NL-OMON51936尚未招募3 期
Effectiveness of ambroxol in children and adults with Gaucher disease 3: n-of-1 series - ATTACK-GD3
适应症
试验速览
- 阶段
- 3 期
- 状态
- 尚未招募
- 入组人数
- 6
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 2 至 99（—）
入选标准
- •1) The patient or the parent(s) / legal guardian(s) must provide written
- •informed consent before start of the study;
- •2) Male and female patients with documented deficiency of GCase activity and
- •GBA genotype fitting GD3;
- •3) 3) Male and female patients > 2 years of age;
- •4) Able to travel to the study site;
- •5) Patients receive ERT with treatment ongoing at the time of enrollment;
排除标准
- •1) The patient is transfusion dependent;
- •2) The patient has received an investigational product within 30 days prior to
- •enrollment;
- •3) Known hypersensitivity reactions, intolerance or adverse reactions to
- •ambroxol or to the inactive ingredients;
- •4) Pregnancy, because there are no sufficient data for the use of ambroxol in
- •pregnant women (see Summary of Product Characteristics (SPC));
- •5) The patient is lactating. Ambroxol crosses into the breast milk. As there is
- •no adequate experience in humans to date, ambroxol should not be used in
- •lactation in a study setting (see SPC);
- •6) The patient is unwilling or, in the investigator*s opinion, unable to adhere
- •to the requirements of the study;
- •7) The patient is unable to swallow powder and has no other enteral access
- •(e.g. gastrostomy);
- •8) Any condition or abnormality which may, in the opinion of the investigator,
- •compromise the safety of patients.
研究者
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