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| Sponsor: | Genzyme |
|---|---|
| Information provided by: | Genzyme |
| ClinicalTrials.gov Identifier: | NCT00943111 |
Purpose
This Phase 3 study was designed to confirm the efficacy and safety of Genz-112638 in patients with Gaucher disease type 1 who have been stabilized on Cerezyme
| Condition | Intervention | Phase |
|---|---|---|
|
Gaucher Disease, Type 1 |
Drug: Genz-112638 Biological: Cerezyme |
Phase III |
| Study Type: | Interventional |
| Study Design: | Treatment, Randomized, Open Label, Parallel Assignment, Safety/Efficacy Study |
| Official 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 |
| Estimated Enrollment: | 186 |
| Study Start Date: | August 2009 |
| Estimated Study Completion Date: | December 2011 |
| Estimated Primary Completion Date: | December 2011 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Investigational: Experimental
Genz-112638
|
Drug: Genz-112638
Capsules: 50, 100, or 150 mg BID
|
|
Active: Active Comparator
Cerezyme
|
Biological: Cerezyme
IV Infusion, Varied Dose, Q2
|
Gaucher disease is characterized by lysosomal accumulation of glucosylceramide due to impaired glucosylceramide hydrolysis. Gaucher disease type 1, which is the most common form, accounts for >90% of cases and does not involve the CNS. Typical manifestations of Gaucher disease type 1 include splenomegaly, hepatomegaly, thrombocytopenia, anemia, bone disease, and decreased quality of life. The disease manifestations are caused by the accumulation of glucosylceramide (storage material) in macrophages (called Gaucher cells) which have infiltrated the spleen and liver as well as other tissues.
Genz-112638 is a small molecule drug developed as an oral therapy which acts to specifically inhibit production of this storage material in Gaucher cells.
This study is designed to determine the efficacy, safety, and pharmacokinetics (PK) of Genz-112638 in adult patients with Gaucher disease type 1 who have been stabilized on Cerezyme.
Eligibility| Ages Eligible for Study: | 18 Years to 65 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contacts and Locations| Contact: Medical Information | 800-745-4447 | medinfo@genzyme.com |
| Contact: Medical Information | 617-252-7832 | medinfo@genzyme.com |
Show 22 Study Locations| Study Director: | Medical Monitor | Genzyme |
More Information
| Responsible Party: | Genzyme Corporation ( Medical Monitor ) |
| Study ID Numbers: | GZGD02607 |
| Study First Received: | July 20, 2009 |
| Last Updated: | December 1, 2009 |
| ClinicalTrials.gov Identifier: | NCT00943111 History of Changes |
| Health Authority: | United States: Food and Drug Administration; Russia: Ministry of Health and Social Development of the Russian Federation; Australia: Department of Health and Ageing Therapeutic Goods Administration |
|
Gaucher disease, Genz-112638, beta-glucosidase, acid ß-glucosidase, |
glucocerebrosidase, glucosylceramide, D-glucosyl-N-acylsphingosine glucohydrolase, substrate reduction therapy |
|
Lipid Metabolism, Inborn Errors Sphingolipidoses Metabolic Diseases Reticuloendotheliosis Lysosomal Storage Diseases, Nervous System Lysosomal Storage Diseases Nervous System Diseases Central Nervous System Diseases Brain Diseases |
Lymphatic Diseases Metabolism, Inborn Errors Genetic Diseases, Inborn Brain Diseases, Metabolic, Inborn Lipidoses Gaucher Disease Lipid Metabolism Disorders Brain Diseases, Metabolic |