Blueprint Medicines

This is a randomized, double-blind, placebo-controlled, Phase 2/3 study comparing the efficacy and safety of elenestinib (BLU-263) + symptom directed therapy (SDT) with placebo + SDT in participants with indolent systemic mastocytosis (ISM) whose symptoms are not adequately controlled by SDT. Parts 1 and 2 will enroll participants with ISM. Participants enrolled in Part 2 will roll over onto Part 3 to receive treatment with elenestinib in an open-label fashion following completion of the earlier Part. Part K will enroll participants with ISM who have previously received an approved selective KIT inhibitor. The study also includes pharmacokinetic (PK) groups that will enroll participants with ISM.Show more

This is a Phase 4, non-interventional, observational study to collect detailed data on the clinical characteristics, clinical outcomes and medical management of ISM in real-world settings. The study will describe the demographic and clinical characteristics of ISM participants, including anaphylaxis and bone manifestations in ISM. Quality of life and disease control will be assessed through participant questionnaires. The study will also evaluate real world ISM treatment management, including use of avapritinib.Show more

This is a multicenter screening study to characterize the prevalence of the KIT D816V mutation in participants with suspected clonal mast cell disease.

This is a non-interventional study assessing the effectiveness of avapritinib (BLU-285) in the management of ISM in real-world settings in Germany. The study also seeks to address the existing data gap in the natural history and management of participants with ISM. The study is designed to follow each participant up to a maximum of 24 months.Show more

Systemic mastocytosis with an associated hematologic neoplasm (SM-AHN) is a challenging disease to treat. Targeted KIT inhibitors have been approved for this indication based on their ability to control the mastocytosis portion of the disease, but patients frequently experience progression of the concomitant myeloid malignancy (i.e. the AHN). Using a combination approach to treat both aspects of the disease has the potential to provide enhanced disease control; however, overlapping toxicity is a concern. In this study, investigators aim to study the safety and tolerability of combined avapritinib and decitabine for the treatment of SM-AHN.Show more

The primary objective of the rollover study is to evaluate the long-term safety of avapritinib in participants who have completed a Blueprint Medicines sponsored study (parent study) and continued to benefit from avapritinib.

This is a Phase 2, randomized, double-blind, placebo-controlled study comparing the efficacy and safety of avapritinib + best supportive care (BSC) with placebo + BSC in patients with indolent systemic mastocytosis (ISM) whose symptoms are not adequately controlled by BSC. The study will be conducted in 3 parts. All patients will receive treatment with avapritinib during Part 3 including those rolling over from the placebo group.Show more

Blueprint Medicines is a precision therapy company focused on genomically defined cancers and blood disorders. Sanofi completed its $9.1 billion acquisition of Blueprint on July 18, 2025 (announced June 2025). The lead product is avapritinib (Ayvakit/Ayvakyt), approved for advanced systemic mastocytosis, indolent systemic mastocytosis (ISM, approved 2023), and PDGFRA D842V-mutant gastrointestinal stromal tumors. Blueprint also discovered pralsetinib (Gavreto) for RET-altered cancers and regained global commercialization rights from Roche in 2024. The pipeline includes elenestinib (BLU-263), a next-generation KIT D816V inhibitor in Phase 2/3 for indolent systemic mastocytosis.

Blueprint Medicines has active programs across 8 rare diseases: Cholangiocarcinoma, Familial Medullary Thyroid Carcinoma, Gastrointestinal Stromal Tumor, Hereditary Diffuse Gastric Cancer, Mastocytosis, Medullary Thyroid Cancer, RET Fusion Cancer, Systemic Mastocytosis. Each disease page on Trial Friend shows the latest recruiting trials, FDA-approved treatments, and patient resources.

Yes. Blueprint Medicines is a rare disease specialist headquartered in Cambridge, United States, founded in 2011. The company focuses primarily on developing treatments for rare and orphan diseases, with programs targeting Cholangiocarcinoma, Familial Medullary Thyroid Carcinoma, Gastrointestinal Stromal Tumor among others. It operates as part of Sanofi.

The clinical trials section on this page shows active and recruiting rare disease trials sponsored by Blueprint Medicines, pulled live from ClinicalTrials.gov. Each trial card includes the study phase, recruitment status, conditions being studied, number of trial sites, and a direct link to the full study record. You can also explore individual disease pages to see trials from all sponsors for a specific condition.

To explore enrollment, find a recruiting trial in the clinical trials list on this page and click "View full study" to see the full ClinicalTrials.gov record, which includes eligibility criteria, study locations, and contact information for each trial site. Your doctor can also help determine if a specific trial is appropriate for your condition. Not all trials are open at every location, so check the site list for one near you.

Blueprint Medicines was founded in 2011 and is headquartered in Cambridge, United States. The company has been active for 15 years. It is a subsidiary of Sanofi. It trades publicly under the ticker symbol BPMC. As a rare disease specialist, the company dedicates its resources to developing therapies for patients with rare and orphan conditions.