Effectiveness of ambroxol in children and adults with Gaucher disease 3: n-of-1 series - ATTACK-GD3

Amsterdam UMC0 个研究点目标入组 4 人开始时间: 2022年7月30日最近更新: 2023年1月9日

试验速览

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•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)Male and female patients of all ages;
•4)Able to travel to the study site;
•5)Patients receive ERT with treatment ongoing at the time of enrollment;
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 6
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 1
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•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.

发起方

Amsterdam UMC