NCT02489344

Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients With Fabry Disease

An Open-label, Multicenter, Multinational Extension Study of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Adult Male Patients Diagnosed With Fabry Disease

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 8 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: Male
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

Primary Objective: To assess the long-term safety of GZ/SAR402671 in adult male participants with Fabry disease who previously completed study ACT13739 (NCT02489344). Secondary Objective: To assess the long-term effect of GZ/SAR402671 on pharmacodynamic and exploratory efficacy endpoints in adult male participants with Fabry disease who previously completed study ACT13739.

Detailed description

The total duration of this extension study (LTS14116) was up to 31 months (30 months of treatment and one month post-treatment follow-up).

Conditions

• Fabry Disease

Interventions

Type	Name	Description
DRUG	GZ/SAR402671	Pharmaceutical form:capsule Route of administration: oral

Timeline

• Start date: 2015-07-07
• Primary completion: 2018-11-20
• Completion: 2018-11-20
• First posted: 2015-07-03
• Last updated: 2021-03-23
• Results posted: 2019-12-09

Locations

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

Regulatory

• FDA-regulated drug study