticosteroids. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering participants a chance to potentially benefit from a promising new therapy.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop all current medications, but it does require that your current symptom management medications are stable for at least 14 days before starting. If you're on corticosteroids, the dose must be 20 mg/d or less and stable for at least 14 days. Some specific treatments like cytoreductive therapy must be stopped for a certain period before screening.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research has shown that elenestinib, the treatment under study for systemic mastocytosis, is generally safe. Earlier studies found that it lowers the number of mast cells (immune cells that can cause symptoms when they accumulate) and improves symptoms. Importantly, these studies suggest that patients tolerate elenestinib well.
One study found that elenestinib might be safer than traditional treatments for this condition. Some side effects have been reported, but they are usually manageable, meaning mild issues may occur, but serious problems are less common.
Elenestinib is currently being tested in later stages of clinical trials, which often provide more detailed information about its safety in humans. This phase of testing typically follows earlier studies that have shown the treatment is safe enough for larger groups.12345Why do researchers think this study treatment might be promising for systemic mastocytosis?
Researchers are excited about elenestinib for systemic mastocytosis because it offers a novel approach compared to current treatments. Most existing options, like antihistamines or tyrosine kinase inhibitors, focus on managing symptoms or targeting specific enzymes. Elenestinib stands out by specifically targeting the KIT D816V mutation, which is a common driver of systemic mastocytosis. This targeted action promises a more precise treatment strategy, potentially leading to better outcomes with fewer side effects. Additionally, elenestinib is taken orally, making it a convenient option for long-term management.
What evidence suggests that this trial's treatments could be effective for indolent systemic mastocytosis?
Research has shown that elenestinib, a new medication, holds potential for treating indolent systemic mastocytosis (ISM). Studies have found that it can reduce the number of problematic mast cells, potentially improving symptoms for those affected. Participants in this trial may receive elenestinib at various doses, while others will receive a placebo. Patients who previously used elenestinib reported symptom relief, and it has been generally safe. Experts believe it could offer a more effective and safer option compared to older treatments. This medication specifically targets a common mutation in systemic mastocytosis, which might lead to better symptom control.12356
Are You a Good Fit for This Trial?
This trial is for patients with indolent systemic mastocytosis (ISM) who still have symptoms despite best supportive care. They should have a blood tryptase level above 8 ng/mL or severe reactions to allergens, an ECOG Performance Status of 0-2, and not controlled symptoms after trying at least two therapies like antihistamines or corticosteroids. People can't join if they've had certain heart issues, other myeloproliferative disorders, recent cancer treatments outside of specific exceptions, or previous treatment with targeted KIT inhibitors.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment Part 1
Participants receive BSC and various doses of elenestinib or placebo until completion of Part 1
Treatment Part 2
Participants receive BSC and the recommended dose of elenestinib or placebo for approximately 24 weeks
Open-label Treatment Part 3
Participants receive open-label BSC and elenestinib for up to approximately 4 years
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- BLU-263
- Placebo
Find a Clinic Near You
Who Is Running the Clinical Trial?
Blueprint Medicines Corporation
Lead Sponsor