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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)

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)

6

Aya Narita

Tottori University Hospital

36-1 Nishi-cho, Yonago, Tottori 683-8504, Japan

aya.luce@nifty.com

Yusuke Endo

Tottori University Hospital

36-1 Nishi-cho, Yonago, Tottori 683-8504, Japan

+81-859-38-6946

gaucher-tuh@c-ctd.co.jp

COMPLETED

May. 07, 2019

Interventional

Single arm

open(masking not used)

No

2-3

(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

Both

Neuronopathic Gaucher disease

Intervention type:DRUG Name of intervention:ADMINISTRATION Dose form / Japanese Medical Device Nomenclature:TABLET Route of administration / Site of application:ORAL Dose per administration: Dosing frequency / Frequency of use: Planned duration of intervention:52 weeks Intended dose regimen: detailes of teratment arms: Comparative intervention name: Dose form / Japanese Medical Device Nomenclature: Route of administration / Site of application: Dose per administration: Dosing frequency / Frequency of use: Planned duration of intervention: Intended dose regimen:

Glucosylsphingosine concentrations in the cerebrospinal fluid

Lymphocyte GBA activities, etc.

Feb. 28, 2019