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Also known as BLU-263
Elenestinib · 1 trial · 2 indications
| Arm | Type | Description |
|---|---|---|
| (Part 1) Elenestinib Dose 1 + SDT | EXPERIMENTAL | Participants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily until completion of Part 1. |
| (Part 1) Elenestinib Dose 2 + SDT | EXPERIMENTAL | Participants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily until completion of Part 1. |
| (Part 1) Elenestinib Dose 3 + SDT | EXPERIMENTAL | Participants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily until completion of Part 1. |
| (Part 1) Placebo + SDT | PLACEBO_COMPARATOR | Participants will receive SDT and matching placebo. SDT will be determined on a per participant basis. Placebo will be administered orally, once daily until completion of Part 1. |
| (Part 2) Elenestinib Dose 1 + SDT | EXPERIMENTAL | Participants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily for approximately 48 weeks. |
| (Part 2) Placebo + SDT | PLACEBO_COMPARATOR | Participants will receive SDT and matching placebo. SDT will be determined on a per participant basis. Placebo will be administered orally, once daily for approximately 48 weeks. |
| (Part 3) Elenestinib + SDT | EXPERIMENTAL | Participants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily for up to approximately 5 years. |
| (Part S) Elenestinib Dose 1 + SDT | EXPERIMENTAL | Participants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily for up to approximately 5 years. |
| (Part K) Elenestinib Dose 1 + SDT | EXPERIMENTAL | Participants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily for up to approximately 5 years. |
| (PK groups) Elenestinib + SDT | EXPERIMENTAL | Participants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily for up to approximately 5 years. |
| Name | Type | Description |
|---|---|---|
| Elenestinib | DRUG | Elenestinib oral tablet |
| Placebo | DRUG | Placebo oral tablet |
Key Inclusion Criteria: All Participants: -Participant must have an Eastern Cooperative Oncology Group Performance Status (ECOG PS) of 0 to 2. Part 1 and PK groups: * Participant has confirmed diagnosis of ISM, confirmed by Central Pathology Review * Participant must have failed to achieve adequ...
Elenestinib is an investigational small molecule being studied for the treatment of indolent systemic mastocytosis, a form of mast cell disease. It is also being evaluated in smoldering systemic mastocytosis. It is not yet approved for any indication and remains in clinical development.
Elenestinib targets KIT, a receptor kinase. It belongs to the -tinib kinase inhibitor class. By inhibiting KIT, the drug is designed to act on the signaling that drives mast cell activity in indolent systemic mastocytosis.
Elenestinib is being developed by Blueprint Medicines Corporation, which trades on the Nasdaq under the ticker BPMC. The company is the sponsor of the clinical program evaluating the drug in systemic mastocytosis.
Elenestinib is in Phase 2 clinical development. It is an investigational drug and has not been approved by the FDA. The ongoing Phase 2 study is evaluating its efficacy and safety in patients with indolent and smoldering systemic mastocytosis.
Elenestinib is being studied in the HARBOR trial, registered as NCT04910685. This Phase 2 study compares the drug with placebo in patients with indolent systemic mastocytosis and smoldering systemic mastocytosis. It is currently recruiting and plans to enroll 534 participants across multiple countries.
The HARBOR trial enrolls adults aged 18 years and older of any sex. It is a randomized, double-blind, placebo-controlled Phase 2 study in patients with indolent or smoldering systemic mastocytosis. Healthy volunteers are not eligible. Enrollment is taking place across sites in the United States, Europe, and other regions.