NCT05609942

Study of Elenestinib (BLU-263) in Advanced Systemic Mastocytosis (AdvSM) and and Other KIT Altered Hematologic Malignancies

A Phase 1/2, Open-label, 2-arm Study Evaluating BLU-263 as Monotherapy and in Combination With Azacitidine, in Patients With KIT Altered Hematologic Malignancies

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 9 (actual)
• Sponsor: Blueprint Medicines Corporation · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The goal of this clinical trial is to evaluate elenestinib (BLU-263) in participants with Advanced Systemic Mastocytosis (AdvSM), SM with an associated hematologic neoplasm (SM-AHN), and other hematologic malignancies. The main questions it aims to answer are: * Determine Recommended Dose of elenestinib (BLU-263) monotherapy for participants with AdvSM * Safety and tolerability of elenestinib (BLU-263) monotherapy * Efficacy of elenestinib (BLU-263) monotherapy in participants with AdvSM * Determine Recommended Dose of elenestinib (BLU-263) in combination with azacitidine in participants with AdvSM * Safety and tolerability of elenestinib (BLU-263) in combination with azacitidine * Efficacy of elenestinib (BLU-263) in combination with azacitidine in participants with AdvSM The estimated study duration for each participant will be approximately 4 years: 2 years of treatment followed by 2 years of follow-up. Participants may be required to attend monthly visits for the first six months, followed by quarterly visits for the remainder of the study.

Detailed description

Systemic mastocytosis includes five major subtypes: Indolent SM (ISM), SM with an associated hematologic neoplasm (SM-AHN), aggressive SM (ASM), and MC leukemia (MCL). In 2016, the smoldering subtype of SM, a former provisional ISM subvariant, was designated as a distinct variant of SM by the World Health Organization (WHO). Aggressive SM, SM-AHN, and MCL together are referred to as Advanced SM (AdvSM).

Conditions

• Advanced Systemic Mastocytosis

Interventions

Type	Name	Description
DRUG	BLU-263	BLU-263 Oral Tablets
DRUG	Azacitidine	Azacitidine powder for suspension for intravenous infusion / subcutaneous injection

Timeline

• Start date: 2023-09-25
• Primary completion: 2025-01-14
• Completion: 2025-03-10
• First posted: 2022-11-08
• Last updated: 2025-04-10

Locations

11 sites across 7 countries: United States, Belgium, France, Germany, Netherlands, Norway, Spain

Regulatory

• FDA-regulated drug study