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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT00943111




Registration number
NCT00943111
Ethics application status
Date submitted
20/07/2009
Date registered
21/07/2009
Date last updated
11/10/2016

Titles & IDs
Public title
A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)
Scientific title
A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients With Gaucher Disease Type 1 Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)
Secondary ID [1] 0 0
2008-005223-28
Secondary ID [2] 0 0
GZGD02607
Universal Trial Number (UTN)
Trial acronym
ENCORE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Gaucher Disease, Type 1 0 0
Gaucher Disease, Type 1 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

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

Experimental: Investigational - Eliglustat tartrate

Active Comparator: Imiglucerase -

Experimental: Investigational - Eliglustat tartrate

Active Comparator: Imiglucerase -


Treatment: Drugs: Eliglustat tartrate
Primary analysis period (PAP): Eliglustat tartrate capsule 50 milligram (mg) twice daily (BID) orally from Day 1 to Week 4 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 8, and then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 52. Dose adjustments after Week 4 and Week 8 were based on Genz-99067 (active moiety of eliglustat tartrate in plasma) trough plasma concentrations. If Genz-99067 trough plasma concentration was less than (<) 5 nanogram per milliliter [ng/mL] next higher dose was administered whereas if Genz-99067 trough plasma concentration was greater than or equal to (>=) 5 ng/mL same dose was continued. Pharmacokinetic (PK) assessment at Week 2 and 6 were used for dose adjustment after Week 4 and Week 8, respectively.
Long-term treatment period (LTTP): Participants originally randomized to eliglustat in PAP continued to receive eliglustat dose, based on their Genz 99067 plasma trough concentration at Week 6.

Treatment: Drugs: Imiglucerase
PAP: Imiglucerase intravenous infusion every other week (q2w) up to Week 52 in doses equivalent to participant's past ERT dose prior to any unanticipated treatment interruption, dose reduction, or regimen change.
LTTP: Participants originally randomized to imiglucerase received eliglustat tartrate capsule 50 mg BID orally from Week 52+1 Day to Week 56 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 60, and then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to 5 years. The dose adjustments after Week 56 and Week 60 were based on Genz-99067 (active moiety of eliglustat tartrate in plasma) trough plasma concentrations. If Genz-99067 trough plasma concentration was <5 ng/mL the next higher dose was administered whereas if the Genz-99067 trough plasma concentration was >=5 ng/mL the same dose was continued. The PK assessment at Week 54 and Week 58 were used for dose adjustment after Week 56 and Week 60, respectively.

Treatment: Drugs: Eliglustat tartrate
Primary analysis period (PAP): Eliglustat tartrate capsule 50 milligram (mg) twice daily (BID) orally from Day 1 to Week 4 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 8, and then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 52. Dose adjustments after Week 4 and Week 8 were based on Genz-99067 (active moiety of eliglustat tartrate in plasma) trough plasma concentrations. If Genz-99067 trough plasma concentration was less than (<) 5 nanogram per milliliter [ng/mL] next higher dose was administered whereas if Genz-99067 trough plasma concentration was greater than or equal to (>=) 5 ng/mL same dose was continued. Pharmacokinetic (PK) assessment at Week 2 and 6 were used for dose adjustment after Week 4 and Week 8, respectively.
Long-term treatment period (LTTP): Participants originally randomized to eliglustat in PAP continued to receive eliglustat dose, based on their Genz 99067 plasma trough concentration at Week 6.

Treatment: Drugs: Imiglucerase
PAP: Imiglucerase intravenous infusion every other week (q2w) up to Week 52 in doses equivalent to participant's past ERT dose prior to any unanticipated treatment interruption, dose reduction, or regimen change.
LTTP: Participants originally randomized to imiglucerase received eliglustat tartrate capsule 50 mg BID orally from Week 52+1 Day to Week 56 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 60, and then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to 5 years. The dose adjustments after Week 56 and Week 60 were based on Genz-99067 (active moiety of eliglustat tartrate in plasma) trough plasma concentrations. If Genz-99067 trough plasma concentration was <5 ng/mL the next higher dose was administered whereas if the Genz-99067 trough plasma concentration was >=5 ng/mL the same dose was continued. The PK assessment at Week 54 and Week 58 were used for dose adjustment after Week 56 and Week 60, respectively.

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percentage of Participants Who Remained Stable for 52 Weeks During the Primary Analysis Period - For a participant to be classified as stable, the participant must have remained stable in hematological parameters (hemoglobin levels and platelet counts) and organ volumes (spleen, when applicable, and liver volumes in multiples of normal [MN]). Stable hematological parameters were defined as hemoglobin level did not decrease more than (>) 1.5 gram per deciliter (g/dL) from baseline and platelet count did not decrease >25% from baseline. Stable organ volumes were defined as spleen volume (in MN) did not increase >25% from baseline, if applicable, and liver volume (in MN) did not increase >20% from baseline.
Timepoint [1] 0 0
Baseline up to Week 52
Primary outcome [2] 0 0
Percentage of Participants Who Remained Stable Annually for 4 Years During the LTTP - For a participant to be classified as stable, the participant must have remained stable in hematological parameters (hemoglobin levels and platelet counts) and organ volumes (spleen, when applicable, and liver volumes in MN). Stable hematological parameters were defined as hemoglobin level did not decrease >1.5 g/dL from baseline and platelet count did not decrease >25% from baseline. Stable organ volumes were defined as spleen volume (in MN) did not increase >25% from baseline, if applicable, and liver volume did not increase >20% from baseline.
Timepoint [2] 0 0
Week 52 up to week 208
Primary outcome [3] 0 0
Percentage of Participants Who Remained Stable for 52 Weeks During the Primary Analysis Period - For a participant to be classified as stable, the participant must have remained stable in hematological parameters (hemoglobin levels and platelet counts) and organ volumes (spleen, when applicable, and liver volumes in multiples of normal [MN]). Stable hematological parameters were defined as hemoglobin level did not decrease more than (>) 1.5 gram per deciliter (g/dL) from baseline and platelet count did not decrease >25% from baseline. Stable organ volumes were defined as spleen volume (in MN) did not increase >25% from baseline, if applicable, and liver volume (in MN) did not increase >20% from baseline.
Timepoint [3] 0 0
Baseline up to Week 52
Primary outcome [4] 0 0
Percentage of Participants Who Remained Stable Annually for 4 Years During the LTTP - For a participant to be classified as stable, the participant must have remained stable in hematological parameters (hemoglobin levels and platelet counts) and organ volumes (spleen, when applicable, and liver volumes in MN). Stable hematological parameters were defined as hemoglobin level did not decrease >1.5 g/dL from baseline and platelet count did not decrease >25% from baseline. Stable organ volumes were defined as spleen volume (in MN) did not increase >25% from baseline, if applicable, and liver volume did not increase >20% from baseline.
Timepoint [4] 0 0
Week 52 up to week 208
Secondary outcome [1] 0 0
Total T-Scores for Bone Mineral Density - Images of the spine and bilateral femur were obtained by dual energy X-Ray absorptiometry (DXA) to determine T-score for each bone area and total bone mineral density. T-score compares participant's bone density with that of healthy young participant. The T-score bone density categories are: normal (score greater than [>]-1), osteopenia (score -2.5 to less than or equal to [<=] -1), and osteoporosis (score <= -2.5).
Timepoint [1] 0 0
Baseline
Secondary outcome [2] 0 0
Absolute Change From Baseline in Total T-Scores for Bone Mineral Density at Week 52 - Images of the spine and bilateral femur were obtained by DXA to determine T-score for each bone area and total bone mineral density. T-score compares participant's bone density with that of healthy young participant. The T-score bone density categories are: normal (score >-1), osteopenia (score -2.5 to <=-1), and osteoporosis (score <= -2.5). Absolute change = T-score at Week 52 minus T-score at baseline.
Timepoint [2] 0 0
Baseline, Week 52
Secondary outcome [3] 0 0
Total Z-Scores for Bone Mineral Density - 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).
Timepoint [3] 0 0
Baseline
Secondary outcome [4] 0 0
Absolute Change From Baseline in Total Z-Scores for Bone Mineral Density at Week 52 - 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). Absolute change = Z-score at Week 52 minus Z-score at baseline.
Timepoint [4] 0 0
Baseline, Week 52
Secondary outcome [5] 0 0
Hemoglobin Level
Timepoint [5] 0 0
Baseline
Secondary outcome [6] 0 0
Absolute Change From Baseline in Hemoglobin Levels at Week 52 - Absolute change = hemoglobin level at Week 52 minus hemoglobin level at baseline.
Timepoint [6] 0 0
Baseline, Week 52
Secondary outcome [7] 0 0
Percent Change From Baseline in Platelet Counts at Week 52 - Percent change in platelet counts = ([platelet count at Week 52 minus platelet count at baseline] divided by [platelet count at baseline]) multiplied by 100.
Timepoint [7] 0 0
Baseline, Week 52
Secondary outcome [8] 0 0
Percent Change From Baseline in Spleen Volume (MN) at Week 52 - Percent change in spleen volume = ([spleen volume at Week 52 minus spleen volume at baseline] divided by [spleen volume at baseline]) multiplied by 100, where all volumes are in MN.
Timepoint [8] 0 0
Baseline, Week 52
Secondary outcome [9] 0 0
Percent Change From Baseline in Liver Volume (in MN) at Week 52 - Percent change in liver volume = ([liver volume at Week 52 minus liver volume at baseline] divided by [liver volume at baseline]) multiplied by 100, where all volumes are in multiples of normal.
Timepoint [9] 0 0
Baseline, Week 52
Secondary outcome [10] 0 0
Absolute Change From Baseline in Total T-Scores for Bone Mineral Density at Week 208 - Images of the spine and bilateral femur were obtained by DXA to determine T-score for each bone area and total bone mineral density. T-score compares participant's bone density with that of healthy young participant. The T-score bone density categories are: normal (score >-1), osteopenia (score -2.5 to <=-1), and osteoporosis (score <= -2.5). Absolute change = T-score at Week 208 minus T-score at baseline.
Timepoint [10] 0 0
Baseline, Week 208
Secondary outcome [11] 0 0
Absolute Change From Baseline in Total Z-Scores for Bone Mineral Density at Week 208 - 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). Absolute change = Z-score at Week 208 minus Z-score at baseline.
Timepoint [11] 0 0
Baseline, Week 208
Secondary outcome [12] 0 0
Absolute Change From Baseline in Hemoglobin Levels at Week 208 - Absolute change = hemoglobin level at Week 208 minus hemoglobin level at baseline.
Timepoint [12] 0 0
Baseline, Week 208
Secondary outcome [13] 0 0
Percent Change From Baseline in Platelet Counts at Week 208 - Percent change in platelet counts = ([platelet count at Week 208 minus platelet count at baseline] divided by [platelet count at baseline]) multiplied by 100.
Timepoint [13] 0 0
Baseline, Week 208
Secondary outcome [14] 0 0
Percent Change From Baseline in Spleen Volume (in MN) at Week 208 - Percent change in spleen volume = ([spleen volume at Week 208 minus spleen volume at baseline] divided by [spleen volume at baseline]) multiplied by 100, where all volumes are in MN.
Timepoint [14] 0 0
Baseline, Week 208
Secondary outcome [15] 0 0
Percent Change From Baseline in Liver Volume (in MN) at Week 208 - Percent change in liver volume = ([liver volume at Week 208 minus liver volume at baseline] divided by [liver volume at baseline]) multiplied by 100, where all volumes are in multiples of normal.
Timepoint [15] 0 0
Baseline, Week 208
Secondary outcome [16] 0 0
Total T-Scores for Bone Mineral Density - Images of the spine and bilateral femur were obtained by dual energy X-Ray absorptiometry (DXA) to determine T-score for each bone area and total bone mineral density. T-score compares participant's bone density with that of healthy young participant. The T-score bone density categories are: normal (score greater than [>]-1), osteopenia (score -2.5 to less than or equal to [<=] -1), and osteoporosis (score <= -2.5).
Timepoint [16] 0 0
Baseline
Secondary outcome [17] 0 0
Absolute Change From Baseline in Total T-Scores for Bone Mineral Density at Week 52 - Images of the spine and bilateral femur were obtained by DXA to determine T-score for each bone area and total bone mineral density. T-score compares participant's bone density with that of healthy young participant. The T-score bone density categories are: normal (score >-1), osteopenia (score -2.5 to <=-1), and osteoporosis (score <= -2.5). Absolute change = T-score at Week 52 minus T-score at baseline.
Timepoint [17] 0 0
Baseline, Week 52
Secondary outcome [18] 0 0
Total Z-Scores for Bone Mineral Density - 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).
Timepoint [18] 0 0
Baseline
Secondary outcome [19] 0 0
Absolute Change From Baseline in Total Z-Scores for Bone Mineral Density at Week 52 - 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). Absolute change = Z-score at Week 52 minus Z-score at baseline.
Timepoint [19] 0 0
Baseline, Week 52
Secondary outcome [20] 0 0
Hemoglobin Level
Timepoint [20] 0 0
Baseline
Secondary outcome [21] 0 0
Absolute Change From Baseline in Hemoglobin Levels at Week 52 - Absolute change = hemoglobin level at Week 52 minus hemoglobin level at baseline.
Timepoint [21] 0 0
Baseline, Week 52
Secondary outcome [22] 0 0
Percent Change From Baseline in Platelet Counts at Week 52 - Percent change in platelet counts = ([platelet count at Week 52 minus platelet count at baseline] divided by [platelet count at baseline]) multiplied by 100.
Timepoint [22] 0 0
Baseline, Week 52
Secondary outcome [23] 0 0
Percent Change From Baseline in Spleen Volume (MN) at Week 52 - Percent change in spleen volume = ([spleen volume at Week 52 minus spleen volume at baseline] divided by [spleen volume at baseline]) multiplied by 100, where all volumes are in MN.
Timepoint [23] 0 0
Baseline, Week 52
Secondary outcome [24] 0 0
Percent Change From Baseline in Liver Volume (in MN) at Week 52 - Percent change in liver volume = ([liver volume at Week 52 minus liver volume at baseline] divided by [liver volume at baseline]) multiplied by 100, where all volumes are in multiples of normal.
Timepoint [24] 0 0
Baseline, Week 52
Secondary outcome [25] 0 0
Absolute Change From Baseline in Total T-Scores for Bone Mineral Density at Week 208 - Images of the spine and bilateral femur were obtained by DXA to determine T-score for each bone area and total bone mineral density. T-score compares participant's bone density with that of healthy young participant. The T-score bone density categories are: normal (score >-1), osteopenia (score -2.5 to <=-1), and osteoporosis (score <= -2.5). Absolute change = T-score at Week 208 minus T-score at baseline.
Timepoint [25] 0 0
Baseline, Week 208
Secondary outcome [26] 0 0
Absolute Change From Baseline in Total Z-Scores for Bone Mineral Density at Week 208 - 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). Absolute change = Z-score at Week 208 minus Z-score at baseline.
Timepoint [26] 0 0
Baseline, Week 208
Secondary outcome [27] 0 0
Absolute Change From Baseline in Hemoglobin Levels at Week 208 - Absolute change = hemoglobin level at Week 208 minus hemoglobin level at baseline.
Timepoint [27] 0 0
Baseline, Week 208
Secondary outcome [28] 0 0
Percent Change From Baseline in Platelet Counts at Week 208 - Percent change in platelet counts = ([platelet count at Week 208 minus platelet count at baseline] divided by [platelet count at baseline]) multiplied by 100.
Timepoint [28] 0 0
Baseline, Week 208
Secondary outcome [29] 0 0
Percent Change From Baseline in Spleen Volume (in MN) at Week 208 - Percent change in spleen volume = ([spleen volume at Week 208 minus spleen volume at baseline] divided by [spleen volume at baseline]) multiplied by 100, where all volumes are in MN.
Timepoint [29] 0 0
Baseline, Week 208
Secondary outcome [30] 0 0
Percent Change From Baseline in Liver Volume (in MN) at Week 208 - Percent change in liver volume = ([liver volume at Week 208 minus liver volume at baseline] divided by [liver volume at baseline]) multiplied by 100, where all volumes are in multiples of normal.
Timepoint [30] 0 0
Baseline, Week 208

Eligibility
Key inclusion criteria
- The participant (and/or their parent/legal guardian) was willing and able to provide
signed informed consent prior to any study-related procedures to be performed

- The participant was at least 18 years old at the time of randomization

- The participant had a confirmed diagnosis of Gaucher disease type 1

- The participant had received treatment with ERT for at least 3 years. Within the 9
months prior to randomization, the participant had received a total monthly dose of 30
to 130 Units/kilogram for at least 6 months

- The participant had reached Gaucher disease therapeutic goals prior to randomization

- Female participants of childbearing potential must have had a documented negative
pregnancy test prior to dosing. In addition, all female participants of childbearing
potential must use a medically accepted form of contraception throughout the study
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- The participant had a partial or total splenectomy within 3 years prior to
randomization

- The participant had received substrate reduction therapies for Gaucher disease within
6 months prior to randomization

- The participant had Gaucher disease type 2 or 3 or was suspected of having Gaucher
disease type 3

- The participant had any clinically significant disease, other than Gaucher disease,
including cardiovascular, renal, hepatic, gastrointestinal (GI), pulmonary,
neurologic, endocrine, metabolic (e.g. hypokalemia, hypomagnesemia), or psychiatric
disease, other medical conditions, or serious intercurrent illnesses that may confound
the study results or, in the opinion of the Investigator, may preclude participation
in the study

- The participant had tested positive for the human immunodeficiency virus (HIV)
antibody, Hepatitis C antibody, or Hepatitis B surface antigen

- The participant had received an investigational product within 30 days prior to
randomization

- The participant was pregnant or lactating

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



Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Royal Perth Hospital - Perth, WA
Recruitment postcode(s) [1] 0 0
- Perth, WA
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Connecticut
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Georgia
Country [6] 0 0
United States of America
State/province [6] 0 0
Illinois
Country [7] 0 0
United States of America
State/province [7] 0 0
Iowa
Country [8] 0 0
United States of America
State/province [8] 0 0
New York
Country [9] 0 0
United States of America
State/province [9] 0 0
North Carolina
Country [10] 0 0
United States of America
State/province [10] 0 0
Ohio
Country [11] 0 0
United States of America
State/province [11] 0 0
Pennsylvania
Country [12] 0 0
United States of America
State/province [12] 0 0
Virginia
Country [13] 0 0
Argentina
State/province [13] 0 0
Buenos Aires
Country [14] 0 0
Brazil
State/province [14] 0 0
Curitiba
Country [15] 0 0
Brazil
State/province [15] 0 0
Rio de Janeiro
Country [16] 0 0
Brazil
State/province [16] 0 0
São Paulo
Country [17] 0 0
Canada
State/province [17] 0 0
Toronto Ontario
Country [18] 0 0
Egypt
State/province [18] 0 0
Cairo
Country [19] 0 0
France
State/province [19] 0 0
Clichy
Country [20] 0 0
Germany
State/province [20] 0 0
Berlin
Country [21] 0 0
Germany
State/province [21] 0 0
Hamburg
Country [22] 0 0
Germany
State/province [22] 0 0
Oberhausen
Country [23] 0 0
Italy
State/province [23] 0 0
Firenze
Country [24] 0 0
Italy
State/province [24] 0 0
Udine
Country [25] 0 0
Russian Federation
State/province [25] 0 0
Moscow
Country [26] 0 0
Spain
State/province [26] 0 0
Zaragoza
Country [27] 0 0
United Kingdom
State/province [27] 0 0
Cambridge

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

Ethics approval
Ethics application status

Summary
Brief summary
This Phase 3 study was designed to confirm the efficacy and safety of eliglustat tartrate
(Genz-112638) in participants with Gaucher disease type 1 who had reached therapeutic goals
with enzyme replacement therapy (ERT).
Trial website
https://clinicaltrials.gov/show/NCT00943111
Trial related presentations / publications
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.
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. Epub 2007 May 16.
Kamath RS, Lukina E, Watman N, Dragosky M, Pastores GM, Arreguin EA, Rosenbaum H, Zimran A, Aguzzi R, Puga AC, Norfleet AM, Peterschmitt MJ, Rosenthal DI. Skeletal improvement in patients with Gaucher disease type 1: a phase 2 trial of oral eliglustat. Skeletal Radiol. 2014 Oct;43(10):1353-60. doi: 10.1007/s00256-014-1891-9. Epub 2014 May 10.
Lukina E, Watman N, Dragosky M, Pastores GM, Arreguin EA, Rosenbaum H, Zimran A, Angell J, Ross L, Puga AC, Peterschmitt JM. Eliglustat, an investigational oral therapy for Gaucher disease type 1: Phase 2 trial results after 4 years of treatment. Blood Cells Mol Dis. 2014 Dec;53(4):274-6. doi: 10.1016/j.bcmd.2014.04.002. Epub 2014 May 15.
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.
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. Epub 2007 May 16.
Kamath RS, Lukina E, Watman N, Dragosky M, Pastores GM, Arreguin EA, Rosenbaum H, Zimran A, Aguzzi R, Puga AC, Norfleet AM, Peterschmitt MJ, Rosenthal DI. Skeletal improvement in patients with Gaucher disease type 1: a phase 2 trial of oral eliglustat. Skeletal Radiol. 2014 Oct;43(10):1353-60. doi: 10.1007/s00256-014-1891-9. Epub 2014 May 10.
Lukina E, Watman N, Dragosky M, Pastores GM, Arreguin EA, Rosenbaum H, Zimran A, Angell J, Ross L, Puga AC, Peterschmitt JM. Eliglustat, an investigational oral therapy for Gaucher disease type 1: Phase 2 trial results after 4 years of treatment. Blood Cells Mol Dis. 2014 Dec;53(4):274-6. doi: 10.1016/j.bcmd.2014.04.002. Epub 2014 May 15.
Public notes

Contacts
Principal investigator
Name 0 0
Medical Monitor
Address 0 0
Genzyme, a Sanofi Company
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications