A Study of Genz-112638 in Patients With Gaucher Disease (ENGAGE)

Tracking Information

Start Date  ICMJEOctober 2009
Estimated Primary Completion DateSeptember 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  ICMJESame as current
Change HistoryComplete 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  ICMJESame as current

Descriptive Information

Brief Title  ICMJEA Study of Genz-112638 in Patients With Gaucher Disease(ENGAGE)
Official Title  ICMJEA 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 PhasePhase III
Study Type  ICMJEInterventional
Study Design  ICMJETreatment, Randomized, Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Parallel Assignment, Safety/Efficacy Study
Condition  ICMJEGaucher Disease, Type 1
Intervention  ICMJE
  • Drug: Genz-112638
    Capsule: 50 or 100 mg BID
  • Drug: Placebo
Study Arms / Comparison Groups
  • Active: Experimental
    Intervention: Drug: Genz-112638
  • Placebo: Placebo Comparator
    Intervention: Drug: Placebo

Recruitment Information

Estimated Enrollment  ICMJE36
Estimated Completion DateDecember 2011
Estimated Primary Completion DateSeptember 2011   (final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • The patient (and/or their parent/legal guardian) is willing and able to provide signed informed consent prior to any study-related procedures to be performed.
  • The patient is 16 to 65 years old at the time of randomization.
  • The patient has a confirmed diagnosis of Gaucher disease type 1.
  • Male patients agree to use a medically accepted method of contraception throughout the study.
  • Female patients of childbearing potential must have a documented negative pregnancy test prior to dosing. In addition all female patients of childbearing potential must use a medically accepted form of contraception throughout the study.

Exclusion Criteria:

  • The patient has had a partial or total splenectomy.
  • The patient has received pharmacological chaperones or miglustat within 6 months prior to randomization
  • The patient has received enzyme replacement therapy within 12 months prior to randomization.
  • The patient has type 2 or 3 Gaucher Disease or is suspected of having type 3 Gaucher Disease
  • The patient has any clinically significant disease, other than Gaucher Disease, including cardiovascular, renal, hepatic. gastrointestinal (GI), Pulmonary, neurologic, endocrine, metabolic, (e.g. hypokalemia, hypomagnesemia), or psychiatric disease, other medical conditions, or serious intercurrent illness that may confound the study results, or, on the opinion of the investigator, may preclude participation in the study.
  • Tha patient has tested positive for the human immunodeficiency virus (HIV)antibody, Hepatitis C antibody, or Hepatitis B surface antigen.
  • The patient has received an investigational product within 30 days prior to randomization.
  • The patient is pregnant or lactating.
Ages16 Years to 65 Years
Accepts Healthy VolunteersNo
Contacts  ICMJE
Contact: Genzyme Medical Information800-745-4447medinfo@genzyme.com
Contact: Genzyme Information617-252-7832medinfo@genzyme.com
Location Countries  ICMJEUnited States,   Canada,   India,   Russian Federation

Administrative Information

Responsible PartyMedical Monitor, Genzyme Corporation
Study ID Numbers  ICMJEGZGD02507
Study Sponsor  ICMJEGenzyme
Collaborators  ICMJE 
Investigators  ICMJE
Study Director:Medical MonitorGenzyme
Information Provided ByGenzyme