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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT00891202
First received: April 30, 2009
Last updated: January 16, 2014
Last verified: December 2013
  Purpose

This Phase 3, Study was designed to confirm the Efficacy and Safety of eliglustat tartrate (Genz-112638) in Patients with Gaucher Disease Type 1


Condition Intervention Phase
Gaucher Disease, Type 1
Drug: eliglustat tartrate
Drug: Placebo
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study Confirming the Efficacy and Safety of Genz-112638 in Gaucher Type 1 Patients

Resource links provided by NLM:


Further study details as provided by Sanofi:

Primary Outcome Measures:
  • Percent change in spleen volume (in multiples of normal (MN)) from Baseline to 39 weeks of treatment with eliglustat tartrate (Genz-112638) as compared to placebo [ Time Frame: 39 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Absolute change in hemoglobin level (g/dL) from Baseline to week 39 [ Time Frame: 39 weeks ] [ Designated as safety issue: No ]
  • Percent change in liver volume (in MN) from Baseline to week 39 [ Time Frame: 39 weeks ] [ Designated as safety issue: No ]
  • Percent change in platelet counts from baseline to week 39 [ Time Frame: 39 weeks ] [ Designated as safety issue: No ]

Enrollment: 40
Study Start Date: November 2009
Estimated Study Completion Date: November 2015
Primary Completion Date: July 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Active
eliglustat tartrate (Genz-112638)
Drug: eliglustat tartrate
Capsule: 50 or 100 mg BID
Other Name: Genz-112638
Placebo Comparator: Placebo
Placebo
Drug: Placebo
Capsule

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 central nervous system (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. Eliglustat tartrate (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 eliglustat tartrate (Genz-112638) in adult patients (> 16 years) with Gaucher Disease type 1 The study will consist of 2 periods: The Double-Blind Primary Analysis Period (Day 1 to Week 39) and the Open-Label Period (post-Week 39 [Day 1 of the Open-Label Period] through study completion). After Week 39 assessments are completed, each patient will enter the Open-Label Period where all patients will receive Genz-112638 from post-Week 39 (Day 1 of the Open-Label Period) through study completion. All patients entering in open label period will receive doses of 50, 100 or 150 mg BID based upon plasma levels. The Open-Label Period will include a dose-adjustment period (post-Week 39 to Week 47), a long-term treatment period (Week 48 through study completion which includes a safety follow-up period [30 to 37 days after the patient's last dose of treatment])

  Eligibility

Ages Eligible for Study:   16 Years and older
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 at least 16 years old at the time of randomization.
  • The patient has a confirmed diagnosis of Gaucher disease type 1.
  • 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 9 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.
  • 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: NCT00891202

  Show 24 Study Locations
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

Publications:
Responsible Party: Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier: NCT00891202     History of Changes
Other Study ID Numbers: GZGD02507, 2008-005222-37
Study First Received: April 30, 2009
Last Updated: January 16, 2014
Health Authority: United States: Food and Drug Administration
India: Drugs Controller General of India
Russia: Ministry of Health of the Russian Federation
Netherlands: Medicines Evaluation Board (MEB)
Canada: Health Canada
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Mexico: Ministry of Health
Bulgaria: Bulgarian Drug Agency
Israel: Israeli Health Ministry Pharmaceutical Administration
Jordan: Ethical Committee
Tunisia: Office of Pharmacies and Medicines
Colombia: INVIMA Instituto Nacional de Vigilancia de Medicamentos y Alimentos
Lebanon: Ministry of Public Health
Chile: Ministry of Health
Serbia and Montenegro: Agency for Drugs and Medicinal Devices

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

Additional relevant MeSH terms:
Gaucher Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders

ClinicalTrials.gov processed this record on April 15, 2014