NCT01266369已完成2 期
A 12-week With Possible Extension, Prospective, Multicenter, Randomized, Open-label, 2-parallel Group, Phase IIa Study to Compare Efficacy and Safety of AB1010 at 3 or 6 mg/kg/Day in Treatment of Patients With Mastocytosis With Handicap and Bearing Activating Point Mutations in the Phosphotransferase Domain of c-Kit Such as the Main Mutation Asp-816-Val (D816V)
AB Science0 个研究点目标入组 21 人开始时间: 2007年2月最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 21
- 主要终点
- efficacy on handicaps
研究概览
简要总结
The objective is to compare efficacy and safety of AB1010 at 3 or 6 mg/kg/day in treatment of patients with mastocytosis with handicap and bearing activating point mutations in the phosphotransferase domain of c-Kit such as the main mutation Asp-816-Val (D816V).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with one of the following documented mastocytosis:
- •Smouldering systemic mastocytosis
- •Indolent systemic mastocytosis with organomegaly
- •Indolent Systemic Mastocytosis having 2 infiltrated organs (skin and bone-marrow)
- •Any mastocytosis with in the last 6 months at least 3 anaphylactic shocks or syncops requiring either use of adrenaline or medical assistance
- •Cutaneous Mastocytosis (CM)
- •Skin biopsy-documented mastocytosis and evaluable disease based upon:
- •Histological criteria: typical infiltrates of mast cells in a multifocal or diffuse pattern in skin biopsy
- •Clinical criteria: typical skin lesions (maculopapular, urticaria pigmentosa, mastocytoma)
- •Missing data (c-kit molecular analysis not done) or documented presence of an activating point mutation in the phosphotransferase domain of c-kit such as D816V c-kit mutation in at least one infiltrated organ (bone marrow or skin)
- •Refractory to at least one of the symptomatic treatments such as:
- •Proton pump inhibitor
- •Osteoclast inhibitor
- •Cromoglycate Sodium
- •Antileukotriene
- •Other therapies used for the symptomatic care
- •Handicap defined as at least one of the following handicaps:
- •pruritus score ≥ 6
- •number of flushes per week ≥ 7
- •number of stools per day ≥ 4 ,
- •number of mictions per day ≥ 8 ,
- •QLQ-C30 score ≥ 60,
- •Hamilton score ≥ 10
排除标准
- •Patients with one of the following mastocytosis:
- •Systemic Mastocytosis with an Associated clonal Hematologic Non Mast cell lineage Disease (SM-AHNMD)
- •Mast cell leukemia (MCL)
- •Aggressive systemic mastocytosis (ASM)
- •Patient with a major surgery within 2 weeks prior to study entry
- •No vulnerable population will be included in this study
- •Life expectancy < 6 months.
- •Patient is < 5 years free of malignancy, except treated basal cell skin cancer or cervical carcinoma in situ.
- •Patient with grade III/IV cardiac problems as defined by the New York Heart Association Criteria. (i.e., congestive heart failure, myocardial infarction within 6 months of study)
- •Patient has a severe and/or uncontrolled medical disease.
- •Patient has a known diagnosis of human immunodeficiency virus (HIV) infection
研究组 & 干预措施
masitinib 3 mg/kg/day
Experimental
masitinib 3 mg/kg/day
干预措施: masitinib (Drug)
masitinib 6 mg/kg/day
Experimental
masitinib 6 mg/kg/day
干预措施: masitinib (Drug)
结局指标
主要结局
efficacy on handicaps
时间窗: week 12
Pruritus score at week 12 Number of flushes per week at week 12 Hamilton score at week 12 Fatigue Impact scale at week 12
次要结局
未报告次要终点
研究者
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