Last updated on June 2017

GZ/SAR402671 in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3


Brief description of study

Primary Objective: Part 1: - Evaluate central nervous system (CNS) biomarkers in adult Gaucher disease (GD) type 3 (GD3) patients that distinguish GD3 from Gaucher disease type 1 (GD1). - Screen adult GD3 patients who qualify for treatment with GZ/SAR402671 in Part 2. Part 2: - Evaluate the safety and tolerability of GZ/SAR402671 in adult GD3 patients. - Evaluate the change in cerebrospinal fluid (CSF) central nervous system (CNS) biomarkers from adult GD3 patients receiving GZ/SAR402671. Secondary Objectives: - Evaluate the pharmacokinetics of GZ/SAR402671 in adult GD3 patients. - Explore the efficacy of GZ/SAR402671 in infiltrative lung disease (IDL) in adult GD3 patients. - Explore the efficacy of GZ/SAR402671 in systemic disease in adult GD3 patients. - Explore the efficacy of GZ/SAR402671 in neurological function and on exploratory CSF biomarkers in adult GD3 patients.

Detailed Study Description

The total duration for GD1 patients is 45 days while for GD3 patients, the total duration is up to 61 weeks.

Clinical Study Identifier: NCT02843035

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Investigational Site Number 840001
Dallas, TX United States
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Mainz, Germany
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London, United Kingdom
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Investigational Site Number 826002
Salford, United Kingdom
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