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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02536755




Registration number
NCT02536755
Ethics application status
Date submitted
27/08/2015
Date registered
1/09/2015

Titles & IDs
Public title
Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies
Scientific 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
Secondary ID [1] 0 0
U1111-1166-6190
Secondary ID [2] 0 0
EFC13781
Universal Trial Number (UTN)
Trial acronym
EXOSKEL
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Gaucher Disease 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Eliglustat, GZ385660

Experimental: 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.


Treatment: Drugs: Eliglustat, GZ385660
Pharmaceutical form: capsule

Route of administration: oral
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208
Timepoint [1] 0 0
Study Baseline, Weeks 52, 104, 156 and 208
Primary outcome [2] 0 0
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208
Timepoint [2] 0 0
Study Baseline, Weeks 52, 104, 156 and 208
Primary outcome [3] 0 0
Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208
Timepoint [3] 0 0
Study Baseline, Weeks 52, 104, 156 and 208
Primary outcome [4] 0 0
Change From Current Study Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208
Timepoint [4] 0 0
Study Baseline, Weeks 52, 104, 156 and 208
Primary outcome [5] 0 0
Change From Eliglustat Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208
Timepoint [5] 0 0
Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study
Primary outcome [6] 0 0
Change From Current Study Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208
Timepoint [6] 0 0
Study Baseline, Weeks 52, 104, 156 and 208
Primary outcome [7] 0 0
Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208
Timepoint [7] 0 0
Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study
Primary outcome [8] 0 0
Change From Current Study Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208
Timepoint [8] 0 0
Study Baseline, Weeks 52, 104, 156 and 208
Primary outcome [9] 0 0
Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208
Timepoint [9] 0 0
Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study
Primary outcome [10] 0 0
Change From Current Study Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208
Timepoint [10] 0 0
Study Baseline, Weeks 52, 104, 156 and 208
Primary outcome [11] 0 0
Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208
Timepoint [11] 0 0
Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study
Primary outcome [12] 0 0
Total Number of New or Worsening Osteonecrosis Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208
Timepoint [12] 0 0
Study Baseline, Weeks 52, 104, 156 and 208
Primary outcome [13] 0 0
Total Number of New or Worsening Fracture Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208
Timepoint [13] 0 0
Study Baseline, Weeks 52, 104, 156 and 208
Primary outcome [14] 0 0
Total Number of New or Worsening Infarcts Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208
Timepoint [14] 0 0
Study Baseline, Weeks 52, 104, 156 and 208
Primary outcome [15] 0 0
Total Number of New or Worsening Lytic Lesions Events for Spine at Study Baseline, Week 104, and 208
Timepoint [15] 0 0
Study Baseline, Weeks 104, and 208
Primary outcome [16] 0 0
Observed Annual Incidence Rate for Spine and Femur Osteonecrosis at Week 52, 104, 156 and 208
Timepoint [16] 0 0
For 52 Weeks (i.e., 1 year),104 Weeks (i.e., 2 year), 156 Weeks (i.e., 3 year) and 208 Weeks (i.e., 4 years)
Primary outcome [17] 0 0
Observed Annual Incidence Rate for Spine and Femur Fracture at Week 52, 104, 156 and 208
Timepoint [17] 0 0
For 52 Weeks (i.e., 1 year),104 Weeks (i.e., 2 year), 156 Weeks (i.e., 3 year) and 208 Weeks (i.e., 4 years)
Primary outcome [18] 0 0
Observed Annual Incidence Rate for Spine and Femur Infarcts at Week 52, 104, 156 and 208
Timepoint [18] 0 0
For 52 Weeks (i.e., 1 year),104 Weeks (i.e., 2 year), 156 Weeks (i.e., 3 year) and 208 Weeks (i.e., 4 years)
Primary outcome [19] 0 0
Observed Annual Incidence Rate for Spine Lytic Lesion at Week 104, and 208
Timepoint [19] 0 0
For 104 Weeks (i.e., 2 year), and 208 Weeks (i.e., 4 years)
Primary outcome [20] 0 0
Change From Current Study Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1ß) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234
Timepoint [20] 0 0
Study Baseline, Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234
Primary outcome [21] 0 0
Change From Eliglustat Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1ß) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234
Timepoint [21] 0 0
Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 of the current study
Primary outcome [22] 0 0
Change From Current Study Baseline in Bone Biomarker Level: Procollagen 1 N- Terminal Propeptide (P1NP) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234
Timepoint [22] 0 0
Study Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234
Primary outcome [23] 0 0
Change From Eliglustat Baseline in Bone Biomarker Level: Procollagen 1 N- Terminal Propeptide (P1NP) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234
Timepoint [23] 0 0
Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 of the current study
Primary outcome [24] 0 0
Change From Current Study Baseline in Bone Biomarker Level: Type 1 Collagen C-Telopeptides (CTx) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234
Timepoint [24] 0 0
Study Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234
Primary outcome [25] 0 0
Change From Eliglustat Baseline in Bone Biomarker Level: Type 1 Collagen C-Telopeptides (CTx) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234
Timepoint [25] 0 0
Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 of the current study
Secondary outcome [1] 0 0
Change From Current Study Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234
Timepoint [1] 0 0
Study Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234
Secondary outcome [2] 0 0
Change From Eliglustat Baseline in Gaucher Disease Type 1 (GD1) Biomarker Levels: Chitotriosidase at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234
Timepoint [2] 0 0
Eliglustat Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study
Secondary outcome [3] 0 0
Change From Current Study Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234
Timepoint [3] 0 0
Study Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234
Secondary outcome [4] 0 0
Change From Eliglustat Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234
Timepoint [4] 0 0
Eliglustat Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study
Secondary outcome [5] 0 0
Change From Current Study Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234
Timepoint [5] 0 0
Study Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234
Secondary outcome [6] 0 0
Change From Eliglustat Baseline in GD1 Biomarker Levels: Lyso Glucosylceramide (Lyso-GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234
Timepoint [6] 0 0
Eliglustat Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study
Secondary outcome [7] 0 0
Change From Current Study Baseline in Short Form-36 Health Survey (SF-36) Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234
Timepoint [7] 0 0
Study Baseline, Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234
Secondary outcome [8] 0 0
Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234
Timepoint [8] 0 0
Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study
Secondary outcome [9] 0 0
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
Timepoint [9] 0 0
From the first administration of the IMP to the last administration of the IMP + 5 days (up to 4 years, or until commercial eliglustat was available to participants through reimbursement or through the compassionate use [expanded access] program)

Eligibility
Key inclusion 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.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
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 ß-human chorionic gonadotropin [ß-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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
27/10/2015
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
24/06/2021
Sample size
Target
Accrual to date
Final
31
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
Canada
State/province [1] 0 0
Montreal
Country [2] 0 0
Russian Federation
State/province [2] 0 0
Moscow
Country [3] 0 0
Russian Federation
State/province [3] 0 0
St-Petersburg
Country [4] 0 0
Tunisia
State/province [4] 0 0
Tunis

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Genzyme, a Sanofi Company
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Clinical Sciences & Operations
Address 0 0
Sanofi
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT02536755