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

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2014-004995-49-PL

Enrollment

Registered

2015-06-15

Start date

2015-07-28

Completion date

Unknown

Last updated

2020-02-01

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Fabry disease MedDRA version: 18.0 Level: PT Classification code 10016016 Term: Fabry's disease System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Product Code: SAR402671, GZ402671 or GZ/SAR402671 Pharmaceutical Form: Capsule CAS Number: 1401090-53-6 Current Sponsor code: SAR402671 / GZ402671

Eligibility

Sex/Gender

Male

Inclusion criteria

Inclusion criteria: Male patient with Fabry disease who previously completed study ACT13739. Patients, willing and able to provide signed informed consent. Sexually active patients, willing to practice true abstinence in line with their preferred and usual lifestyle or using two acceptable effective methods of contraception. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 8 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: Patients, in the opinion of the Investigator, unable to adhere to the requirements of the study.

Design outcomes

Primary

Measure	Time frame
Main Objective: To assess the long-term safety of GZ/SAR402671 in adult male patients with Fabry disease who previously completed study ACT13739;Secondary Objective: To assess the long-term effect of GZ/SAR402671 on pharmacodynamic and exploratory efficacy endpoints in adult male patients with Fabry disease who previously completed study ACT13739.; Primary end point(s): Characterization of the safety profile of GZ/SAR402671, including the frequency, duration, and severity of adverse events Clinically significant changes in laboratory parameters including hematology, biochemistry, and urinanlysis Clinically significant changes in physical examinations including vital signs and body weight ;Timepoint(s) of evaluation of this end point: Through 30 months	—

Measure

Time frame

Main Objective: To assess the long-term safety of GZ/SAR402671 in adult male patients with Fabry disease who previously completed study ACT13739;Secondary Objective: To assess the long-term effect of GZ/SAR402671 on pharmacodynamic and exploratory efficacy endpoints in adult male patients with Fabry disease who previously completed study ACT13739.; Primary end point(s): Characterization of the safety profile of GZ/SAR402671, including the frequency, duration, and severity of adverse events Clinically significant changes in laboratory parameters including hematology, biochemistry, and urinanlysis Clinically significant changes in physical examinations including vital signs and body weight ;Timepoint(s) of evaluation of this end point: Through 30 months

—

Secondary

Measure	Time frame
Secondary end point(s): Change from baseline in plasma globotriaosylceramide (GL-3) Change from baseline in plasma lyso GL-3 Change from baseline in plasma glucosylceramide (GL 1) Urine GL-3 change from baseline ;Timepoint(s) of evaluation of this end point: Through 30 months	—

Countries

Czech Republic, France, Poland, Russian Federation, United Kingdom, United States

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026