Advanced Systemic Mastocytosis Clinical Trial
Official title:
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
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.
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). ;
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|---|---|---|---|
| Active, not recruiting |
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Phase 2 | |
| Completed |
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A Safety, Tolerability and PK Study of DCC-2618 in Patients With Advanced Malignancies
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Phase 1 | |
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Retrospective Study Assessing the Effect of Avapritinib Versus Best Available Therapy in Patients With AdvSM
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