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Phase II/III Ambroxol (JT408T) Chaperone Therapy in Neuronopathic Gaucher Disease (J-LO Study)
jRCT2091220421Phase 3Completed
- Sponsor
- Tottori University Hospital
- Start
- Apr 2019
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 3, open-label, single-arm trial of ambroxol in Gaucher disease (GD), sponsored by Tottori University Hospital. Primary endpoint: Glucosylsphingosine concentration in cerebrospinal fluid. Started Apr 2019; status: completed.
02
Drugs
ambroxol
03
Conditions
Gaucher disease (GD)
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