prGCD - safety and efficacy switchover study in Gaucher patients –V1.0

• Research type

  Research Study

• Full title

  A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease Treated with Imiglucerase (Cerezyme®) Enzyme Replacement Therapy

• IRAS ID

  13278

• Sponsor organisation

  Protalix Biotherapeutics

• Eudract number

  2008-005825-12

• Clinicaltrials.gov Identifier

  NCT00712348

• Research summary

  Gaucher disease is a rare inherited metabolic condition caused by a mutation in a human gene. The clinical signs and symptoms of the disease result in severe anemia, enlarged liver and spleen, bone pain, neurological deterioration and in some cases may even result in death.The annual treatment per patient is approximately US$250 000.The current treatment for Gaucher is life-long enzyme replacement therapy (ERT). Sometimes despite treatment, symptoms are not alleviated, but rather stabilized, such as enlarged spleen, anemia, etc. The objective of this trial is to prove that prGCD is at least as safe and efficacious as the commercially available ERT treatment.prGCD is an ERT produced by biologically engineered plant cells. It has potential advantages over the commercially available ERT, among them prolonged half life, and hopefully significant cost reduction due to the manufacturing process. In this trial, 15 patients treated with imiglucerase (Cerezyme©) with a verified stable disease, will be switched over to prGCD for 38 weeks.This trial will take place in Addenbrooke's Hospital (Cambridge University Hospitals), and in other locations worldwide.The study Sponsor (funder) for this trial is Protalix Biotherapeutics.

• REC name

  East of England - Cambridge Central Research Ethics Committee

• REC reference

  09/H0308/58

• Date of REC Opinion

  21 Apr 2009

• REC opinion

  Further Information Favourable Opinion