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Clinical Trials/JPRN-jRCT2091220421
JPRN-jRCT2091220421
Completed
Phase 2

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 sites6 target enrollmentApril 26, 2019
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Conditionseuronopathic Gaucher disease

Overview

Phase
Phase 2
Intervention
Not specified
Conditions
euronopathic Gaucher disease
Sponsor
Tottori University Hospital, Departrment of Child Neurology
Enrollment
6
Status
Completed
Last Updated
2 years ago
OverviewInvestigatorsEligibility CriteriaOutcomesSimilar Trials

Overview

Brief Summary

No summary available.

Registry
who.int
Start Date
April 26, 2019
End Date
January 25, 2021
Last Updated
2 years ago
Study Type
Interventional
Sex
All

Investigators

Sponsor
Tottori University Hospital, Departrment of Child Neurology

Eligibility 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

  • •(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

Outcomes

Primary Outcomes

Not specified

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