Efficacy and Safety of Midostaurin in Patients With Aggressive Systemic Mastocytosis or Mast Cell Leukemia

Phase 2

Completed

Brief Summary: The purpose of this study was to determine the efficacy and safety of twice daily (bid) oral midostaurin in patients with Aggressive Systemic Mastocytosis (ASM) or Mast Cell Leukemia (MCL) with or without an Associated Hematological clonal Non-Mast cell lineage Disease (AHNMD).

Recruitment & Eligibility

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Arm && Interventions

Group	Intervention	Description
Midostaurin (PKC412)	Midostaurin (PKC412)	Midostaurin was administered at a dose of 100 mg twice daily (bid) in continuous cycles of 28 days until disease progression, intolerable toxicity or withdrawal due to any cause, whichever occurred first.

Primary Outcome Measures

Name	Time	Method
Percentage of Participants With Overall Response Rate (ORR)	6 months	Overall Response Rate (ORR) was defined as the percentage of participants who classified as confirmed responders (Major Response (MR) or Partial Response (PR)) by the adjudication of the SSC and based on a Modified Valent Criteria. A major responder had complete resolution of at least one C-Finding and no progression in other C-Findings. A partial responder showed a measurable improvement in one or more C-Finding(s) without confirmed progression in other C-Findings. A C-Finding was a Clinical Finding, which was considered by the investigator and corroborated by the Study Steering Committee (SSC) Chairperson or designee, attributable to the mast cell disease component and not the associated hematological clonal non-mast cell lineage disease (AHNMD) component or any other cause.

Secondary Outcome Measures

Name	Time	Method
Long-term Safety and Tolerability of Midostaurin	Up to 30 days after last dose of study treatment	Analysis of frequencies for treatment emergent Adverse Event (AE), Serious Adverse Event (SAE) and Deaths by primary System Organ Class (SOC)
Median Time to Progression-Free Survival (PFS)	Up 5 years	The Progression-free survival (PFS) is defined as the time from start of treatment to the date of the first documented and confirmed progression or death due to any cause.
Median Time to Overall Survival (OS)	Up 5 years	The Overall Survival (OS) is defined as the time from start of treatment to the date of death due to any cause.
Histopathologic Response	Up 5 years	Histopathologic response was summarized to demonstrate the change from baseline in percentage of mast cell infiltrations in the Bone Marrow (BM) and related serum tryptase levels.
Median Time to Duration of Response (DoR)	Up 5 years	The Duration of response (DoR) was defined as the time from first onset of confirmed response (MR or PR) to the date of first documented and confirmed progression or death due to ASM/MCL.
Median Time to Response (TTR)	Up 5 years	The Time to response (TTR) was defined as the time from start of treatment until the date of onset of confirmed response (MR or PR).

Locations (10): Dana Farber Cancer Institute Hematology / Oncology
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Boston, Massachusetts, United States
University of Michigan Comprehensive Cancer Center DeptofMichiganCancerCenter(3)
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Ann Arbor, Michigan, United States
Georgia Health Sciences University Dept.ofMedicalCollegeOfGeorgia
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Augusta, Georgia, United States
Stanford University Medical Center Stanford University 2
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Stanford, California, United States
University of California at Los Angeles Dept. of Hematology Clinic
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Los Angeles, California, United States
Memorial Sloan Kettering Cancer Center Dept. of MSKCC (2)
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New York, New York, United States
Oregon Health and Science University Dept. Hematologic Malignancies
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Portland, Oregon, United States
University of Pennsylvania
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Philadelphia, Pennsylvania, United States
Virginia Commonwealth University SC
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Richmond, Virginia, United States
Novartis Investigative Site
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London, United Kingdom