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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT01074944
Registration number
NCT01074944
Ethics application status
Date submitted
23/02/2010
Date registered
24/02/2010
Date last updated
6/02/2017
Titles & IDs
Public title
A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)
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Scientific title
A Phase 3, Randomized, Multi-Center, Multi-National, Double-Blind Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Once Daily Versus Twice Daily Dosing of Genz-112638 in Patients With Gaucher Disease Type 1 Who Have Demonstrated Clinical Stability on a Twice Daily Dose of Genz-112638
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Secondary ID [1]
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2009-015811-42
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Secondary ID [2]
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GZGD03109
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Gaucher Disease
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Condition category
Condition code
Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Eliglustat tartrate
Experimental: Twice Daily (BID) Dose Regimen - Patients will receive either 50 mg BID or 100 mg BID
Experimental: Once Daily (QD) Dose Regimen - Patients will receive either 100 mg QD or 200 mg QD
Treatment: Drugs: Eliglustat tartrate
Oral Capsule in 50 mg or 100 mg dosages
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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PAP: Percentage of Participants Who Remained Stable for 52 Weeks During the PAP
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Assessment method [1]
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Participants were considered as stable if they met all of the following criteria: 1) no more than 2 bone crisis during PAP (with no more than 1 bone crisis during either the first 6 months or the later 6 months of the period), and were free of other clinically symptomatic bone disease during the entire 52-week PAP; 2) hemoglobin level not decreased >1.5 g/dL from Baseline for PAP; 3) platelet count not decreased >25% from Baseline for PAP; 4) spleen volume (in multiples of normal [MN]) did not increase >25% from Baseline for PAP; 5) liver volume (in MN) did not increase >20% from Baseline for PAP. Baseline for PAP was defined as the last assessment prior to randomization.
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Timepoint [1]
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PAP Baseline up to the end of PAP (Week 52)
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Secondary outcome [1]
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PAP: Mean Hemoglobin (Hb) Level at Baseline, Weeks 26 and 52
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Assessment method [1]
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Timepoint [1]
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Baseline, Week 26, Week 52
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Secondary outcome [2]
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PAP: Mean Platelet Count at Baseline, Weeks 26, 52
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Assessment method [2]
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Timepoint [2]
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Baseline, Week 26, Week 52
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Secondary outcome [3]
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PAP: Mean Spleen Volume at Baseline, Weeks 26, 52
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Assessment method [3]
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Timepoint [3]
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Baseline, Week 26, Week 52
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Secondary outcome [4]
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PAP: Mean Liver Volume at Baseline, Weeks 26, 52
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Assessment method [4]
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Timepoint [4]
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Baseline, Week 26 and Week 52
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Secondary outcome [5]
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PAP: Mean Biomarker (Chitotriosidase) Value at Baseline, Weeks 26 and Week 52
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Assessment method [5]
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Chitotriosidase biomarker was assayed from plasma.
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Timepoint [5]
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Baseline, Week 26, Week 52
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Secondary outcome [6]
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PAP: Mean Biomarker (GL-1 on DBS) Value at Baseline, Week 26 and Week 52
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Assessment method [6]
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GL-1 on DBS biomarker was assayed from dried blood spot (DBS).
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Timepoint [6]
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Baseline, Week 26 and week 52
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Secondary outcome [7]
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PAP: Mean Biomarker Macrophage Inflammatory Protein-1 Beta (MIP1-beta) Value at Baseline, Weeks 26, 52
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Assessment method [7]
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MIP1-beta biomarker was assayed from plasma.
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Timepoint [7]
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Baseline, Week 26, Week 52
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Secondary outcome [8]
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PAP: Bone Mineral Density (BMD) at Baseline and Week 52
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Assessment method [8]
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BMD measurements of the spine and bilateral femur were acquired by dual-energy x-ray absorptiometry (DXA) scan.
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Timepoint [8]
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Baseline, Week 52
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Secondary outcome [9]
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PAP: Total T-Scores for BMD at Baseline and Week 52
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Assessment method [9]
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Images of the spine and bilateral femur were obtained by DXA to determine T-score for each bone area and total bone mineral density. The T-score bone density categories were: normal (score >-1), osteopenia (score -2.5 to <=-1), and osteoporosis (score <= -2.5).
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Timepoint [9]
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Baseline, Week 52
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Secondary outcome [10]
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PAP: Total Z-scores for BMD at Baseline and Week 52
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Assessment method [10]
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Images of the spine and bilateral femur were obtained by DXA to determine Z-score for each bone area and total bone mineral density. The Z-score bone density categories are: normal (score >-2) and below normal (score <=-2).
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Timepoint [10]
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Baseline, Week 52
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Secondary outcome [11]
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PAP: Number of Participants With Mobility Status Asessments (MS) at Baseline, Weeks 26, and 52.
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Assessment method [11]
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Mobility, i.e., ability to walk was assessed as a part of Gaucher disease assessment in participants. In this outcome, 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.
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Timepoint [11]
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Baseline, Week 26 and Week 52
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Secondary outcome [12]
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PAP: Number of Participants With Bone Crises at Baseline, Weeks 26 and 52
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Assessment method [12]
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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 debilitation lasting several days or longer and requires treatment with immobilization, hydration, and opioid analgesics. Participants were categorized as 0= no bone crisis, 1= 1 bone crisis, and 2= 2 bone crises during the assessment period. In this outcome, number of participants with different bone crises levels at specified time points were reported.
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Timepoint [12]
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Baseline, Week 26, and Week 52
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Secondary outcome [13]
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PAP: Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Weeks 26 and 52
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Assessment method [13]
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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. In this outcome, number of participants with different level of bone pain during the past 4 weeks at specified time points were reported.
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Timepoint [13]
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Baseline, Week 26, and Week 52
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Secondary outcome [14]
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PAP: Total Bone Marrow Burden Score (BMB) at Baseline and Week 52
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Assessment method [14]
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BMB Score was measured using magnetic resonance imaging (MRI), range 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 score was calculated as the sum of scores for femur and lumbar spine regions which ranged from 0 (no abnormalities) -16 (severe disease) points. A higher BMB score signified more severe bone marrow involvement.
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Timepoint [14]
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Baseline, Week 52
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Secondary outcome [15]
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LIP: Mean Hemoglobin (Hb) Level at Baseline, Weeks 26, 52 and 78
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Assessment method [15]
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Timepoint [15]
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Baseline, Week 26, Week, 52, and Week 78
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Secondary outcome [16]
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LIP: Mean Platelet Count at Baseline, Weeks 26, 52 and 78
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Assessment method [16]
0
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Timepoint [16]
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Baseline, Week 26, Week 52, Week 78
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Secondary outcome [17]
0
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LIP: Mean Liver Volume at Baseline, Weeks 26, 52 and 78
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Assessment method [17]
0
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Timepoint [17]
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Baseline, Week 26, Week 52, Week 78
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Secondary outcome [18]
0
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LIP: Mean Spleen Volume at Baseline, Weeks 26, 52 and 78
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Assessment method [18]
0
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Timepoint [18]
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Baseline, Week 26, Week 52, Week 78
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Secondary outcome [19]
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LIP: Mean Biomarker (Chitotriosidase) Value at Baseline, Weeks 26, 52, and 78
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Assessment method [19]
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Chitotriosidase biomarker was assayed from plasma.
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Timepoint [19]
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Baseline, Week 26, Week 52 and Week 78
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Secondary outcome [20]
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LIP: Mean Biomarker (GL-1 on DBS) Value at Baseline, Week 26, Week 52, and Week 78
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Assessment method [20]
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GL-1 on DBS biomarker was assayed from dried blood spot.
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Timepoint [20]
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Baseline, Week 26, Week 52 and Week 78
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Secondary outcome [21]
0
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LIP: Mean Biomarker (MIP1-beta) Value at Baseline, Week 78
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Assessment method [21]
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MIP1-beta biomarker was assayed from plasma.
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Timepoint [21]
0
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Baseline and Week 78
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Secondary outcome [22]
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LIP: Number of Participants With Mobility Status (MS) at Baseline, Weeks 26, 52 and 78
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Assessment method [22]
0
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Mobility, i.e., ability to walk was assessed as a part of Gaucher disease assessment in participants. In this outcome, 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.
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Timepoint [22]
0
0
Baseline, Week 26, Week 52, Week 78
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Secondary outcome [23]
0
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LIP: Number of Participants With Bone Crises Assessment at Baseline, Weeks 26, 52 and 78
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Assessment method [23]
0
0
Bone crises was assessed as a part of Gaucher disease assessment in participants. Acute, excruciating episodic bone pain is characteristic of Gaucher bone crises, which typically causes debilitation lasting several days or longer and requires treatment with immobilization, hydration, and opioid analgesics. Participants were categorized as 0= no bone crisis, 1= 1 bone crisis, 2= 2 bone crises, 6= 6 bone crises, and 24= 24 bone crises during the assessment period. In this outcome, number of participants with different bone crises levels at specified time points were reported.
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Timepoint [23]
0
0
Baseline, Week 26, Week 52, Week 78
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Secondary outcome [24]
0
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LIP: Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Weeks 26, 52 and 78
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Assessment method [24]
0
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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. In this outcome, number of participants with different type of bone pain during the past 4 weeks at specified time points were reported.
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Timepoint [24]
0
0
Baseline, Week 26, Week 52, Week 78
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Secondary outcome [25]
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LTTP: Percentage of Participants Who Maintained a Stable Bone Criterion ,Hemoglobin Level, Platelet Count, Liver Volume and Spleen Volume at 1 Year and 2 Years
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Assessment method [25]
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Participant were considered as stable if they met the following criteria: hemoglobin level did not decrease >1.5 g/dL from baseline for PAP, platelet count does not decrease >25% below Baseline for PAP, liver volume does not increase >20% above Baseline for PAP, spleen volume does not increase >25% above Baseline for PAP. Baseline for PAP was defined as last available assessment prior to randomization.
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Timepoint [25]
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1 Year, 2 Years
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Secondary outcome [26]
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LTTP: Number of Participants With Mobility Status (MS) at Baseline, 1 Year and 2 Years
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Assessment method [26]
0
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Mobility, i.e., ability to walk was assessed as a part of Gaucher disease assessment in participants. In this outcome, 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.
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Timepoint [26]
0
0
Baseline, 1 year, and 2 years
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Secondary outcome [27]
0
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LTTP: Number of Participants With Bone Crises Assessment at Baseline, 1 Year and 2 Years
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Assessment method [27]
0
0
Bone crises was assessed as a part of Gaucher disease assessment in participants. Acute, excruciating episodic bone pain is characteristic of Gaucher bone crises, which typically causes debilitation lasting several days or longer and requires treatment with immobilization, hydration, and opioid analgesics. Participants were categorized as 0= no bone crises, 1= 1 bone crisis during the assessment period. In this outcome, number of participants with different bone crises levels at specified time points were reported.
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Timepoint [27]
0
0
Baseline, 1 year and 2 years
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Secondary outcome [28]
0
0
LTTP: Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, 1 Year, and 2 Years
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Assessment method [28]
0
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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. In this outcome, number of participants with different level of bone pain during the past 4 weeks at specified time points were reported.
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Timepoint [28]
0
0
Baseline, 1 year and 2 years
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Secondary outcome [29]
0
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LTTP: Bone Mineral Density (BMD) at Baseline, 1 Year, and 2 Years
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Assessment method [29]
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BMD measurements of the spine and bilateral femur were acquired by DXA scan.
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Timepoint [29]
0
0
Baseline, 1 year, and 2 years
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Secondary outcome [30]
0
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LTTP: Total T-Scores for BMD at Baseline, 1 Year, and 2 Years
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Assessment method [30]
0
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Images of the spine and bilateral femur were obtained by DXA to determine T-score for each bone area and total bone mineral density. The T-score bone density categories were: normal (score >-1), osteopenia (score -2.5 to <=-1), and osteoporosis (score <= -2.5).
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Timepoint [30]
0
0
Baseline, 1 year, and 2 years
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Secondary outcome [31]
0
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LTTP: Total Z-scores for BMD at Baseline, 1 Year, and 2 Years
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Assessment method [31]
0
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Images of the spine and bilateral femur were obtained by DXA to determine Z-score for each bone area and total bone mineral density. The Z-score bone density categories are: normal (score >-2) and below normal (score <=-2).
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Timepoint [31]
0
0
Baseline, 1 year, and 2 years
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Secondary outcome [32]
0
0
LTTP: Total Bone Marrow Burden Score (BMB) at Baseline, 1 Year, and 2 Years
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Assessment method [32]
0
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BMB Score was measured using MRI, range 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 score was calculated as the sum of scores for femur and lumbar spine regions which ranged from 0 (no abnormalities) -16 (severe disease) points. A higher BMB score signified more severe bone marrow involvement.
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Timepoint [32]
0
0
Baseline, 1 year, and 2 years
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Secondary outcome [33]
0
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LTTP: Mean Biomarker (Chitotriosidase) Value at Baseline, 1 Year, and 2 Years
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Assessment method [33]
0
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Chitotriosidase biomarker was assayed from plasma.
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Timepoint [33]
0
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Baseline, 1 year, and 2 years
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Secondary outcome [34]
0
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LTTP: Mean Biomarker (GL-1 on DBS) Value at Baseline, 1 Year, and 2 Years
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Assessment method [34]
0
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GL-1 on DBS biomarker was assayed from dried blood spot.
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Timepoint [34]
0
0
Baseline, 1 year, and 2 years
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Secondary outcome [35]
0
0
LTTP: Mean Biomarker (MIP1-beta) Value at Baseline, 1 Year, and 2 Years
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Assessment method [35]
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MIP1-beta biomarker was assayed from plasma.
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Timepoint [35]
0
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Baseline, 1 year, and 2 years
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Eligibility
Key inclusion criteria
- The participant who was willing and provided signed informed consent prior to any
study-related procedures.
- The participant was =18 years of age.
- The participant diagnosed with GD 1 confirmed by a documented deficiency of acid
ß-glucosidase activity by enzyme assay.
- Female participants of childbearing potential had a documented negative pregnancy test
prior to administration of the first dose of eliglustat tartrate (Genz-112638) in this
study. In addition, all female participants of childbearing potential used a medically
accepted form of contraception throughout the study, i.e., either a barrier method or
hormonal contraceptive with norethindrone and ethinyl estradiol or similar active
components
- The participant met all of the following criteria at the time of screening: hemoglobin
level =9 g/dL (mean of 2 measurements); platelet count =70,000/mm^3 (mean of 2
measurements); spleen volume =25 multiples of normal (MN); liver volume =2.0 MN.
- The participant consented to provide a blood sample for genotyping for Gaucher disease
and for CYP2D6 to categorize the participant's predicted rate of metabolism, if these
genotyping results were not already available for the participant.
- The participant was willing to abstain from consumption of grapefruit, grapefruit
juice, or grapefruit products for 72 hours prior to administration of the first dose
of Genz-112638 and throughout the duration of the study.
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
- The participant was participating in GZGD02607 study, "A Phase 3, Randomized,
Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the
Efficacy and Safety of Genz-112638 in Participants with GD1 who have been Stabilized
with Cerezyme ® ," or was eligible for inclusion in GZGD02607 (while enrollment was
ongoing) and had access to a physician participating in GZGD02607, or the participant
was participating in GZGD02507 study, "A Phase 3, Randomized, Double-Blind,
Placebo-Controlled, Multi-Center Study Confirming the Efficacy and Safety of
Genz-112638 in Participants with GD1," or was eligible for inclusion in GZGD02507
(while enrollment was ongoing) and had access to a physician participating in
GZGD02507.
- The participant received miglustat within 6 months prior to administration of the
first dose of Genz-112638 in this study.
- The participant had a partial or total splenectomy within 3 years prior to
randomization.
- The participant received pharmacological chaperones or miglustat within 6 months prior
to administration of the first dose of eliglustat tartrate (Genz-112638) in this
study.
- The participant had any evidence of neurologic disorder (e.g., peripheral neuropathy,
tremor, seizures, Parkinsonism or cognitive impairment) or pulmonary involvement
(e.g., pulmonary hypertension) as related to Gaucher disease.
- The participant was transfusion-dependent.
- The participant had a documented deficiency of iron, vitamin B-12, or folate that
requires treatment not yet initiated or, if initiated, the participant had not been
stable under treatment for at least 3 months prior to administration of the first dose
of Genz-112638 in this study.
- The participant had documented prior esophageal varices or clinically significant
liver infarction or current liver enzymes (alanine transaminase [ALT]/aspartate
aminotransferase [AST]) or total bilirubin >2 times the upper limit of normal (ULN),
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 (including hypokalaemia or hypomagnesemia), or psychiatric
disease, other medical conditions, or serious intercurrent illnesses that, in the
opinion of the Investigator, precluded participation in the study.
- The participant was known to have any of the following: Clinically significant
coronary artery disease including history of myocardial infarction [MI] or ongoing
signs or symptoms consistent with cardiac ischemia or heart failure; or clinically
significant arrhythmias or conduction defect such as 2nd or 3rd degree AV block,
complete bundle branch block, prolonged QTc interval, or sustained ventricular
tachycardia (VT).
- The participant who tested positive for the human immunodeficiency virus (HIV)
antibody, Hepatitis C antibody, or Hepatitis B surface antigen.
- The participant received an investigational product (other than eliglustat tartrate
(Genz-112638)) within 30 days prior to administration of the first dose of eliglustat
tartrate (Genz-112638) in this study.
- The participant was scheduled for in-participant hospitalization, including elective
surgery, during the study.
- The participant had a history of cancer, with the exception of basal cell carcinoma,
within 5 years prior to administration of the first dose of Genz-112638 in this study.
- The participant was pregnant or lactating.
- The participant had received any medication that may cause QTc interval prolongation
within 30 days prior to the first dose of Genz-112638. Exception: Diphenhydramine
(Benadryl) or other medications used as premedication for ERT infusions were allowed
up to 7 days prior to the first dose of Genz-112638.
- The participant had received for the first time (i.e., the participant was not already
chronically using) any of the following medications within 30 days prior to the first
dose of Genz-112638:
- Strong inhibitors of CYP2D6 or CYP3A4;
- Inducers of CYP3A4. Exception: Premedications for ERT infusions were allowed up
to 7 days prior to the first dose of Genz-112638.
- The participant was a CYP2D6 non-poor metabolizer or an indeterminate metabolizer with
one allele identified as active who was chronically receiving both a strong
competitive inhibitor of CYP2D6 and a strong competitive inhibitor of CYP3A4 and for
whom no reasonable alternative medication exists. or
- The participant was a CYP2D6 poor metabolizer or an indeterminate metabolizer with
neither allele known to be active who was chronically receiving a strong competitive
inhibitor of CYP3A4 and for whom no reasonable alternative medication exists.
Exception for both cases: Premedications for ERT infusions were allowed up to 7 days prior
to the first dose of Genz-112638.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/06/2010
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
1/10/2015
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Sample size
Target
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Accrual to date
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Final
170
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
0
0
Royal Prince Alfred Hospital - Camperdown
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Recruitment hospital [2]
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0
Monash Medical Centre - Clayton, VIC
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Recruitment hospital [3]
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0
Royal Perth Hospital - Perth, WA
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Recruitment postcode(s) [1]
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0
- Camperdown
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Recruitment postcode(s) [2]
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0
- Clayton, VIC
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Recruitment postcode(s) [3]
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0
- Perth, WA
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Recruitment outside Australia
Country [1]
0
0
United States of America
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State/province [1]
0
0
California
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Country [2]
0
0
United States of America
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State/province [2]
0
0
Connecticut
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Country [3]
0
0
United States of America
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State/province [3]
0
0
Georgia
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Country [4]
0
0
United States of America
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State/province [4]
0
0
Illinois
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Country [5]
0
0
United States of America
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State/province [5]
0
0
New York
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Country [6]
0
0
United States of America
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State/province [6]
0
0
Pennsylvania
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Country [7]
0
0
United States of America
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State/province [7]
0
0
Utah
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Country [8]
0
0
United States of America
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State/province [8]
0
0
Virginia
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Country [9]
0
0
Austria
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State/province [9]
0
0
Vienna
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Country [10]
0
0
Brazil
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State/province [10]
0
0
Belo Horizonte
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Country [11]
0
0
Brazil
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State/province [11]
0
0
Brasília
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Country [12]
0
0
Brazil
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State/province [12]
0
0
Campinas
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Lund
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Funding & Sponsors
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Name
Genzyme, a Sanofi Company
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Summary
Brief summary
The primary objective of this study was to evaluate the efficacy and safety of once daily
(QD) versus twice daily (BID) dosing of eliglustat tartrate (Genz-112638) in participants
with Gaucher disease type 1 who had demonstrated clinical stability on BID dosing of
eliglustat tartrate (Genz-112638). The secondary objective was to evaluate the
pharmacokinetics (PK) of Genz-99067 when eliglustat tartrate (Genz-112638) was administered
QD and BID in participants with Gaucher disease type 1 who had demonstrated clinical
stability on BID dosing of eliglustat tartrate (Genz-112638).
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Trial website
https://clinicaltrials.gov/ct2/show/NCT01074944
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Trial related presentations / publications
McEachern KA, Fung J, Komarnitsky S, Siegel CS, Chuang WL, Hutto E, Shayman JA, Grabowski GA, Aerts JM, Cheng SH, Copeland DP, Marshall J. A specific and potent inhibitor of glucosylceramide synthase for substrate inhibition therapy of Gaucher disease. Mol Genet Metab. 2007 Jul;91(3):259-67. doi: 10.1016/j.ymgme.2007.04.001. Epub 2007 May 16.
Lukina E, Watman N, Arreguin EA, Banikazemi M, Dragosky M, Iastrebner M, Rosenbaum H, Phillips M, Pastores GM, Rosenthal DI, Kaper M, Singh T, Puga AC, Bonate PL, Peterschmitt MJ. A phase 2 study of eliglustat tartrate (Genz-112638), an oral substrate reduction therapy for Gaucher disease type 1. Blood. 2010 Aug 12;116(6):893-9. doi: 10.1182/blood-2010-03-273151. Epub 2010 May 3.
Lukina E, Watman N, Arreguin EA, Dragosky M, Iastrebner M, Rosenbaum H, Phillips M, Pastores GM, Kamath RS, Rosenthal DI, Kaper M, Singh T, Puga AC, Peterschmitt MJ. Improvement in hematological, visceral, and skeletal manifestations of Gaucher disease type 1 with oral eliglustat tartrate (Genz-112638) treatment: 2-year results of a phase 2 study. Blood. 2010 Nov 18;116(20):4095-8. doi: 10.1182/blood-2010-06-293902. Epub 2010 Aug 16. Erratum In: Blood. 2011 May 19;117(20):5551.
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Public notes
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Contacts
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Genzyme, a Sanofi Company
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Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT01074944
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