Genzyme, a Sanofi Company have launched a phase 2 clinical trial in adults (18 years and older) with Type 3 Gaucher disease. This 52-week two-part study is currently open and recruiting patients in the UK and the US.
To be eligible for this study all participants MUST be on Cerezyme for at least 6 months prior to enrollment, at a stable monthly dose and must continue at the same monthly dose during the study.
The Primary Objective of the study is:
· 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.
· 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.
And 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.
If you are interested in being considered for this study, please contact your Gaucher Consultant.
Further details on the details of this study can be found online at; https://clinicaltrials.gov/ct2/show/study/NCT02843035?term=Gaucher+Disease&rank=50#contacts