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A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT01074944
First received: February 23, 2010
Last updated: September 2, 2014
Last verified: September 2014
  Purpose

The primary objective of this study is to evaluate the efficacy and safety of once daily (QD) versus twice daily (BID) dosing of eliglustat tartrate (Genz-112638) in patients with Gaucher disease type 1 who have demonstrated clinical stability on BID dosing of eliglustat tartrate (Genz-112638). The secondary objective is to evaluate the pharmacokinetics (PK) of Genz-99067 when eliglustat tartrate (Genz-112638) is administered QD and BID in patients with Gaucher disease type 1 who have demonstrated clinical stability on BID dosing of eliglustat tartrate (Genz-112638).


Condition Intervention Phase
Gaucher Disease
Drug: eliglustat tartrate
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, Multi-Center, Multi-National, Double-Blind Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Once Daily Versus Twice Daily Dosing of Genz-112638 in Patients With Gaucher Disease Type 1 Who Have Demonstrated Clinical Stability on a Twice Daily Dose of Genz-112638

Resource links provided by NLM:


Further study details as provided by Sanofi:

Primary Outcome Measures:
  • The number of randomized patients who remain stable after treatment with eliglustat tartrate (Genz-112638) for both dosing regimens (BID full dose, QD full dose) separately, along with a difference between the two dosing regimens. [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Hemoglobin level [ Time Frame: 12 months ] [ Designated as safety issue: No ]
  • Platelet count [ Time Frame: 12 months ] [ Designated as safety issue: No ]
  • Spleen volume [ Time Frame: 12 months ] [ Designated as safety issue: No ]
  • Liver volume [ Time Frame: 12 months ] [ Designated as safety issue: No ]
  • Biomarkers [ Time Frame: 12 months ] [ Designated as safety issue: No ]
  • Bone disease assessments [ Time Frame: 12 months ] [ Designated as safety issue: No ]
  • Gaucher assessments (mobility, bone crisis, bone pain) [ Time Frame: 12 months ] [ Designated as safety issue: No ]

Enrollment: 171
Study Start Date: June 2010
Estimated Study Completion Date: August 2015
Estimated Primary Completion Date: August 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Twice Daily (BID) Dose Regimen
Patients will receive either 50 mg BID or 100 mg BID
Drug: eliglustat tartrate
Oral Capsule in 50 mg or 100mg dosages
Other Name: Genz-112638
Experimental: Once Daily (QD) Dose Regimen
Patients will receive either 100 mg QD or 200 mg QD
Drug: eliglustat tartrate
Oral Capsule in 50 mg or 100mg dosages
Other Name: Genz-112638

Detailed Description:

NOTE: Other Phase 3 studies being conducted with eliglustat tartrate (Genz-112638) are GZGD02507 (ENGAGE): NCT00891202 and GZGD02607 (ENCORE): NCT00943111

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • The patient is willing and able to provide signed informed consent prior to any study-related procedures.
  • The patient has a diagnosis of Gaucher disease type 1 confirmed by a documented deficiency of acid β-glucosidase activity by enzyme assay.
  • Female patients of childbearing potential must have a documented negative pregnancy test prior to administration of the first dose of eliglustat tartrate (Genz-112638) in this study. In addition, all female patients of childbearing potential must use a medically accepted form of contraception throughout the study, i.e., either a barrier method or hormonal contraceptive with norethindrone and ethinyl estradiol or similar active components.

Exclusion Criteria:

  • The patient has had a partial or total splenectomy within 3 years prior to randomization.
  • The patient received pharmacological chaperones or miglustat within 6 months prior to administration of the first dose of eliglustat tartrate (Genz-112638) in this study.
  • The patient has any clinically significant disease, other than Gaucher disease, including cardiovascular, renal, hepatic, gastrointestinal, pulmonary, neurologic, endocrine, metabolic (including hypokalaemia or hypomagnesemia), or psychiatric disease, other medical conditions, or serious intercurrent illnesses that, in the opinion of the Investigator, may preclude participation in the study.
  • The patient is known to have tested positive for the human immunodeficiency virus (HIV) antibody, Hepatitis C antibody, or Hepatitis B surface antigen.
  • The patient has received an investigational product (other than eliglustat tartrate (Genz-112638)) within 30 days prior to administration of the first dose of eliglustat tartrate (Genz-112638) in this study.
  • The patient is pregnant or lactating.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01074944

  Show 46 Study Locations
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

Additional Information:
Publications:
Responsible Party: Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier: NCT01074944     History of Changes
Other Study ID Numbers: GZGD03109, 2009-015811-42
Study First Received: February 23, 2010
Last Updated: September 2, 2014
Health Authority: United States: Food and Drug Administration
Australia: Department of Health and Ageing Therapeutic Goods Administration
Canada: Health Canada
China: Food and Drug Administration
Croatia: Ministry of Health and Social Care
France: Ministry of Health
Greece: Ministry of Health and Welfare
India: Drugs Controller General of India
Japan: Ministry of Health, Labor and Welfare
Portugal: National Pharmacy and Medicines Institute
Russia: Pharmacological Committee, Ministry of Health
Serbia and Montenegro: Agency for Drugs and Medicinal Devices
Sweden: The National Board of Health and Welfare
Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)
Austria: Federal Office for Safety in Health Care
Romania: National Medicines Agency

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

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

ClinicalTrials.gov processed this record on November 19, 2014