NCT02228460

Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-naïve Adult Male Patients With Fabry Disease

A Phase 2 Study to Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Enzyme Replacement Therapy (ERT) Treatment-naïve Adult Male Patients Diagnosed With Fabry Disease

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 11 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: Male
• Age: 18 Years – 49 Years
• Healthy volunteers: Not accepted

Summary

Primary Objective: To assess the safety, pharmacokinetics (PK), pharmacodynamics (PD), and exploratory efficacy of GZ/SAR402671 in enzyme replacement therapy treatment-naïve adult male participants diagnosed with Fabry disease.

Detailed description

The total duration of study per participant was 7 to 8 months for participants who entered a planned extension study and approximately 13 to 14 months for participants who did not enter a planned extension study. A 2-year extension study was planned for eligible participants.

Conditions

• Fabry Disease

Interventions

Type	Name	Description
DRUG	GZ/SAR402671	Pharmaceutical form: Capsule; Route of administration: Oral

Timeline

• Start date: 2014-11-01
• Primary completion: 2016-09-01
• Completion: 2016-09-01
• First posted: 2014-08-29
• Last updated: 2019-12-17
• Results posted: 2019-12-17

Locations

8 sites across 5 countries: United States, France, Poland, Russia, United Kingdom

Regulatory

• FDA-regulated drug study