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A Study of Genz-112638 in Patients With Gaucher Disease Who Have Been Stabilized on Cerezyme (ENCORE)
This study is currently recruiting participants.
Verified by Genzyme, July 2009
First Received: July 20, 2009   Last Updated: December 1, 2009   History of Changes
Sponsor: Genzyme
Information provided by: Genzyme
ClinicalTrials.gov Identifier: NCT00943111
  Purpose

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


Condition Intervention Phase
Gaucher Disease, Type 1
Drug: Genz-112638
Biological: Cerezyme
Phase III

Study Type: Interventional
Study Design: Treatment, Randomized, Open Label, Parallel Assignment, Safety/Efficacy Study
Official Title: 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

Resource links provided by NLM:


Further study details as provided by Genzyme:

Primary Outcome Measures:
  • 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 ]

Secondary Outcome Measures:
  • 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 ]

Estimated Enrollment: 186
Study Start Date: August 2009
Estimated Study Completion Date: December 2011
Estimated Primary Completion Date: December 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Investigational: Experimental
Genz-112638
Drug: Genz-112638
Capsules: 50, 100, or 150 mg BID
Active: Active Comparator
Cerezyme
Biological: Cerezyme
IV Infusion, Varied Dose, Q2

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.

  Eligibility

Ages Eligible for Study:   18 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00943111

Contacts
Contact: Medical Information 800-745-4447 medinfo@genzyme.com
Contact: Medical Information 617-252-7832 medinfo@genzyme.com

  Show 22 Study Locations
Sponsors and Collaborators
Genzyme
Investigators
Study Director: Medical Monitor Genzyme
  More Information

No publications provided

Responsible Party: Genzyme Corporation ( Medical Monitor )
Study ID Numbers: GZGD02607
Study First Received: July 20, 2009
Last Updated: December 1, 2009
ClinicalTrials.gov Identifier: NCT00943111     History of Changes
Health Authority: United States: Food and Drug Administration;   Russia: Ministry of Health and Social Development of the Russian Federation;   Australia: Department of Health and Ageing Therapeutic Goods Administration

Keywords provided by Genzyme:
Gaucher disease,
Genz-112638,
beta-glucosidase,
acid ß-glucosidase,
glucocerebrosidase,
glucosylceramide,
D-glucosyl-N-acylsphingosine glucohydrolase,
substrate reduction therapy

Additional relevant MeSH terms:
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

ClinicalTrials.gov processed this record on February 08, 2010