Date: 2015-04-21
Type of information: Treatment of the first patient
phase: 2a
Announcement: treatment of the first patient
Company: Genzyme (USA - MA), a Sanofi company (France)
Product: GZ/SAR402671
Action
mechanism: glucosylceramide synthase inhibitor. Fabry disease is a rare lysosomal storage disorder that results in abnormal tissue deposits of a particular fatty substance (called globotriaosylceramide, also referred to as GL-3 or Gb3) throughout the body. GZ/SAR402671 is a glucosylceramide synthase inhibitor that blocks the formation of glucosylceramide (GL-1), a key intermediate in the synthesis of GL-3.
Disease: Fabry disease
Therapeutic area: Rare diseases - Genetic diseases
Country: Czech Republic, France, Poland, Russian Federation, UK, USA
Trial
details: This phase 2 study will 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. (NCT02228460)
Latest
news: * On April 21, 2015, Genzyme, a Sanofi company, announced that patients have begun treatment in a Phase 2a clinical trial focused on evaluating the pharmacodynamics of GZ/SAR402671, a new oral substrate reduction therapy being investigated for the treatment of Fabry disease. The safety, tolerability and pharmacokinetics of GZ/SAR402671 will also be assessed. Genzyme plans to enroll nine treatment-naïve male adult patients with Fabry disease in this international, multicenter study. The Phase 2a trial is an open label, single-arm clinical trial. Patients will receive GZ/SAR402671 orally, once daily. The primary endpoint in the Phase 2a trial will assess the ability of GZ/SAR402671 to reduce accumulated GL-3 from the skin capillary endothelium. Upon completion of the 26 week study, patients will have the option to enroll into an extension study.
