A multicenter, double-blind, randomized, placebo-controlled, parallel-group study to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease. - MODIFY

Idorsia Pharmaceuticals Ltd0 个研究点目标入组 108 人开始时间: 2020年5月25日最近更新: 2020年11月30日

适应症

试验速览

暂无简介。

•1. Signed and dated ICF prior to any study-mandated procedure.
•2. Male or female subjects; 18-years old and above.
•3. FD diagnosis confirmed with local genetic test results (i.e., presence of at least 1 mutation in GLA, the gene coding for alfa-
•galactosidase A).
•4. Fabry-associated neuropathic pain, as defined by the subject, in the last 3 months prior to screening.
•Are the trial subjects under 18? no
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 97
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 11

•1. Pregnant, planning to be become pregnant up to 30 days after study treatment discontinuation or lactating subject;
•2. Severe renal insufficiency defined as an estimated glomerular filtration rate (eGFR) per the Chronic Kidney Disease Epidemiology Collaboration creatinine equation < 30 mL/min/1.73 m2 at screening;
•3. Subject on regular dialysis for the treatment of chronic kidney disease;
•4. Subject has undergone, or is on a waiting list for, or is scheduled to undergo kidney or other organ transplantation;
•5. Known and documented transient ischemic attack, stroke, unstable angina or myocardial infarction within 6 months prior to screening;
•6. Clinically significant unstable cardiac disease in the opinion of the investigator (e.g., uncontrolled symptomatic arrhythmia, New York Heart Association class III or IV congestive heart failure);
•7. Any other subject at high risk for developing clinical signs of organ involvement within the time period of the study, as per investigator judgment;
•8. Subject planned for imminent initiation of treatment with ERT.

发起方