Study of Skeletal Response to Eliglustat in Patients With Gaucher Disease

• Status: Completed
• Trial end date: 2021-06-24
• Target enrollment: 0
• Participant gender: All

Summary

Primary Objective:

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

Secondary Objective:

Evaluate the safety of eliglustat (by [serious] adverse event [AE] 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 patients who successfully completed one of the Phase 2 or Phase 3 studies.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Genzyme, a Sanofi Company
• Treatments: Eliglustat

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eliglustat through the end of the study and completed the end-of-study visit without having discontinued or been withdrawn prematurely.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 or Class III 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 a CYP2D6 intermediate (IMs), extensive (EMs) or ultra-rapid (URM) metabolizer and is taking a strong or moderate CYP2D6 inhibitor concomitantly with a strong or moderate CYP3A inhibitor.

• Patient is a CYP2D6 poor metaboliser (PM) having taken 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.