Intervention: Eliglustat
Trial phase: Phase 3
ATC code: A16AX10
SMILES: CCCCCCCC(=O)N[C@H](CN1CCCC1)[C@H](O)c1ccc2c(c1)OCCO2
Sex: All
Age groups: Adult | Older Adult
Healthy volunteers: No
Participants at risk: 31
Group description: Participants who completed one of the Phase 2 (GZGD00304) or Phase 3 studies (GZGD02507, GZGD02607 or GZGD03109) were enrolled in this current (EFC13781) study. Participants who were CYP2D6 IM, EM and URM received eliglustat 84 mg twice daily and participants who were CYP2D6 PM received eliglustat 84 mg once daily, for duration of minimum 2 years (unless early discontinuation occurred) and up to 4 years, or until commercial eliglustat was available to participants through reimbursement or through the compassionate use (expanded access) program.
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eliglustat through the end of the study and completed the end-of-study visit without having discontinued or been withdrawn prematurely.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.