Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies

NCT ID: NCT02536755

Last Updated: 2022-07-15

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 31 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-10-27
• Study Completion Date: 2021-06-24

Brief 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.

Detailed Description

Study duration for individual participants was to be of minimum 2 years and up to 4 years, or until commercial eliglustat was available to participants through reimbursement. If after 4 years since the beginning of the study eliglustat was not approved and available to participants in participating country, participants in this country might continue to receive study treatment until eliglustat was available to participants through the compassionate use (expanded access) program or was approved and available through reimbursement, whichever came first.

Conditions

Gaucher Disease

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Eliglustat

Participants who completed one of the Phase 2 (GZGD00304 \[NCT00358150\]) or Phase 3 studies (GZGD02507 \[NCT00891202\], GZGD02607 \[NCT00943111\], or GZGD03109 \[NCT01074944\]) were enrolled in this current (EFC13781) study. Participants who were cytochrome P450 (CYP) 2D6 intermediate metabolizer (IM), extensive metabolizer (EM) and ultra-rapid metabolizers (URM) received eliglustat 84 milligrams (mg) twice daily and participants who were CYP2D6 poor metabolizer (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.

Group Type: EXPERIMENTAL

Eliglustat, GZ385660

Intervention Type: DRUG

Pharmaceutical form: capsule

Route of administration: oral

Interventions

Eliglustat, GZ385660

Pharmaceutical form: capsule

Route of administration: oral

Intervention Type: DRUG

Other Intervention Names

Cerdelga

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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Genzyme, a Sanofi Company

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Clinical Sciences & Operations

Role: STUDY_DIRECTOR

Sanofi

Locations

Investigational Site Number 124002

Montreal, , Canada

Investigational Site Number 643001

Moscow, , Russia

Investigational Site Number 643002

Saint Petersburg, , Russia

Investigational Site Number 788001

Tunis, , Tunisia

Countries

Canada; Russia; Tunisia

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

U1111-1166-6190

Identifier Type: OTHER

Identifier Source: secondary_id

EFC13781

Identifier Type: -

Identifier Source: org_study_id