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
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.
Detailed Summary: The total duration for GD1 patients is 45 days while for GD3 patients, the total duration is up to 61 weeks.
Sponsor: Genzyme, a Sanofi Company
Current Primary Outcome:
- Number of patients with adverse events [ Time Frame: From screening through week 52 ]
- Change from baseline in biomarker levels [ Time Frame: From screening through week 52 ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Assessment of pharmacokinetic parameter: Plasma maximum concentration (Cmax) [ Time Frame: Day 1, Week 4, Week 26, and Week 52 ]
- Assessment of pharmacokinetic parameter: Time at Cmax (Tmax) [ Time Frame: Day 1, Week 4, Week 26, and Week 52 ]
- Assessment of pharmacokinetic parameter: Area under the curve (AUC) [ Time Frame: Day 1, Week 4, Week 26, and Week 52 ]
Original Secondary Outcome: Same as current
Information By: Sanofi
Dates:
Date Received: July 20, 2016
Date Started: January 4, 2017
Date Completion: October 2022
Last Updated: May 10, 2017
Last Verified: May 2017