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)
- Registration Number
- jRCT2091220421
- Lead Sponsor
- Tottori University Hospital, Departrment of Child Neurology
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- Not specified
- Target Recruitment
- Not specified
(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
Study & Design
- Study Type
- Not specified
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method - Glucosylsphingosine concentrations in the cerebrospinal fluid
- Secondary Outcome Measures
Name Time Method