A Study of Genz-112638 in Patients With Gaucher Disease Who Have Been Stabilized on Cerezyme (ENCORE)


Tracking Information

Start Date  ICMJEAugust 2009
Estimated Primary Completion DateDecember 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 HistoryComplete 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  ICMJEA Study of Genz-112638 in Patients With Gaucher Disease Who Have Been Stabilized on Cerezyme
Official Title  ICMJEA 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 PhasePhase III
Study Type  ICMJEInterventional
Study Design  ICMJETreatment, Randomized, Open Label, Parallel Assignment, Safety/Efficacy Study
Condition  ICMJEGaucher Disease, Type 1
Intervention  ICMJE
  • Drug: Genz-112638
    Capsules: 50, 100, or 150 mg BID
  • Biological: Cerezyme
    IV Infusion, Varied Dose, Q2
Study Arms / Comparison Groups
  • Investigational: Experimental
    Genz-112638
    Intervention: Drug: Genz-112638
  • Active: Active Comparator
    Cerezyme
    Intervention: Biological: Cerezyme

Recruitment Information

Estimated Enrollment  ICMJE186
Estimated Completion DateDecember 2011
Estimated Primary Completion DateDecember 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 18 to 65 years old at the time of randomization.
  • The patient has a confirmed diagnosis of Gaucher disease type 1.
  • The patient has received treatment with Cerezyme for at least 3 years at a prescribed dose of ≥ 20 U/kg to ≤ 60 U/kg (± 5 U/kg) q2w during the last year of treatment and has not had a dose reduction, regimen change, or treatment interruption for greater than 6 consecutive months prior to randomization.
  • The patient has clinically stable Gaucher disease prior to randomization.
  • 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 within 3 years prior to randomization.
  • The patient has received pharmacological chaperones or miglustat within 6 months prior to randomization.
  • The patient has Gaucher disease type 2 or 3 or is suspected of having Gaucher disease type 3.
  • 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 illnesses that may confound the study results or, in the opinion of the Investigator, may preclude participation in the study.
  • The 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.
GenderBoth
Ages18 Years to 65 Years
Accepts Healthy VolunteersNo
Contacts  ICMJE
Contact: Medical Information800-745-4447medinfo@genzyme.com
Contact: Medical Information617-252-7832medinfo@genzyme.com
Location Countries  ICMJEUnited States,   Argentina,   Australia,   Brazil,   Czech Republic,   France,   Germany,   Italy,   Netherlands,   Russian Federation,   Spain,   United Kingdom

Administrative Information

NCT ID  ICMJENCT00943111
Responsible PartyMedical Monitor, Genzyme Corporation
Study ID Numbers  ICMJEGZGD02607
Study Sponsor  ICMJEGenzyme
Collaborators  ICMJE 
Investigators  ICMJE
Study Director:Medical MonitorGenzyme
Information Provided ByGenzyme