Tracking Information
Start Date ICMJE | August 2009 |
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Estimated Primary Completion Date | December 2011 (final data collection date for primary outcome measure) |
Current Primary Outcome Measures ICMJE (submitted: November 5, 2009) | The primary objective of this study is to assess the efficacy and safety of Genz-112638 compared with Cerezyme after 52 weeks of treatment in patients with Gaucher disease type 1 who have been stabilized with Cerezyme. [ Time Frame: 52 weeks ] [ Designated as safety issue: No ] |
Original Primary Outcome Measures ICMJE (submitted: July 21, 2009) | The primary objective of this study in patients with Gaucher disease type 1 who have been stabilized with Cerezyme is to demonstrate that, the majority of patients who receive Genz-112638 remain stable. [ Time Frame: 39 weeks ] [ Designated as safety issue: No ] |
Change History | Complete list of historical versions of study NCT00943111 on ClinicalTrials.gov Archive Site |
Current Secondary Outcome Measures ICMJE (submitted: November 5, 2009) | The secondary objective of this study is to demonstrate that, in patients with Gaucher disease type 1 who have been stabilized with Cerezyme, the majority of patients who receive Genz-112638 remain stable after 52 weeks of treatment. [ Time Frame: 52 weeks ] [ Designated as safety issue: No ] |
Original Secondary Outcome Measures ICMJE (submitted: July 21, 2009) | The secondary objective is to assess the stability rates and safety of patients treated with Genz-112638 to Cerezyme. [ Time Frame: 39 weeks ] [ Designated as safety issue: No ] |
Descriptive Information
Brief Title ICMJE | A Study of Genz-112638 in Patients With Gaucher Disease Who Have Been Stabilized on Cerezyme |
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Official Title ICMJE | 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 |
Brief Summary | 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 |
Detailed Description | 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. |
Study Phase | Phase III |
Study Type ICMJE | Interventional |
Study Design ICMJE | Treatment, Randomized, Open Label, Parallel Assignment, Safety/Efficacy Study |
Condition ICMJE | Gaucher Disease, Type 1 |
Intervention ICMJE |
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Study Arms / Comparison Groups |
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Recruitment Information
Estimated Enrollment ICMJE | 186 | ||||||||
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Estimated Completion Date | December 2011 | ||||||||
Estimated Primary Completion Date | December 2011 (final data collection date for primary outcome measure) | ||||||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Gender | Both | ||||||||
Ages | 18 Years to 65 Years | ||||||||
Accepts Healthy Volunteers | No | ||||||||
Contacts ICMJE |
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Location Countries ICMJE | United States, Argentina, Australia, Brazil, Czech Republic, France, Germany, Italy, Netherlands, Russian Federation, Spain, United Kingdom |
Administrative Information
NCT ID ICMJE | NCT00943111 | ||||
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Responsible Party | Medical Monitor, Genzyme Corporation | ||||
Study ID Numbers ICMJE | GZGD02607 | ||||
Study Sponsor ICMJE | Genzyme | ||||
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Investigators ICMJE |
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Information Provided By | Genzyme |