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

MetaKids, Vriendenloterij0 个研究点目标入组 4 人开始时间: 待定最近更新: 2024年2月28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) All ages
•4) Able to travel to the study site
•5) Patients receive ERT with treatment ongoing at the time of enrollment
•6) There are no sufficient data for the use of ambroxol in pregnant women (see Summary of product characteristics (SPC), section 4.6). This particularly concerns the period up to the 28th week of pregnancy. Postmenarchal female patients must be willing to practice true abstinence in line with their preferred and usual lifestyle, or use a medically accepted form of contraception throughout the study (barrier method such as condom or diaphragm+spermicide or non-barrier method such as oral, injected, or implanted hormonal contraceptive with ethinylestradiol and norethindrone or similar active components

•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) 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, section 4.6)
•5) Pregnancy
•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

MetaKids, Vriendenloterij