Open label, multicentre study, evaluating the safety, tolerability, efficacy, compliance and the acceptability of alkalising treatments at long-term in patients with cystinuria

Advicenne0 个研究点目标入组 97 人开始时间: 2018年10月9日最近更新: 2022年3月2日

研究概览

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•ADV7103 Cohort :
•Patient who has participated to and completed the previous B12CS Study or B13CS Study.
•SoC Cohort :
•Patient who has a diagnosis of cystinuria based on medical diagnosis (at least one previous or current episode of calculus of cystine, and/or one previous or current episode of cystine crystalluria) or on genetic diagnosis (only for patients of Subset 4).
•Patient treated with an alkalising treatment at a well-adapted dose (defined as a daily dose deemed by the investigator aiming to maintain overtime urinary pH value = 7.0 and/or compatible with an acceptable safety profile and/or patient’s constraints or compliance).
•Patient male or female, including child aged between 6 months and 17 years old and adult aged = 18 years old up to 70 years old.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 42
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 12
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 2

•ADV7103 Cohort :
•Patient who has not participated to B12CS Study or B13CS Study
•Patient for whom any safety issue could contraindicate her/his participation to the extension study.
•Soc Cohort :
•Patient that is receiving the second line therapy -- cystine chelating agents (sulfhydryl compounds).
•Patient who presents kalaemia > 5.0 mmol/L.
•Patient who presents a moderate or severe renal impairment (estimated glomerular filtration rate (eGFR) < 45 mL/min/1.73 m2 according to Schwartz formula for the children and both MDRDs and CKD-EPI for adults).

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