EUCTR2016-001447-39-RO进行中（未招募）1 期
A 24-week with possible extension, prospective, multicenter, randomized, double blind, placebo-controlled, 2-parallel group with a randomization 1:1, phase 3 study to compare efficacy and safety of masitinib to placebo in treatment of patients with Smouldering or Indolent Severe Systemic mastocytosis with handicap, unresponsive to optimal symptomatic treatment
AB Science0 个研究点目标入组 140 人开始时间: 2020年3月20日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 140
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
- •1.Patients with one of the following documented mastocytosis:
- •?Smouldering Systemic Mastocytosis
- •?Indolent Systemic Mastocytosis
- •The retained classification of systemic mastocytosis is based on an excess of mast cells or a presence of abnormal mast cells in at least two organs.
- •The criteria used will be as follows:
- •i.Bone marrow biopsy and/or aspirate associated with at least a sign of abnormality of mast cells:
- •Signs of abnormality of mast cells are:
- •a)Abnormal aggregates of mast cells in a sample in bone marrow:
- •The criteria is deemed satisfied if the aggregate i) is quantified and is strictly above 15 mast cells per aggregated (corresponding to WHO major criterion), or ii) is not quantified but is described as nodule, seat, cluster, focus, or granuloma and therefore pathological
- •b)=25% atypical mast cells in a sample of bone marrow (corresponding to WHO minor criterion)
- •c)c-Kit point mutation at codon 816 in bone marrow (corresponding to WHO minor criterion)
- •d)Abnormal mast cells in the sample of bone marrow while microscopic testing that can be described by the following words: Spindled; Abnormal; Atypical; Fusiform; Dystrophic; Pathologic; Dysmorphic (corresponding to WHO minor criterion)
- •e)Abnormal immunohistochemistry signs: mast cells in bone marrow express CD2 or/and CD25 present (corresponding to WHO minor criterion)
- •f)Abnormal infiltration of mast cells in the bone marrow
- •The criteria is deemed satisfied if the infiltration i) is quantified and is strictly above 3% in the biopsy, or ii) is not quantified but is abnormal as described with infiltration, contingent of mast cells, or proliferation and therefore pathological.
- •ii.Detection of c-kit 816 in the bone marrow without evidence of mast cells in bone marrow but with evidence of c-Kit 816 in skin, justifying clonality
- •iii.Excess of mast cells in digestive organs
- •2.Patient with documented treatment failure of his/her handicap(s) with at least one of the following therapy used at optimized dose (Table S1):
- •?Proton pump inhibitor
- •?Osteoclast inhibitor
- •?Cromoglycate Sodium
- •?Antileukotriene
- •3.Handicapped status defined as at least one of the following handicaps:
- •Pruritus score = 9
- •Number of flushes per week = 8
- •Hamilton rating scale for depression(HAMD-17) score = 19
- •4.Patients with adequate organ function:
- •-Absolute neutrophils count (ANC) = 2.0 x 109/L,
- •-Haemoglobin = 10 g/dL
- •-Platelets (PLT) = 100 x 109/L
- •-AST and ALT = 3x ULN
- •-Bilirubin = 1.5x ULN
- •-Creatinine clearance > 60mL/min (Cockcroft and Gault formula)
- •-Albuminemia >1 x LLN
- •-Proteinuria at screening < 30 mg/mL (1+) on the dipstick. If proteinuria is > 1+ on the dipstick, 24 hours proteinuria must be < 1.5g/24 hours
- •5.Male or female patient aged 18 to 75 years, weight > 41 kg and BMI between 18 and 35 kg/m2
- •6.Contraception:
- •Female patient of childbearing potential (entering the study after a menstrual period and who has a negative pregnancy test), who agrees to use a highly effective method of contraception and an acceptable method of contraception by her male partner during the study and for 3 months after the last treatment intake.
- •Male patient with a female partner of childbearing potential who agrees to use a highly effective method of contraception and an acceptable method of contraception by his female partner during the study and for 3 months after the last treatment intake or who agrees to use an acceptable method of contraception a
排除标准
- •1.Previous treatment with any Tyrosine Kinase Inhibitor.
- •2.Patient presenting with cardiac disorders defined by at least one of the following conditions:
- •Patient with recent cardiac history (within 6 months) of:
- •-Acute coronary syndrome
- •-Acute heart failure (class III or IV of the NYHA classification)
- •-Significant ventricular arrhythmia (persistent ventricular tachycardia, ventricular fibrillation, resuscitated sudden death)
- •Patient with cardiac failure class III or IV of the NYHA classification
- •Patient with severe conduction disorders which are not prevented by permanent pacing (atrio-ventricular block 2 and 3, sino-atrial block)
- •Syncope without known aetiology within 3 months
- •Uncontrolled severe hypertension, according to the judgment of the investigator, or symptomatic hypertension
- •3.Patient who had major surgery within 2 weeks prior to screening visit
- •4.Patient presenting with edema of cardiac origin and left ventricular ejection fraction = 50%
- •5.Patient with chronic diarrhea defined as an episode that lasts longer than 14 days.
- •6.Vulnerable population defined as:
- •Life expectancy < 6 months
- •Patient with < 5 years free of malignancy, except treated basal cell skin cancer or cervical carcinoma in situ
- •Patient with any severe and/or uncontrolled medical condition
- •Patient with known diagnosis of human immunodeficiency virus (HIV) infection
- •7.Patient with history of poor compliance or history of drug/alcohol abuse, or excessive alcohol beverage consumption that would interfere with the ability to comply with the study protocol, or current or past psychiatric disease that might interfere with the ability to comply with the study protocol or give informed consent according to the judgment of the investigator , or institutionalized by court decision
- •8.Patient with any condition that the physician judges could be detrimental to patients participating in this study; including any clinically important deviations from normal clinical laboratory values or concurrent medical events
- •Previous treatment
- •9.Change in the symptomatic treatment of systemic mastocytosis, including the systemic corticosteroids,or administration of any new treatment for systemic mastocytosis (including any Tyrosine Kinase Inhibitor) within 4 weeks prior to baseline
- •10.Treatment with any investigational agent within 4 weeks prior to baseline
研究者
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