An open, phase I/II, investigator initiated trial to evaluate the safety and efficacy of high dose ambroxol in combination with enzyme replacement therapy in Gaucher disease patients

Asan Medical Center0 个研究点目标入组 10 人开始时间: 待定最近更新: 2019年3月11日

适应症

试验速览

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•1) Male and female subjects aged between 2 and 60 years old, inclusive
•2) Patients diagnosed with Gaucher disease by genetic testing or enzyme activity assay
•3) Voluntarily signed written informed consent by patients or by both patients and their representatives

•1) Patients with severe renal impairment (eGFR < 30 mL/min/1.73 m2)
•2) Patients with severe hepatic impairment (Child-Pugh class C)
•3) Patients with genetic predisposition to lactose intolerance, galactose intolerance, Lapp lactase deficiency, or glucose-galactose malabsorption
•4) Patients with known active hepatitis, HIV positive, or other uncontrolled infectious diseases
•5) Patients who are unable to orally administer the study drug
•6) Patients with hypersensitivities to the study drug or any of its components
•7) Pregnant and lactating women, men and women of childbearing potential who are not willing to practice abstinence or use appropriate methods of contraception during the study
•8) Patients who participated in any clinical trials other than GD-AMBX-01 within 90 days from treatment with the investigational product
•9) Patients who are unable to participate in the study for other reasons, according to the investigator

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