Tracking Information
Start Date ICMJE | October 2009 |
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Estimated Primary Completion Date | September 2011 (final data collection date for primary outcome measure) |
Current Primary Outcome Measures ICMJE (submitted: April 30, 2009) | The Primary objective is to compare the effects of Genz-112638 as compared to placebo in patients with Type 1 Gaucher Disease [ Time Frame: 39 weeks ] [ Designated as safety issue: Yes ] |
Original Primary Outcome Measures ICMJE | Same as current |
Change History | Complete list of historical versions of study NCT00891202 on ClinicalTrials.gov Archive Site |
Current Secondary Outcome Measures ICMJE (submitted: April 30, 2009) | The secondary objectives for this study include an evaluation of safety and the effects on disease manifestations and disease specific bio-markers [ Time Frame: 39 weeks ] [ Designated as safety issue: No ] |
Original Secondary Outcome Measures ICMJE | Same as current |
Descriptive Information
Brief Title ICMJE | A Study of Genz-112638 in Patients With Gaucher Disease(ENGAGE) |
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Official Title ICMJE | A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study Confirming the Efficacy and Safety of Genz-112638 in Patients With Gaucher Disease Type 1 |
Brief Summary | This Phase 3, Study was designed to confirm the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 |
Detailed Description | 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 |
Study Phase | Phase III |
Study Type ICMJE | Interventional |
Study Design ICMJE | Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), 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 | 36 | ||||||||
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Estimated Completion Date | December 2011 | ||||||||
Estimated Primary Completion Date | September 2011 (final data collection date for primary outcome measure) | ||||||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Gender | Both | ||||||||
Ages | 16 Years to 65 Years | ||||||||
Accepts Healthy Volunteers | No | ||||||||
Contacts ICMJE |
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Location Countries ICMJE | United States, Canada, India, Russian Federation |
Administrative Information
NCT ID ICMJE | NCT00891202 | ||||
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Responsible Party | Medical Monitor, Genzyme Corporation | ||||
Study ID Numbers ICMJE | GZGD02507 | ||||
Study Sponsor ICMJE | Genzyme | ||||
Collaborators ICMJE | |||||
Investigators ICMJE |
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Information Provided By | Genzyme |