A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.
|ClinicalTrials.gov Identifier: NCT00365131|
Recruitment Status : Completed
First Posted : August 17, 2006
Last Update Posted : March 5, 2015
This is a multicenter, open-label, prospective study of the efficacy of Cerezyme in treating patients with skeletal manifestations secondary to Type I Gaucher disease.
The study objective is to evaluate and quantify skeletal responses as compared to baseline in Type I gaucher disease patients receiving Cerezyme therapy for 48 months. Additional objectives were to assess the usefulness of various skeletal parameters, such as bone pain, bone crises, bone mineral density, and serum and urine bone markers, as indicative of treatment response and may be useful in dose management.
|Condition or disease||Intervention/treatment||Phase|
|Gaucher Disease Type I Cerebroside Lipidosis Syndrome Clucocerebrosidase Deficiency Disease Glucosylceramide Beta-Glucosidase Deficiency Disease Gaucher Disease, Non-Neuronopathic Form||Drug: Cerezyme (imiglucerase for injection)||Phase 4|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||40 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Study Start Date :||December 1997|
|Study Completion Date :||July 2004|
- Skeletal response over 4 years of Cerezyme therapy
- Assess use of skeletal parameter as indicative of treatment response and use in dose management
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00365131
|United States, Florida|
|Coral Springs, Florida, United States, 33065|
|Study Director:||Medical Monitor||Genzyme, a Sanofi Company|