Phase II/III Study to Evaluate the Efficacy and Safety of Chaperone Therapy with Ambroxol Hydrochloride (JT408T) in Patients with Neuronopathic Gaucher disease (Japan- Lysosomal Optimization Study: J-LO study) (J-LO study)

Tottori University Hospital, Departrment of Child Neurology0 个研究点开始时间: 待定最近更新: 2022年6月20日

试验速览

暂无简介。

•(1) Patients with decreased GBA activity in the lymphocyte or cultured dermal fibroblasts, genetically diagnosed with Gaucher's disease
•(2) Patients with neurological symptoms
•(3) Patients who or whose legally acceptable representatives gave voluntary written consent to participate in this study, after receiving sufficient explanations and fully understanding the explanations

•(1) Patients with hypersensitive to ABX or excipients
•(2) Patients with serious hepatic, renal, or cardiovascular disorders to be considered by the investigator or subinvestigator to be ineligible
•(3) Patients showing total bilirubin, ALT, AST, or creatinine levels exceeding 3 times the facility reference value in the laboratory test, within 4 weeks before enrollment
•(4) Patients who are pregnant or nursing or who desire to become pregnant by 3 months after the completion of the treatment with the investigational product. Patients who or whose partners are unwilling to agree to practice appropriate double barrier contraception during the study period
•(5) Patients who participated in any clinical study of the chaperone therapy using ABX in the past
•(6) Patients who participated in other clinical trials within 3 months before enrollment
•(7) Patients considered by the investigator or subinvestigator to be ineligible for the study

Glucosylsphingosine concentrations in the cerebrospinal fluid

未报告次要终点

发起方

Tottori University Hospital, Departrment of Child Neurology