EUCTR2008-000280-42-IT进行中（未招募）1 期
A Single Arm, Phase II, Open-Label Study to Determine the Efficacy of 100mg Twice Daily Oral Dosing of Midostaurin Administered to Patients with Aggressive Systemic Mastocytosis or Mast Cell Leukemia +/- an Associated Hematological Clonal Non-Mast Cell Lineage Disease. - ND
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 116
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •Aged >18 and ≤ 70 years of age Patient must give written informed consent ECOG performance status of 0-3 Life expectancy > 12 weeks ECG QTc interval ≤ 450 ms Meeting the following laboratory values: AST and ALT ≤ 2.5 x Upper Limit of Normal (ULN), if caused by ASM/MCL ≤ 5 x ULN Serum Bilirubin ≤ 1.5 x ULN, if related to ASM/MCL ≤ 3 x ULN Serum Creatinine ≤ 2.0 mg/dL Diagnosed with aggressive systemic mastocytosis (ASM) or mast cell leukemia (MCL) according to the WHO criteria for SM plus the established criteria for ASM or MCL documented by: Diagnosis of SM will be based: either on the presence of one major plus at least one minor criterion, or of at least 3 minor criteria: Major criterion: Multifocal dense infiltrates of mast cells (>15 mast cells in aggregates) in bone marrow biopsies and/or in sections of other extracutaneous organ(s). Minor criteria: >25% of all mast cells are atypical cells (type I or type II) on bone marrow smears or are spindle-shaped in mast cell infiltrates detected on sections of visceral organs c-kit point mutation at codon 816 in the bone marrow or another extracutaneous organ Mast cells in bone marrow or blood or another extracutaneous organ express CD2 or/and CD25 Baseline serum tryptase concentration > 20 ng/ml (in the case of an associated myeloid neoplasm, is not valid as an SM criterion) Additionally, patients with ASM and MCL also have to present with one or more of the following measurable C-Findings*: ANC<1.0 x 109/L or Hgb < 10g/L or platelets <100 x109/L Hepatomegaly with impaired liver function - i.e. elevated transaminases and/or bilirubin levels and/or hypoalbuminemia (with or without ascites or portal hypertension) Palpable splenomegaly with hypersplenism (e.g. as documented by thrombocytopenia i.e. platelets < 100,000/ μL) Malabsorption with hypoalbuminemia and/or significant weight loss defined as >10% weight loss over the last 6 months *Note: Patients presenting with only non-evaluable C-Findings such as skeletal lesions or hepatomegaly only with ascites or portal hypertension, are not permitted to be included into the study. CFindings need to be documented to result from local aggressive mast cell infiltrates i.e., any other cause/disease must be ruled out For patients with MCL the following criterion has to be met (in addition to the SM and ASM criteria as mentioned above): Bone marrow aspirate smears show 20% or more immature mast cells Typically, there is also an elevated percentage of mast cells in the blood.
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Patients unwilling or unable to comply with the protocol. Any other concurrent severe known disease (except carcinoma in-situ) concurrent severe and/or uncontrolled medical condition (e.g.uncontrolled diabetes, cardiovascular disease including congestive heart failure, myocardial infarction within previous 6 months and poorly controlled hypertension, chronic renal disease, or active uncontrolled infection) which could compromise participation in the study. Confirmed diagnosis of HIV infection or active viral hepatitis. Female patients who are pregnant or breast-feeding or adults of reproductive potential not employing an effective method of birth control. Barrier contraceptives must be used throughout the study in both sexes for fertile patients. Women of childbearing potential must have a negative serum pregnancy test 48 hours prior to administration of midostaurin. Women considered not of childbearing potential include any of the following: no menses for at least 2 years or menses within 2 years but amenorrheic for at least 6 months and luteinizing hormone (LH) and follicular stimulating hormone (FSH) values within normal postmenopausal range (according to definition of postmenopausal for laboratory used) or bilateral oophorectomy or radiation castration and amenorrheic for at least 6 months. Patients who have demonstrated relapse to more than two prior regimen of SM treatment - regardless of treatment regimen for supportive care (e.g. symptom limiting therapies). Patients who have received any investigational agent, chemotherapy, interferon-α, or 2-chlorodeoxyadenosine (2-CdA, cladribine) within 30 days prior to Day 1. Patients who have aggressive systemic mastocytosis with eosinophilia and known positivity for the FIP1L1-PDGFRα fusion unless they have demonstrated relapse or disease progression on prior imatinib therapy. Patients on imatinib and known to be KIT D816V negative unless they have demonstrated relapse, resistance or intolerance to imatinib. Patients who have received any treatment of midostaurin prior to study entry. Patients who have received hematopoietic growth factor support within 14 days of Day 1 of midostaurin treatment. Patients who have had any surgical procedure, excluding central venous catheter placement or other minor procedures (e.g. skin biopsy) within 14 days of Day 1. Patients with any pulmonary infiltrate including those suspected to be of infectious origin. In particular, patients with resolution of clinical symptoms of pulmonary infection but with residual pulmonary infiltrates on chest x-ray are not eligible until pulmonary infiltrates have completely resolved. Exception: Patients with ASM/MCL +/- AHNMD related pleural effusion as confirmed by the investigator are permitted to enter the study
研究者
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