Genzyme Corporation is recruiting patients for a multicenter, randomized, dose frequency study of the safety and efficacy of Cerezyme infusions every four weeks versus every two weeks in the maintenance therapy of patients with Type I Gaucher disease.
In 1994, the U.S. Food and Drug Administration (FDA) approved the enzyme replacement Cerezyme for the treatment of Type I Gaucher Disease. The majority of patients currently on Cerezyme are treated every two weeks for 1.5 hours per infusion. Approximately 90 patients will participate in the current study, which will evaluate safety, efficacy, and quality of life for those who receive one infusion every four weeks, as compared to every two weeks.
There will be 15 treatment centers worldwide. All patients will be followed for 24 months. To be eligible, patients must be at least 18 years old, with a confirmed diagnosis of Type I Gaucher disease. Both males and females will be included. Patients must have been on Cerezyme for at least two years, and on a stable dose of 20-40U/kg every two weeks for at least six months prior to study enrollment.
For information, please contact:
Medical Information
Genzyme Corporation
One Kendall Square
Cambridge, MA 02139
Phone: (800) 745-4447 (press Option 2)
Genzyme is also conducting a multicenter study of the efficacy of Cerezyme in treating skeletal disease in patients with Type I Gaucher disease. Patients are not currently being recruited for this study.
Read about events on Capitol Hill, funding for rare-disease research, and other topics of interest from NORD's office in Washington, DC.
