Gaucher DiseasePhase 3EXOSKELNCT02536755

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

A Phase 3 study testing Eliglustat, GZ385660 in people with Gaucher Disease, run by Genzyme, a Sanofi Company at 4 sites in 3 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial 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… Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Gaucher Disease
What is being tested?: Eliglustat, GZ385660
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 31 participants
Where: 4 sitesin 3 countries
Study dates: 27 Oct 2015to 24 Jun 2021
Phase: Phase 3
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

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eliglustat through the end of the study and completed the end-of-study visit without having discontinued or been withdrawn prematurely.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.

Who cannot join

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.

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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.

Trial locations and contacts

4 sites in 3 countries.

Country

City, postcode or hospital

4 sites in 3 countries

Russia2 sites

Investigational Site Number 643001
Moscow, 125167
Investigational Site Number 643002
Saint Petersburg, 197341

Other trials in Russia

Canada1 site

Investigational Site Number 124002
Montreal, H3T 1E2

Other trials in Canada

Tunisia1 site

Investigational Site Number 788001
Tunis, 1007

Other trials in Tunisia

About this study

Official 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

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.

Read the 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
Being tested: Eliglustat, GZ385660
Medical subject headings: Gaucher Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Eliglustat, GZ385660Drug
Also called Cerdelga
Pharmaceutical form: capsule
Route of administration: oral

Study groups in NCT02536755
Study group	Role	What they receive
Eliglustat	Experimental	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. Receives: Drug: Eliglustat, GZ385660

What is being measured

The primary outcome is the main question the study is designed to answer.

Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208
Mobility, i.e., ability to walk was assessed as a part of Gaucher disease assessment in participants. In this outcome measure, number of participants with their different mobility status along with the use of mobility aids (unrestricted mobility, walks with difficulty, walks with orthopaedic aid, requires wheelchair, bedridden) at specified time points were reported. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Measured: Study Baseline, Weeks 52, 104, 156 and 208
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208
Bone pain was assessed as a part of Gaucher disease assessment in participants. Participants were categorized as none (no bone pain), very mild bone pain, mild bone pain, moderate bone pain, severe bone pain and extreme bone pain during the past 4 weeks at each specified visit. In this outcome measure, number of participants with different level of bone pain during the past 4 weeks at specified time points were reported. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Measured: Study Baseline, Weeks 52, 104, 156 and 208
Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208
Bone crisis was assessed as a part of Gaucher disease assessment in participants. Acute, excruciating episodic bone pain is characteristic of Gaucher bone crisis, which typically causes periosteal elevation, elevated white blood cell count, fever, or debilitation lasting several days or longer and requires treatment with immobilization of the affected area, and opioid analgesics. Participants were categorized as 0= no bone crisis, 1= 1 bone crisis, 2= 2 bone crisis and >=3 = more than 3 bone crisis during the assessment period. In this outcome measure, number of participants with different bone crises levels at specified time points were reported. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Measured: Study Baseline, Weeks 52, 104, 156 and 208
Change From Current Study Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208
Bone marrow burden (BMB) scores indicate the degree of bone marrow infiltration. BMB score was measured using MRI (magnetic resonance imaging (MRI), ranged from 0 (no abnormalities) to 8 points (severe disease) for the lumbar spine and from 0 (no abnormalities) to 8 points (severe disease) for the femurs. The total BMB score was calculated as the sum of scores for femur and lumbar spine regions which ranged from 0 (no abnormalities) to 16 (severe disease) points. A higher BMB score signified more severe bone marrow involvement. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Measured: Study Baseline, Weeks 52, 104, 156 and 208
Change From Eliglustat Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208
Bone marrow burden (BMB) scores indicate the degree of bone marrow infiltration was measured using MRI, ranged from 0 (no abnormalities) to 8 points (severe disease) for the lumbar spine and from 0 (no abnormalities) to 8 points (severe disease) for the femurs. The total BMB score was calculated as the sum of scores for femur and lumbar spine regions which ranged from 0 (no abnormalities) to 16 (severe disease) points. A higher BMB score signified more severe bone marrow involvement. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.
Measured: Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study
Change From Current Study Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208
Bone Mineral Density (BMD) measurements of the spine and bilateral femur were acquired by dual energy X-Ray absorptiometry (DXA) scan. Worst total femur at Baseline refers to the "worst" diseased left or right femur at Baseline. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Measured: Study Baseline, Weeks 52, 104, 156 and 208
Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208
BMD measurements of the spine and bilateral femur were acquired by DXA scan. Worst total femur at Baseline refers to the "worst" diseased left or right femur at baseline. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.
Measured: Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study
Change From Current Study Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208
BMD measurements of the spine and bilateral femur were acquired by DXA scan. The T-score bone density categories were: normal (score >-1), osteopenia (score -2.5 to <=-1), and osteoporosis (score <= -2.5). Worst total femur at Baseline refers to the "worst" diseased left or right femur at baseline. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Measured: Study Baseline, Weeks 52, 104, 156 and 208
Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208
BMD measurements of the spine and bilateral femur were acquired by DXA scan. The T-score bone density categories were: normal (score >-1), osteopenia (score -2.5 to <=-1), and osteoporosis (score <= -2.5). Worst total femur at Baseline refers to the "worst" diseased left or right femur at baseline. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.
Measured: Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study
Change From Current Study Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208
BMD measurements of the spine and bilateral femur were acquired by DXA scan. The Z-score bone density categories were: normal (score >-2) and below normal (score <=-2). Worst total femur at Baseline refers to the "worst" diseased left or right femur at baseline. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Measured: Study Baseline, Weeks 52, 104, 156 and 208
Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208
BMD measurements of the spine and bilateral femur were acquired by DXA scan. The Z-score bone density categories are: normal (score >-2) and below normal (score <=-2). Worst total femur at Baseline refers to the "worst" diseased left or right femur at baseline. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.
Measured: Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study
Total Number of New or Worsening Osteonecrosis Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208
Osteonecrosis was assessed by bone MRI and X-Ray for spine and by MRI for femur. Total number of new or worsening osteonecrosis events among all the participants with corresponding assessment at specified time points were reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Measured: Study Baseline, Weeks 52, 104, 156 and 208
Total Number of New or Worsening Fracture Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208
Fracture was assessed by bone MRI and X-Ray for spine and by MRI for femur. Total number of new or worsening fracture events among all the participants with corresponding assessment at specified time points were reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Measured: Study Baseline, Weeks 52, 104, 156 and 208
Total Number of New or Worsening Infarcts Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208
Infarcts were assessed by bone MRI and X-Ray for spine and by MRI for femur. Total number of new or worsening infarcts events among all the participants with corresponding assessment at specified time points were reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Measured: Study Baseline, Weeks 52, 104, 156 and 208
Total Number of New or Worsening Lytic Lesions Events for Spine at Study Baseline, Week 104, and 208
Lytic Lesions were assessed by bone X-Ray for spine. Total number of new or worsening lytic lesions events among all the participants with corresponding assessment at specified time points were reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Measured: Study Baseline, Weeks 104, and 208
Observed Annual Incidence Rate for Spine and Femur Osteonecrosis at Week 52, 104, 156 and 208
Observed annual incidence rate was estimated using the total number of events divided by the total years of follow-up in each specified year (for all participants). Osteonecrosis was assessed by bone MRI and X-Ray for spine and by MRI for femur.
Measured: 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)
Observed Annual Incidence Rate for Spine and Femur Fracture at Week 52, 104, 156 and 208
Observed annual incidence rate was estimated using the total number of events divided by the total years of follow-up in each specified year (for all participants). Fracture was assessed by bone MRI and X-Ray for spine and by MRI for femur.
Measured: 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)
Observed Annual Incidence Rate for Spine and Femur Infarcts at Week 52, 104, 156 and 208
Observed annual incidence rate was estimated using the total number of events divided by the total years of follow-up in each specified year (for all participants). Infarcts were assessed by bone MRI and X-Ray for spine and by MRI for femur.
Measured: 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)
Observed Annual Incidence Rate for Spine Lytic Lesion at Week 104, and 208
Observed annual incidence rate was estimated using the total number of events divided by the total years of follow-up in each specified year (for all participants). Lytic Lesions were assessed by bone X-Ray for spine.
Measured: For 104 Weeks (i.e., 2 year), and 208 Weeks (i.e., 4 years)
Change From Current Study Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234
MIP-1β considered a biomarker of active bone disease, was assayed from plasma. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Measured: Study Baseline, Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234
Change From Eliglustat Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234
MIP-1β considered a biomarker of active bone disease, was assayed from plasma. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.
Measured: Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 of the current study
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
P1NP, a marker of bone formation was assayed from plasma. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Measured: Study Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234
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
P1NP, a marker of bone formation was assayed from plasma. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.
Measured: Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 of the current study
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
CTx, a marker of bone resorption was assayed from plasma. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Measured: Study Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234
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
CTx, a marker of bone resorption was assayed from plasma. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.
Measured: Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 of the current study

Secondary outcomes (9)

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
Chitotriosidase biomarker was assayed from plasma. Chitotriosidase biomarker levels for participants who were CYP2D6 non-Ultra Rapid Metabolizers (non-URM) was reported in this outcome measure. For this outcome measure, baseline refers to the study baseline, which was defined as status at study entry.
Measured: Study Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234
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
Chitotriosidase biomarker was assayed from plasma. Chitotriosidase biomarker levels for participants who were CYP2D6 non-URM was reported in this outcome measure. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. Data for this outcome measure was planned to be collected and analyzed in reference to eliglustat baseline separately for Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from ERT).
Measured: Eliglustat Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study
Change From Current Study Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234
Glucosylceramide (GL-1) biomarker was assayed from plasma. GL-1 biomarker levels for participants who were CYP2D6 non-URM was reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Measured: Study Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234
Change From Eliglustat Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234
Glucosylceramide (GL-1) biomarker was assayed from plasma. GL-1 biomarker levels for participants who were CYP2D6 non-URM was reported in this outcome measure. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. Data for this outcome measure was planned to be collected and analyzed in reference to eliglustat baseline separately for participants from Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from ERT).
Measured: Eliglustat Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study
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
Lyso-GL-1 biomarker was assayed from plasma. Lyso-GL-1 biomarker levels for participants who were CYP2D6 non-URM was reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Measured: Study Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234
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
Lyso-GL-1 biomarker was assayed from plasma. Lyso-GL-1 biomarker levels for participants who were CYP2D6 non-URM was reported in this outcome measure. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study. Data for this outcome measure was planned to be collected and analyzed in reference to eliglustat baseline separately for participants from Phase 2, ENGAGE and EDGE studies (GD treatment naïve participants) and from ENCORE study (participants switched from ERT).
Measured: Eliglustat Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study
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
The 36-Item Short-Form Health Survey (SF-36) is standardized survey evaluating 8 aspects of functional health and well-being. Physical Component Summary (PCS) with 4 sub-scales: physical function, role limitations due to physical problems, bodily pain, and general health perception; and Mental Component Summary (MCS) with 4 sub-scales: vitality, social function, role limitations due to emotional problems, and mental health. Summations of item scores of the same sub-scale give the sub-scale scores, which are transformed into a range from 0 to 100; 0= worst and 100=best outcome. Both PCS and MCS range from 0 to 100 with higher scores indicating better physical and mental health. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Measured: Study Baseline, Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234
Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234
SF-36 is a standardized survey evaluating 8 aspects of functional health and well-being. PCS with 4 sub-scales: physical function, role limitations due to physical problems, pain, and general health perception; and MCS with 4 sub-scales: vitality, social function, role limitations due to emotional problems, and mental health. Summations of item scores of same sub-scale give the sub-scale scores, which are transformed into range from 0 to 100; 0= worst, and 100=best outcome. Both PCS and MCS range from 0 to 100, higher scores indicating better physical and mental health. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.
Measured: Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug and did not necessarily had to have a causal relationship with the treatment. A serious adverse event (SAE) was any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a medically important event. TEAEs were defined as AEs that developed, worsened or became serious during the treatment-emergent period (time from the first administration of the investigational medicinal product (IMP) to the last administration of the IMP + 5 days).
Measured: 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)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

27 Oct 201524 Jun 2021

First posted1 Sep 2015 (estimated)
Study start27 Oct 2015
Primary completion24 Jun 2021
Study completion24 Jun 2021

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Gaucher DiseaseStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 3
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 31 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02536755
Sponsor's protocol ID: EFC13781
Other IDs: U1111-1166-6190
Acronym: EXOSKEL
Status last verified: Jun 2022
First submitted: 27 Aug 2015
Last updated: 15 Jul 2022
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
Individual data sharing: Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org.

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

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Study at a glance

From the registry record, last updated by the sponsor on 15 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.

Official 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
ClinicalTrials.gov ID: NCT02536755
Acronym / sponsor ID: EXOSKEL / EFC13781
Conditions: Gaucher Disease
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 31 participants (actual)
Lead sponsor: Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
Study start: 27 Oct 2015 (actual)
Primary completion: 24 Jun 2021 (actual)
Study completion: 24 Jun 2021 (actual)
First posted: 1 Sep 2015 (estimated)
Results: Posted 15 Jul 2022

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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:

Detailed description

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Eliglustat	Experimental	Drug: Eliglustat, GZ385660Participants 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.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (4 listed)

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.

Key exclusion criteria (13 listed)

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.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Eliglustat, GZ385660Also: CerdelgaPharmaceutical form: capsule Route of administration: oral	Drug	Eliglustat

Primary outcome

Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208
Time frame: Study Baseline, Weeks 52, 104, 156 and 208
Mobility, i.e., ability to walk was assessed as a part of Gaucher disease assessment in participants. In this outcome measure, number of participants with their different mobility status along with the use of mobility aids (unrestricted mobility, walks with difficulty, walks with orthopaedic aid, requires wheelchair, bedridden) at specified time points were reported. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208
Time frame: Study Baseline, Weeks 52, 104, 156 and 208
Bone pain was assessed as a part of Gaucher disease assessment in participants. Participants were categorized as none (no bone pain), very mild bone pain, mild bone pain, moderate bone pain, severe bone pain and extreme bone pain during the past 4 weeks at each specified visit. In this outcome measure, number of participants with different level of bone pain during the past 4 weeks at specified time points were reported. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208
Time frame: Study Baseline, Weeks 52, 104, 156 and 208
Bone crisis was assessed as a part of Gaucher disease assessment in participants. Acute, excruciating episodic bone pain is characteristic of Gaucher bone crisis, which typically causes periosteal elevation, elevated white blood cell count, fever, or debilitation lasting several days or longer and requires treatment with immobilization of the affected area, and opioid analgesics. Participants were categorized as 0= no bone crisis, 1= 1 bone crisis, 2= 2 bone crisis and >=3 = more than 3 bone crisis during the assessment period. In this outcome measure, number of participants with different bone crises levels at specified time points were reported. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Change From Current Study Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208
Time frame: Study Baseline, Weeks 52, 104, 156 and 208
Bone marrow burden (BMB) scores indicate the degree of bone marrow infiltration. BMB score was measured using MRI (magnetic resonance imaging (MRI), ranged from 0 (no abnormalities) to 8 points (severe disease) for the lumbar spine and from 0 (no abnormalities) to 8 points (severe disease) for the femurs. The total BMB score was calculated as the sum of scores for femur and lumbar spine regions which ranged from 0 (no abnormalities) to 16 (severe disease) points. A higher BMB score signified more severe bone marrow involvement. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Change From Eliglustat Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208
Time frame: Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study
Bone marrow burden (BMB) scores indicate the degree of bone marrow infiltration was measured using MRI, ranged from 0 (no abnormalities) to 8 points (severe disease) for the lumbar spine and from 0 (no abnormalities) to 8 points (severe disease) for the femurs. The total BMB score was calculated as the sum of scores for femur and lumbar spine regions which ranged from 0 (no abnormalities) to 16 (severe disease) points. A higher BMB score signified more severe bone marrow involvement. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.
Change From Current Study Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208
Time frame: Study Baseline, Weeks 52, 104, 156 and 208
Bone Mineral Density (BMD) measurements of the spine and bilateral femur were acquired by dual energy X-Ray absorptiometry (DXA) scan. Worst total femur at Baseline refers to the "worst" diseased left or right femur at Baseline. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208
Time frame: Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study
BMD measurements of the spine and bilateral femur were acquired by DXA scan. Worst total femur at Baseline refers to the "worst" diseased left or right femur at baseline. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.
Change From Current Study Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208
Time frame: Study Baseline, Weeks 52, 104, 156 and 208
BMD measurements of the spine and bilateral femur were acquired by DXA scan. The T-score bone density categories were: normal (score >-1), osteopenia (score -2.5 to <=-1), and osteoporosis (score <= -2.5). Worst total femur at Baseline refers to the "worst" diseased left or right femur at baseline. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208
Time frame: Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study
BMD measurements of the spine and bilateral femur were acquired by DXA scan. The T-score bone density categories were: normal (score >-1), osteopenia (score -2.5 to <=-1), and osteoporosis (score <= -2.5). Worst total femur at Baseline refers to the "worst" diseased left or right femur at baseline. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.
Change From Current Study Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208
Time frame: Study Baseline, Weeks 52, 104, 156 and 208
BMD measurements of the spine and bilateral femur were acquired by DXA scan. The Z-score bone density categories were: normal (score >-2) and below normal (score <=-2). Worst total femur at Baseline refers to the "worst" diseased left or right femur at baseline. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208
Time frame: Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study
BMD measurements of the spine and bilateral femur were acquired by DXA scan. The Z-score bone density categories are: normal (score >-2) and below normal (score <=-2). Worst total femur at Baseline refers to the "worst" diseased left or right femur at baseline. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.
Total Number of New or Worsening Osteonecrosis Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208
Time frame: Study Baseline, Weeks 52, 104, 156 and 208
Osteonecrosis was assessed by bone MRI and X-Ray for spine and by MRI for femur. Total number of new or worsening osteonecrosis events among all the participants with corresponding assessment at specified time points were reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Total Number of New or Worsening Fracture Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208
Time frame: Study Baseline, Weeks 52, 104, 156 and 208
Fracture was assessed by bone MRI and X-Ray for spine and by MRI for femur. Total number of new or worsening fracture events among all the participants with corresponding assessment at specified time points were reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Total Number of New or Worsening Infarcts Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208
Time frame: Study Baseline, Weeks 52, 104, 156 and 208
Infarcts were assessed by bone MRI and X-Ray for spine and by MRI for femur. Total number of new or worsening infarcts events among all the participants with corresponding assessment at specified time points were reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Total Number of New or Worsening Lytic Lesions Events for Spine at Study Baseline, Week 104, and 208
Time frame: Study Baseline, Weeks 104, and 208
Lytic Lesions were assessed by bone X-Ray for spine. Total number of new or worsening lytic lesions events among all the participants with corresponding assessment at specified time points were reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Observed Annual Incidence Rate for Spine and Femur Osteonecrosis at Week 52, 104, 156 and 208
Time frame: 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)
Observed annual incidence rate was estimated using the total number of events divided by the total years of follow-up in each specified year (for all participants). Osteonecrosis was assessed by bone MRI and X-Ray for spine and by MRI for femur.
Observed Annual Incidence Rate for Spine and Femur Fracture at Week 52, 104, 156 and 208
Time frame: 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)
Observed annual incidence rate was estimated using the total number of events divided by the total years of follow-up in each specified year (for all participants). Fracture was assessed by bone MRI and X-Ray for spine and by MRI for femur.
Observed Annual Incidence Rate for Spine and Femur Infarcts at Week 52, 104, 156 and 208
Time frame: 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)
Observed annual incidence rate was estimated using the total number of events divided by the total years of follow-up in each specified year (for all participants). Infarcts were assessed by bone MRI and X-Ray for spine and by MRI for femur.
Observed Annual Incidence Rate for Spine Lytic Lesion at Week 104, and 208
Time frame: For 104 Weeks (i.e., 2 year), and 208 Weeks (i.e., 4 years)
Observed annual incidence rate was estimated using the total number of events divided by the total years of follow-up in each specified year (for all participants). Lytic Lesions were assessed by bone X-Ray for spine.
Change From Current Study Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234
Time frame: Study Baseline, Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234
MIP-1β considered a biomarker of active bone disease, was assayed from plasma. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
Change From Eliglustat Baseline in Bone Biomarker Level: Macrophage Inflammatory Protein 1 Beta (MIP-1β) at Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234
Time frame: Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 of the current study
MIP-1β considered a biomarker of active bone disease, was assayed from plasma. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.
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
Time frame: Study Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234
P1NP, a marker of bone formation was assayed from plasma. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
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
Time frame: Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 of the current study
P1NP, a marker of bone formation was assayed from plasma. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.
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
Time frame: Study Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234
CTx, a marker of bone resorption was assayed from plasma. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.
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
Time frame: Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156,182, 208 and 234 of the current study
CTx, a marker of bone resorption was assayed from plasma. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.

9 secondary outcomes

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
Time frame: Study Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234
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
Time frame: Eliglustat Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study
Change From Current Study Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234
Time frame: Study Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234
Change From Eliglustat Baseline in GD1 Biomarker Levels: Glucosylceramide (GL-1) at Week 26, 52, 78, 104, 130, 156, 182, 208 and 234
Time frame: Eliglustat Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study
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
Time frame: Study Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234
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
Time frame: Eliglustat Baseline, Week 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study
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
Time frame: Study Baseline, Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234
Change From Eliglustat Baseline in SF-36 Scores at Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234
Time frame: Eliglustat Baseline, Weeks 26, 52, 78, 104, 130, 156, 182, 208 and 234 of the current study
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
Time frame: 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)

Results and safety

Results posted to the registry on 15 Jul 2022, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Eliglustat	31	27

Primary outcome: Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208

Time frame: Study Baseline, Weeks 52, 104, 156 and 208. Count of participants. Unit: Participants

Measure	Eliglustat n = 31
Unrestricted mobility: Study Baseline	31
Walks with difficulty: Study Baseline	0
Walks with orthopedic aid: Study Baseline	0
Requires wheelchair: Study Baseline	0
Bedridden: Study Baseline	0
Unrestricted mobility: Week 52	31
Walks with difficulty: Week 52	0
Walks with orthopedic aid: Week 52	0

Population analysed: Analysis was performed on FAS population. Here, "number analyzed" = participants with available data for each specified category. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.

Primary outcome: Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208

Time frame: Study Baseline, Weeks 52, 104, 156 and 208. Count of participants. Unit: Participants

Measure	Eliglustat n = 31
None: Study Baseline	28
Very Mild: Study Baseline	1
Mild: Study Baseline	1
Moderate: Study Baseline	1
Severe: Study Baseline	0
Extreme: Study Baseline	0
None: Week 52	29
Very Mild: Week 52	1

Primary outcome: Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208

Time frame: Study Baseline, Weeks 52, 104, 156 and 208. Count of participants. Unit: Participants

Measure	Eliglustat n = 31
Bone Crisis (0): Study Baseline	31
Bone Crisis (1): Study Baseline	0
Bone Crisis (2): Study Baseline	0
Bone Crisis (>=3): Study Baseline	0
Bone Crisis (0): Week 52	31
Bone Crisis (1): Week 52	0
Bone Crisis (2): Week 52	0
Bone Crisis (>=3): Week 52	0

Primary outcome: Change From Current Study Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208

Time frame: Study Baseline, Weeks 52, 104, 156 and 208. Mean (Standard Deviation). Unit: score on a scale

Measure	Eliglustat n = 31
Total BMB Score: Study Baseline	8.6667 (3.3860)
Total BMB Score: Week 52	8.5057 (2.6482)
Total BMB Score: Change at Week 52	-0.0256 (1.6969)
Total BMB Score: Week 104	9.1111 (2.9627)
Total BMB Score: Change at Week 104	1.2639 (2.0897)
Total BMB Score: Week 156	8.7821 (2.6178)
Total BMB Score: Change at Week 156	0.7101 (1.8404)
Total BMB Score: Week 208	8.9067 (2.8844)

9 secondary outcomes are reported in the full results.

Adverse events

Time frame: Time 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). Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Eliglustat	4 of 31 (12.9%)	12 of 31 (38.7%)	0 of 31

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 15 Jul 2022.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: Yes. Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org.

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Clinical Sciences & Operations study director

Sanofi

4 sites in 3 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Eliglustat, GZ385660 in people with Gaucher Disease, run by Genzyme, a Sanofi Company at 4 sites in 3 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 15 Jul 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in Russia, Canada, Tunisia. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 15 Jul 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208 (time frame: Study Baseline, Weeks 52, 104, 156 and 208). The study lists 25 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02536755.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Jul 2022.