FABRY SRT MONO

• Research type

  Research Study

• Full title

  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

• IRAS ID

  159119

• Contact name

  Patrick Deegan

• Contact email

  patrick.deegan@addenbrookes.nhs.uk

• Sponsor organisation

  Genzyme Corporation

• Eudract number

  2013-005324-41

• Clinicaltrials.gov Identifier

  U1111-1152-1456, Universal Trial Number

• Research summary

  A phase 2 study to evaluate the safety and efficacy of GZ/SAR402671 in treatment-naïve adult male patients diagnosed with Fabry disease.

  The study will consist of a screening visit(s), treatment period visits and follow-up visits. Patients enrolled in the study will receive GZ/SAR402671, once daily, orally for 26 weeks. Skin biopsies will be obtained at baseline, week12 and week26. Blood and urine samples will be collected. Upon completion of the 26 week study patients will have the option to enroll into a two year extension study.

  Primary endpoint- Change from Baseline in globotriaosylceramide (GL-3) scores as evaluated by light microscopy(LM).

• REC name

  East of England - Cambridge Central Research Ethics Committee

• REC reference

  14/EE/1096

• Date of REC Opinion

  7 Oct 2014

• REC opinion

  Further Information Favourable Opinion