Phase 2 Midostaurin in Aggressive Systemic Mastocytosis and Mast Cell Leukemia

Phase 2

Completed

• Conditions: Systemic Mastocytosis, Aggressive (ASM); Leukemia, Mast Cell; Hematological Non-mast Cell Lineage Disease (AHNMD)
• Interventions: Drug: Midostaurin

• Registration Number: NCT00233454
• Lead Sponsor: Jason Robert Gotlib

Brief 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.

Study Timeline

• Study Start: 2005-03
• Study First Submitted: 2005-10-03
• Study First Submitted QC: 2005-10-03
• Study First Posted: 2005-10-05
• Primary Completion: 2010-06-18
• Study Completion: 2011-04-16
• Results First Submitted: 2015-06-16
• Results First Submitted QC: 2015-06-16
• Results First Posted: 2015-07-09
• Status Verified: 2018-09
• Last Update Submitted: 2018-09-19
• Last Update Posted: 2018-09-20

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 26

Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Midostaurin	Midostaurin	100 mg midostaurin twice daily as oral capsules

Primary Outcome Measures

Name	Time	Method
Subjects With Clinical Response [Partial Response (PR) + Complete Response (CR)]	2 months	Clinical Response \[PR + CR\] will be assessed after 2 cycles of treatment, with each cycle being 28 days (4 weeks) in length.; Except as otherwise noted, the minimum criteria for PR is improvement by at least 50% from the baseline value towards the indicated value for one or more of the criteria below: BONE MARROW \& BLOOD; * ANC \<1000/uL; * Hb \<10 g/dL; * Platelets \>100,000/uL LIVER; * If hepatomegaly with ascites, decrease in frequency of paracenteses by 50%; * Elevated enzyme levels \> upper limit of normal (ULN); * Hypoalbuminemia \< ULN; * Portal hypertension \> ULN SPLEEN; * If palpable splenomegaly with hypersplenism/thrombocytopenia, hypersplenism markers improved GI TRACT; * If malabsorption with hypoalbuminemia and/or weight loss, albumin improved BONES; * If huge osteolyses or/and severe osteoporosis with pathologic fractures, partial resolution of osteolyses; Subjects with PR or greater continue, those without response discontinue.

Secondary Outcome Measures

Name	Time	Method
Overall Survival (OS)	40 months	Overall survival was assessed as the median duration of survival at the time of data cut-off, and reported with 95% confidence interval.

Trial Locations

Locations (3)

Stanford University School of Medicine; 🇺🇸 Stanford, California, United States

Dana Farber Cancer Institute; 🇺🇸 Boston, Massachusetts, United States

Washington University-St. Louis; 🇺🇸 Saint Louis, Missouri, United States