Extension trial of prGCD in Gaucher patients – Dr. Mehta V1.0
Research type
Research Study
Full title
A Multicenter, Double-Blind, Extension Trial of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease
IRAS ID
19238
Sponsor organisation
Protalix Biotherapeutics
Eudract number
2008-005826-35
Clinicaltrials.gov Identifier
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 current treatment for Gaucher is life-long enzyme replacement therapy (ERT). The annual cost of current ERT treatment, per patient, is approximately US$250 000. 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 significant cost reduction due to the manufacturing process. This PB-06-003 study is an extension of the previous PB-06-001 study that investigated the efficacy and safety of two doses of prGCD in untreated Gaucher patients. The main purpose of this study is to extend the assessment of the safety and efficacy of prGCD in Gaucher patients who have completed 9 months of treatment as a part of the PB-06-001 study, and to further investigate whether a lower dose of prGCD is as safe and efficacious in the treatment of Gaucher patients as is the higher dose. The use of a lower dose will substantially decrease the cost of treatment. Up to 30 patients world wide, that have successfully completed the PB-06-001 study, will be enrolled by invitation to the PB-06-003 extension study. The patients will receive a bi-weekly dose of prGCD, either of 30 units/kg or 60 units/kg, in accordance with the dose they received in the previous PB-06-001 study. This trial will take place in Royal Free Hospital, London and in other locations worldwide. The sponsor of the trial is Protalix Biotherapeutics. The duration of treatment will be 15 months.
REC name
London - Hampstead Research Ethics Committee
REC reference
09/H0720/79
Date of REC Opinion
24 Jun 2009
REC opinion
Further Information Favourable Opinion