Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies
Condition: Gaucher Disease
MeSH terms: Gaucher Disease
Intervention: Eliglustat, GZ385660
Intervention description: Pharmaceutical form: capsule
Route of administration: oral
Intervention MeSH terms: Eliglustat
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 31.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No

Summary:

      Primary Objective:

      Evaluate long term skeletal response to eliglustat in adult participants who successfully
      completed one of the Phase 2 or Phase 3 eliglustat studies.

      Secondary Objective:

      Evaluate the safety of eliglustat (by serious adverse event continuous monitoring), the
      quality of life (Short Form-36 Health Survey [SF-36]) and biomarkers of Gaucher disease type
      1 (GD1) (chitotriosidase, plasma glucosylceramide [GL-1] and lyso glucosylceramide
      [lyso-GL-1]) in adult participants who successfully completed one of the Phase 2 or Phase 3
      studies.
    

Eligibility criteria:

        Inclusion criteria :

          -  The participant must have successfully completed the Phase 2 (GZGD00304) or a Phase 3
             study (GZGD02507, GZGD02607 or GZGD03109). Successful completion was defined as
             participants 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             discontinued or been withdrawn prematurely.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 participant was willing and able to provide signed informed consent prior to any
             protocol-required procedures being performed.

          -  Female participants of childbearing potential must have had a documented negative
             pregnancy test prior to enrollment and while they were receiving eliglustat treatment.

          -  Female participants of childbearing potential must have been willing to practice true
             abstinence in line with their preferred and usual lifestyle, or used 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 participant was unwilling to comply with the requirements of the protocol.

          -  The participant had received an investigational product (other than eliglustat) within
             30 days prior to enrollment.

          -  The participant had received miglustat within the 6 months prior to enrollment.

          -  The participant had documented prior esophageal varices or liver infarction or current
             liver enzymes (alanine transaminase, aspartate aminotransferase) or total bilirubin
             greater than (>)2 times the upper limit of normal, unless the participant had a
             diagnosis of Gilbert Syndrome.

          -  The participant had 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 might preclude
             participation in the study.

          -  The participant was 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 or Class
             III antiarrhythmic medicinal products.

          -  The participant had tested positive for the human immunodeficiency virus antibody,
             hepatitis C antibody, or hepatitis B surface antigen.

          -  The participant had a history of cancer within 6 months of enrolment, with the
             exception of basal cell carcinoma.

          -  Participant was a CYP2D6 IM, EM or URM and was taking a strong or moderate CYP2D6
             inhibitor concomitantly with a strong or moderate CYP3A inhibitor.

          -  Participant was a CYP2D6 PM having taken a strong CYP3A inhibitor within 2 weeks prior
             to enrolment.

          -  If a female participant of childbearing potential had a positive pregnancy test (blood
             β-human chorionic gonadotropin [β-HCG]) or was breastfeeding prior to first dosing of
             eliglustat in this study, the participant could not enroll in the study at that time,
             but might have been rescreened after the end of the pregnancy, and/or when she was no
             longer breast feeding, provided rescreening took place before the end of the
             enrollment period.

          -  Women of childbearing potential who were unwilling or unable to be tested for
             pregnancy.

        The above information was not intended to contain all considerations relevant to a
        participant's potential participation in a clinical trial.