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

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

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Official Title: A 52-week Two-part, Open-label, Multicenter, Multinational Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Combination With Cerezyme

Brief Summary:

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.