Study of Elenestinib (BLU-263) for Advanced Systemic Mastocytosis and Other KIT Altered Blood Cancers

Sponsor: Blueprint Medicines Corporation

Sponsor score: 40
Inactive

ClinicalTrials ID:NCT05609942

Provider View

This clinical trial is testing a new drug, elenestinib (BLU-263), to see if it is safe and effective for treating Advanced Systemic Mastocytosis (AdvSM) and other blood cancers with KIT gene changes. The study will also explore combining elenestinib with another drug, azacitidine, to see if this combination works better.

Patient Parameters

Program Overview

The purpose of this study is to evaluate the safety and effectiveness of a new drug called elenestinib (BLU-263) in people with Advanced Systemic Mastocytosis (AdvSM) and other blood cancers that have changes in the KIT gene. The study will also test if combining elenestinib with another drug, azacitidine, is beneficial. Participants will be involved in the study for about 4 years, with 2 years of treatment and 2 years of follow-up. Initially, participants will have monthly visits for the first six months, then every three months for the rest of the study.

Description

Systemic mastocytosis is a condition that includes several types, such as Indolent SM, SM with an associated blood cancer (SM-AHN), aggressive SM, and mast cell leukemia (MCL). In 2016, the World Health Organization recognized a subtype called smoldering SM as a distinct type. Advanced SM (AdvSM) includes aggressive SM, SM-AHN, and MCL. This study focuses on these advanced types and other blood cancers with KIT gene changes.

Eligibility Criteria

Exclusion Criteria

  • Diagnosis of a specific type of cancer called Philadelphia chromosome positive cancer.
  • Acute myeloid leukemia.
  • Taking corticosteroids without a stable dose for at least 7 days.
  • Received any cancer treatment or experimental drug within 14 days before joining the study.
  • Used hydroxyurea within 7 days before starting elenestinib.
  • Previously treated with azacitidine or similar drugs for the current diagnosis.
  • Not eligible for a stem cell transplant.
  • Had radiation therapy within 14 days of a bone marrow biopsy.
  • Used certain growth factors within 14 days of a bone marrow biopsy, except for stable erythropoietin use.
  • Previously used more than one selective KIT inhibitor.
  • Certain abnormal lab results, such as high liver enzymes or low kidney function.
  • Major surgery within 14 days before starting the study drug.
  • Another primary cancer diagnosed or treated within 1 year before starting the study drug, except for certain skin cancers and localized prostate cancer.
  • Heart issues like prolonged QT interval or significant heart disease.

For Monotherapy Arm:

  • High-risk myelodysplastic syndrome (MDS).
  • A type of blood cancer with a high percentage of blasts.
  • Low platelet count or recent platelet transfusions.

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