Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients with Fabry Disease
Phase 1
- Conditions
- Fabry diseaseMedDRA version: 20.0 Level: PT Classification code 10016016 Term: Fabry's disease System Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Hormonal diseases [C19]
- Registration Number
- EUCTR2014-004995-49-FR
- Lead Sponsor
- Genzyme Corporation
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 11
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
Patients, in the opinion of the Investigator, unable to adhere to the requirements of the study.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 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.;Timepoint(s) of evaluation of this end point: Through 30 months;<br> Primary end point(s): Characterization of the safety profile of GZ/SAR402671, including the frequency, duration, and severity of adverse events<br> Clinically significant changes in laboratory parameters including hematology, biochemistry, and urinanlysis<br> Clinically significant changes in physical examinations including vital signs and body weight<br>
- Secondary Outcome Measures
Name Time Method <br> Secondary end point(s): Change from baseline in plasma globotriaosylceramide (GL-3)<br> Change from baseline in plasma lyso GL-3<br> Change from baseline in plasma glucosylceramide (GL 1)<br> Urine GL-3 change from baseline<br> ;Timepoint(s) of evaluation of this end point: Through 30 months