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Phase 2 Midostaurin in Aggressive Systemic Mastocytosis and Mast Cell Leukemia | Clinical Trials | Clareo Health

COMPLETEDPHASE2

Phase 2 Midostaurin in Aggressive Systemic Mastocytosis and Mast Cell Leukemia

A Single Arm, Phase 2, Open-Label Study to Determine the Efficacy of Twice Daily Oral Dosing of PKC412 <Midostaurin> Administered to Patients With Aggressive Systemic Mastocytosis (ASM) and Mast Cell Leukemia (MCL)

NCT00233454•Jason Robert Gotlib

View on ClinicalTrials.gov

Summary

The safety and efficacy of midostaurin (PKC412), a novel investigational drug, will be evaluated on the basis of response rate, when administered to patients with aggressive systemic mastocytosis (ASM) or mast cell leukemia (MCL)

Detailed Description

This study assesses the activity and safety profile of twice-daily oral doses of midostaurin in patients with aggressive systemic mastocytosis (ASM) or mast cell leukemia (MCL) with or without associated clonal hematological non-mast cell lineage disease (AHNMD). Aggressive systemic mastocytosis (ASM) and mast cell leukemia (MCL) are characterized by excessive bone marrow production of mast cells which can can infiltrate tissues and release harmful substances, resulting in organ damage. These diseases have very limited treatment options and poor prognosis. Existing treatments for in advanced mast cell disease, eg, interferon-alpha; corticosteroids; and/or cladribine, exhibit low response rates that are usually partial in nature.

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•At least 18 years of age.
•Karnofsky performance status (KPS) of \> 30% (equivalent to ECOG 0 to 3)
•Mast cell disease, histologically confirmed and documented to be
•* Aggressive systemic mastocytosis (ASM) OR
•* Mast cell leukemia (MCL) meeting the following criteria
•* Meets criteria for systemic mastocytosis
•* Biopsy indicates diffuse infiltration by atypical, immature mast cells
•* Bone marrow aspirate smears show at least 20% mast cells
•Confirmed availability of tissue sample within 6 months prior to entry into study, for evaluation of KIT mutation status of the tumor cells. Subjects who have systemic mastocytosis PLUS eosinophilia AND known positivity for FIP1L1-PDGFR-alpha fusion are eligible only if they have demonstrated relapse or disease progression on prior imatinib therapy
•Blood levels of liver enzymes within normal limits (EXCEPTION: If the sole cause of elevated blood levels of liver enzymes is ASM/MCL, then AST and ALT ≤ 4X upper limit of normal (ULN), and/or bilirubin ≤ 4X ULN)
+10 more criteria

Exclusion Criteria

•Active pulmonary disease based on physical assessment or lateral chest X-ray, considered by the investigator to be unrelated to mastocytosis
•Any pulmonary infiltrate or abnormality on the baseline chest X-ray known to be new in the previous 4 weeks (EXCEPTION: pleural effusion related to systemic mastocytosis, eg, secondary to ascites, AND not causing symptomatic respiratory complaints, may be eligible)
•Cardiovascular disease, including congestive heart failure
•Myocardial infarction within 6 months
•Poorly-controlled hypertension with any Grade 3/4 cardiac problems (per New York Heart Association Criteria)
•Uncontrolled diabetes
•Chronic renal disease
•Active uncontrolled infection
•Known malignant disease involving the central nervous system (CNS)
•Known confirmed diagnosis of HIV infection or active viral hepatitis.
+7 more criteria

Locations (3)

Stanford University School of Medicine

Stanford, California, United States

Dana Farber Cancer Institute

Boston, Massachusetts, United States

Washington University-St. Louis

St Louis, Missouri, United States

Key Dates

Start Date

March 1, 2005

Primary Completion Date

June 18, 2010

Completion Date

April 16, 2011

Last Updated

September 20, 2018

Enrollment

ACTUAL participants

Conditions

Systemic Mastocytosis, Aggressive (ASM)Leukemia, Mast CellHematological Non-mast Cell Lineage Disease (AHNMD)

Interventions

Midostaurin

DRUG