Home
Search
Study Topics
Glossary
|
|
|
|
|
Sponsored by: |
Genzyme |
---|---|
Information provided by: | Genzyme |
ClinicalTrials.gov Identifier: | NCT00891202 |
This Phase 3, Study was designed to confirm the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1
Condition | Intervention | Phase |
---|---|---|
Gaucher Disease Type 1 |
Drug: Genz-112638 Drug: Placebo |
Phase III |
Study Type: | Interventional |
Study Design: | Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Parallel Assignment, Safety/Efficacy Study |
Official Title: | A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study Conforming the Efficacy and Safety of Genz-112638 in Patients With Gaucher Disease Type 1 |
Estimated Enrollment: | 36 |
Study Start Date: | June 2009 |
Estimated Study Completion Date: | December 2011 |
Estimated Primary Completion Date: | September 2011 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
---|---|
Active: Experimental
Genz-112638
|
Drug: Genz-112638
Capsule: 50 or 100 mg BID
|
Placebo: Placebo Comparator
Placebo
|
Drug: Placebo
Capsule
|
Gaucher disease is characterised by lysosomal accumulation of glucosylceramide due to impaired glucosylceramide hydrolysis. Type 1 Gaucher Disease, the most common form accounts for > 90% of cases and does not involve the CNS. Typical manifestations of type 1 Gaucher Disease include splenomegaly, hepatomegaly, thrombocytopenia, anemia, skeletal pathology and decreased quality of life. The disease manifestations are caused by the accumulations of glucosylceramide (storage material) in Gaucher cells which have infiltrated the spleen and liver as well as other tissue. Genz-112638 is a small molecule 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 (> 16 years) with Gaucher Disease type 1
Ages Eligible for Study: | 16 Years to 65 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contact: Genzyme Medical Information | 800-745-4447 | medinfo@genzyme.com |
Study Director: | Medical Monitor | Genzyme |
Responsible Party: | Genzyme Corporation ( Medical Monitor ) |
Study ID Numbers: | GZGD02507 |
Study First Received: | April 30, 2009 |
Last Updated: | April 30, 2009 |
ClinicalTrials.gov Identifier: | NCT00891202 History of Changes |
Health Authority: | United States: Food and Drug Administration |
Gaucher, beta-glucosidase, acid ß-glucosidase, glucocerebrosidase, glucosylceramide, D-glucosyl-N-acylsphingosine glucohydrolase, substrate reduction
therapy |
Lipid Metabolism, Inborn Errors Sphingolipidoses Metabolic Diseases Gaucher Disease Type 1 Lysosomal Storage Diseases Sphingolipidosis Central Nervous System Diseases Brain Diseases Lymphatic Diseases |
Metabolism, Inborn Errors Genetic Diseases, Inborn Brain Diseases, Metabolic, Inborn Lipidoses Gaucher Disease Metabolic Disorder Lipid Metabolism Disorders Brain Diseases, Metabolic |
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 |