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Trial record 3 of 11 for:    eliglustat

Skeletal Response to Eliglustat in Patients With Gaucher Disease (EXOSKEL)

This study is enrolling participants by invitation only.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02536755
First Posted: September 1, 2015
Last Update Posted: July 14, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
  Purpose

Primary Objective:

Evaluate long term skeletal response to eliglustat in adult patients who successfully completed the Phase 2 or Phase 3 studies.

Secondary Objective:

Evaluate the safety of eliglustat in adult patients who successfully completed the Phase 2 or Phase 3 studies.


Condition Intervention Phase
Gaucher's Disease Drug: eliglustat Phase 3

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Open Label Interventional Multicenter Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Successfully Completed the Phase 2 or Phase 3 Studies

Resource links provided by NLM:


Further study details as provided by Sanofi ( Genzyme, a Sanofi Company ):

Primary Outcome Measures:
  • Change from baseline in bone disease manifestations (clinical, radiological, and biological) [ Time Frame: Baseline to Week 104 ]

Secondary Outcome Measures:
  • Assessment of quality of life using Short Form-36 [ Time Frame: Baseline to Week 104 ]
  • Measurement of Gaucher disease type I biomarkers [ Time Frame: Baseline to Week 104 ]
  • Incidence of adverse events [ Time Frame: Baseline to Week 208 ]

Estimated Enrollment: 40
Actual Study Start Date: October 28, 2015
Estimated Study Completion Date: June 22, 2021
Estimated Primary Completion Date: June 22, 2021 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: eliglustat
Cytochrome P450 (CYP) 2D6 Intermediate (IM), Extensive (EM) and Ultra-Rapid (URM) Metaboliser patients will be treated at 84 mg twice daily. CYP2D6 Poor Metabolisers (PM) will be treated at 84 mg once daily.
Drug: eliglustat

Pharmaceutical form:capsule

Route of administration: oral

Other Name: GZ385660

Detailed Description:
The duration of the study period per patient will be at least 2 years.
  Eligibility

Information from the National Library of Medicine

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, Learn About Clinical Studies.


Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion criteria :

  • The patient must have successfully completed the Phase 2 (GZGD00304) or a Phase 3 study (GZGD02507, GZGD02607 or GZGD03109). Successful completion is defined as patients enrolled in one of the above mentioned studies who received eliglustat through the end of the study and completed the end-of-study visit without having discontinued or been withdrawn prematurely.
  • The patient is willing and able to provide signed informed consent prior to any protocol-required procedures being performed.
  • Female patients of childbearing potential must have a documented negative pregnancy test prior to enrollment and while they are receiving eliglustat treatment.
  • Female patients of childbearing potential must be willing to practice true abstinence in line with their preferred and usual lifestyle, or use a medically accepted form of contraception (either a barrier method, such as condom or diaphragm + spermicide, or a non-barrier method such as oral, injected, or implanted hormonal methods, or an intra-uterine device or system ) while receiving eliglustat.

Exclusion criteria:

  • The patient is unwilling to comply with the requirements of the protocol.
  • The patient has received an investigational product (other than eliglustat) within 30 days prior to enrollment.
  • The patient has received miglustat within the 6 months prior to enrollment.
  • The patient has documented prior esophageal varices or liver infarction or current liver enzymes (alanine transaminase, aspartate aminotransferase) or total bilirubin >2 times the upper limit of normal unless the patient has a diagnosis of Gilbert Syndrome.
  • The patient has any clinically significant disease, other than Gaucher disease, including cardiovascular, renal, hepatic, gastrointestinal, pulmonary, neurologic, endocrine, metabolic (eg, hypokalemia, hypomagnesemia), or psychiatric disease, other medical conditions, or serious intercurrent illnesses that may preclude participation in the study.
  • The patient is known to have any of the following: cardiac disease (congestive heart failure, recent acute myocardial infarction, bradycardia, heart block, ventricular arrhythmia), long QT syndrome, or current treatment with Class IA (eg, quinidine) or Class III (eg, amiodarone, sotalol) antiarrhythmic medicinal products.
  • The patient has tested positive for the human immunodeficiency virus (HIV) antibody, hepatitis C antibody, or hepatitis B surface antigen.
  • The patient has a history of cancer within 6 months of enrolment, with the exception of basal cell carcinoma.
  • Patient is CYP 2D6 IM, EM or URM taking a strong or moderate CYP2D6 inhibitor concomitantly with a strong or moderate CYP3A inhibitor
  • Patient is CYP2D6 PM taking a strong CYP3A inhibitor within 2 weeks prior to enrolment.
  • If a female patient of childbearing potential has a positive pregnancy test (blood β-Human Chorionic Gonadotropin (β-HCG) or is breast feeding prior to first dosing of eliglustat in this study, the patient cannot enroll in the study at this time, but may be rescreened after the end of the pregnancy, and/or when she is no longer breast feeding, provided rescreening takes place before the end of the enrollment period.
  • Women of childbearing potential who are unwilling or unable to be tested for pregnancy.

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02536755


Locations
Canada
Investigational Site Number 124002
Montreal, Canada, H3T 1E2
Russian Federation
Investigational Site Number 643001
Moscow, Russian Federation, 125167
Investigational Site Number 643002
St-Petersburg, Russian Federation, 197341
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
Study Director: Clinical Sciences & Operations Sanofi
  More Information

Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT02536755     History of Changes
Other Study ID Numbers: EFC13781
U1111-1166-6190 ( Other Identifier: UTN )
First Submitted: August 27, 2015
First Posted: September 1, 2015
Last Update Posted: July 14, 2017
Last Verified: July 2017

Additional relevant MeSH terms:
Eliglustat
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
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action