An Open, Phase I/II, Investigator Initiated Trial to Evaluate the Safety and Efficacy and to Extend the Evaluation Period 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: 2024年5月27日

适应症

试验速览

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•1) Male and female subjects aged between 2 and 60 years old
•2) Patients diagnosed with Gaucher disease by genetic testing or enzyme activity assay
•(Patients who meet any of the following criteria are eligible to be included in this study.)
•A.New patients receiving Ambroxol for the first time (only applicable to Part A)
•B.Existing patients who participated Study GD-AMBX-02 or patients who completed Part A of this study (only applicable to Part B)
•3) Patients who voluntarily consented to study participation and signed the written consent, or patients who gave voluntary written consent for study participation both directly and from their legally authorized 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 women, breastfeeding women, and women and men of childbearing potential who do not intend to remain abstinent or use appropriate contraceptive methods (oral contraceptive, hormone implant, intrauterine device, combination of spermicide and diaphragm [condom])
•8) Patients who participated in any clinical trials other than GD-AMBX-02 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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