Clinical Trial: Phase II Midostaurin in Aggressive Systemic Mastocytosis and Mast Cell Leukemia
Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional
Official Title: A Single Arm, Phase II, Open-Label Study to Determine the Efficacy of Twice Daily Oral Dosing of PKC412 <Midostaurin> Administered to Patients With Aggressive Systemic Mastocytosis (ASM) and
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 Summary:
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.
Sponsor: Jason Robert Gotlib
Current Primary Outcome: Subjects With Clinical Response [Partial Response (PR) + Complete Response (CR)] [ Time Frame: 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.
Original Primary Outcome:
Current Secondary Outcome: Overall Survival (OS) [ Time Frame: 11 months ]
Original Secondary Outcome:
Information By: Stanford University
Dates:
Date Received: October 3, 2005
Date Started: March 2005
Date Completion: December 2017
Last Updated: June 16, 2015
Last Verified: June 2015
