NCT05084872Unknown2 期
Hydroxychloroquine in Isolated Cutaneous Mastocytosis Patients or Indolent Systemic Mastocytosis With Associated Skin Involvement Patients: Proof of Concept Study
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Change of mast cell activation symptoms
研究概览
简要总结
The treatment of systemic mastocytosis has two main axes:
- Control of mast cell activation symptoms and
- The control of proliferation (accumulation) of mast cells.
There is no standard treatment and no treatment has a marketing authorization for the treatment of monoclonal indolent mastocytosis.
详细描述
Mastocytosis is an orphan disease related to the accumulation and / or the proliferation of abnormal mast cells in different tissues.
In adults, a classic distinction is made between isolated cutaneous forms (10 to 15%) and systemic forms (85 to 90%).
The treatment of systemic mastocytosis has two main axes:
- Control of mast cell activation symptoms and
- The control of proliferation (accumulation) of mast cells.
There is no standard treatment and no treatment has a marketing authorization for the treatment of monoclonal indolent mastocytosis.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 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
- •Age > 18 years
- •Isolated Cutaneous mastocytosis or indolent systemic mastocytosis with associated skin lesions defined according to WHO criteria (and / or international standards for cutaneous mastocytosis)
- •Patient with at least one disability defined by the presence of the following symptoms assessed as moderate to severe:
- •Cutaneous pruritus with score ≥ 5 on a VAS scale from 0 to 10
- •Number of flushes / week ≥ 7
- •Skin KIT mutation known
- •Performance scale: OMS/ECOG ≤ 1
- •Woman and man of childbearing age* under effective contraception during all the treatment by hydroxychloroquine, until 8 months after its cessation
排除标准
- •Non-symptomatic mastocytosis and / or without skin involvement
- •Advanced Systemic mastocytosis
- •History of ophthalmic disease and / or cardiac conduction disorders, in particular the prolongation of the QT interval as well as the risk factors for prolongation of the QT interval, such as heart disease (heart failure, myocardial infarction), pro-arrhythmic conditions (eg bradycardia <50 bpm), history of ventricular dysrhythmias, uncorrected hypokalemia and / or hypomagnesemia, concomitant treatment with interval prolonging agents QTagainst-indicating the use of hydroxychloroquine
- •Treatment with citalopram, escitalopram, hydroxyzine, domperidone, piperaquine due to the increased risk of ventricular rhythm disorders, especially torsades de pointes
- •Specific anti-tumor treatment (chemotherapy, radiotherapy) of less than 4 weeks before inclusion.
- •Concomitant specific anti-mast cell treatment
- •Contre-indication(s) to XYLOCAINE 10 mg/ml ADRENALINE 0,005 mg/ml, injectable solution: Known hypersensitivity to chlorhydrate de lidocaïne, to amide-type local anaesthetics or one of its excipients (sulfites), patients suffering from recurring porphyrias, coronary insufficiency, ventricular rhythm disorders, severe arterial hypertension, obstructive cardiomyopathy, hyperthyroidism.
- •Inclusion in another trial with an experimental therapeutic molecule
- •Change symptomatic treatment (including dosage) in the 4 weeks preceding the inclusion visit
- •Moderate to severe renal or hepatic failure or diabetes
- •History of organ transplant
- •Inability to give informed consent
- •Inability to undergo medical monitoring for geographical, social or psychic
- •Patients with major surgery scheduled in the next two weeks screening
- •Patient without health insurance
- •Pregnancy, Breastfeeding
- •Vulnerable Patient, defined as:
- •Esperanzae survival < 6 months
- •Patient with another uncontrolled severe disease
- •Patient under juridical protection
研究组 & 干预措施
Treatment
Experimental
Patients will be treated by hydroxychloroquine at a dose of 6 to 6.5mg/kg/day
干预措施: Hydroxychloroquine (Drug)
结局指标
主要结局
Change of mast cell activation symptoms
时间窗: 12 month
The primary endpoint of this study is the change of mast cell activation symptoms as flushes between the start of treatment and 12 months later. The skin flush will be evaluated according to the absolute number of flushes / week at each visit
次要结局
- Difference on mast cell burden - serum tryptase level(12 month)
- Difference on skin mast cell burden - mast cells/mm²(12 month)
- Difference of mast cell activation symptoms : arthralgia(12 month)
- Difference of mast cell activation symptoms : diarrhea(12 month)
- Difference of mast cell activation symptoms : discomfort(12 month)
- The safety of hydroxychloroquine treatment.(12 month)
- Difference of mast cell activation symptoms : pollakiuria(12 month)
- effectiveness of treatment(12 month)
研究者
研究点 (1)
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