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)

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

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT2091220421

Enrollment

Registered

2019-04-26

Start date

2019-05-07

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Neuronopathic Gaucher disease

Interventions

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 fr

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: (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

Exclusion criteria

Exclusion criteria: (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

Design outcomes

Primary

Measure	Time frame
Glucosylsphingosine concentrations in the cerebrospinal fluid	—

Secondary

Measure	Time frame
Lymphocyte GBA activities, etc.	—

Countries

Japan

Contacts

Public ContactYusuke Endo

Tottori University Hospital

gaucher-tuh@c-ctd.co.jp+81-859-38-6946

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026