Phase III comparing Cerezyme/Genz 112638 in Gaucher Type 1 patients.

• Research type

  Research Study

• Full title

  A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have been Stabilized with Cerezyme

• IRAS ID

  25331

• Contact name

  Tim Cox

• Eudract number

  2008-005223-28

• ISRCTN Number

  n/a

• Clinicaltrials.gov Identifier

  n/a

• Research summary

  A phase 3 study looking at the efficacy of Genz-112638 when compared to Cerezyme in Gaucher Type 1 patient who have been previously stabilised on Cerezyme (for at least 3 years). The study is open-label, and patients will be stratified based on their current Cerezyme dose and then randomized in a 2:1 ratio to receive Genz-112638: Cerezyme. After a 4 week screening period, participants will enter a primary analysis treatment period of 39 weeks. Following this, participants will continue to be treated until study withdrawal or until Genz-112628 becomes commercially available. Approx 96 patients globally will be recruited into the study. Primary efficacy endpoint will be the percentage (%) of patients who remain stable after treatment with Genz-112638 for 39 weeks. Secondary efficacy endpoint will be assessed for the Genz-112638 and Cerezyme treatment groups by calculating the difference in the stability rates at 39 weeks. Safety will be assessed based on adverse events, serious adverse events and changes from baseline through study completion in vital signs, physical examinations, neuro examinations, chest X-rays, bone disease assessments, 24-hour Holter, ECGs and laboratory testing.

• REC name

  East Midlands - Nottingham 2 Research Ethics Committee

• REC reference

  09/H0408/83

• Date of REC Opinion

  16 Nov 2009

• REC opinion

  Further Information Favourable Opinion