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A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE) (ENCORE)

Primary Purpose

Gaucher Disease, Type 1

Status

Completed

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

Eliglustat tartrate

Imiglucerase

About this trial

This is an interventional treatment trial for Gaucher Disease, Type 1 focused on measuring Gaucher disease,, Genz-112638,, beta-glucosidase,, acid ß-glucosidase,, glucocerebrosidase,, glucosylceramide,, D-glucosyl-N-acylsphingosine glucohydrolase,, substrate reduction therapy

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

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

Sites / Locations

Tower Hematology Oncology Medical Group
UCSF MS Center
University of Colorado Health Science Center - Aurora
Yale University School of Medicine
Northwest Oncology Hematology Associates PA
Emory University Medical Genetics
Children's Memorial Hospital
University of Iowa Hospitals and Clinics
Albany Medical Center
North Shore University Medical Center
Mount Sinai School of Medicine
New York University School of Medicine
Duke University Medical Center
Cincinnati Children's Hospital Medical Center
Children's Hospital of Philadelphia
University of Pittsburgh Medical Center
O and O Alpan LLC
Hospital General de Agudos J.M Ramos Mejia
Hospital General de Ninos Dr. Ricardo Gutierrez
Royal Perth Hospital
Hospital de Clinicas da Universidade Federal do Parana
Instituto de Estadual de Hematologia Arthur de Siqueria Cavalcanti
IGEIM
Mount Sinai Hospital and the Samuel Lunenfeld Research Institute
Abou El Reesh Children's University Hospital (El Mounira), Faculty of Medicine (Kasr Al-Aini), Cairo University Hospitals, El Mounira, Cairo, Egypt
Hôpital Beaujon
Charité Universitätsmedizin Berlin
Asklepios Klinik St. Georg
Katholische Kliniken Oberhausen gem. GmbH
Azienda Ospedaliero Universitaria Careggi
Azienda Ospedialiero-Universitaria S. Maria Della Misericordia
Hematology Research Center of Ministry of Healthcare of the Russian Federation
Hospital University Miguel Servet
Cambridge University Hosptials, Addenbrookes Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Active Comparator

Arm Label

Investigational

Imiglucerase

Arm Description

Eliglustat tartrate

Outcomes

Primary Outcome Measures

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.

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.

Secondary Outcome Measures

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).

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.

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).

Absolute Change From Baseline in Total Z-Scores for Bone Mineral Density at Week 52

Hemoglobin Level

Absolute Change From Baseline in Hemoglobin Levels at Week 52

Absolute change = hemoglobin level at Week 52 minus hemoglobin level at baseline.

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.

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.

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.

Absolute Change From Baseline in Total T-Scores for Bone Mineral Density at Week 208

Absolute Change From Baseline in Total Z-Scores for Bone Mineral Density at Week 208

Absolute Change From Baseline in Hemoglobin Levels at Week 208

Absolute change = hemoglobin level at Week 208 minus hemoglobin level at baseline.

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.

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.

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.

Full Information

NCT ID

NCT00943111

First Posted

July 20, 2009

Last Updated

October 11, 2016

Sponsor

Genzyme, a Sanofi Company

1. Study Identification

Unique Protocol Identification Number

NCT00943111

Brief Title

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)

Acronym

ENCORE

Official 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)

Study Type

Interventional

2. Study Status

Record Verification Date

October 2016

Overall Recruitment Status

Completed

Study Start Date

September 2009 (undefined)

Primary Completion Date

November 2012 (Actual)

Study Completion Date

June 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Genzyme, a Sanofi Company

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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).

Detailed Description

Gaucher disease is characterized by lysosomal accumulation of glucosylceramide due to impaired glucosylceramide hydrolysis. Gaucher disease type 1, which is the most common form, accounts for greater than (>) 90% of cases and does not involve the central nervous system (CNS). Typical manifestations of Gaucher disease type 1 include splenomegaly, hepatomegaly, thrombocytopenia, anemia, bone disease, and decreased quality of life. The disease manifestations are caused by the accumulation of glucosylceramide (storage material) in macrophages (called Gaucher cells) which have infiltrated the spleen and liver as well as other tissues. Eliglustat tartrate is a small molecule drug developed as an oral therapy which acts to specifically inhibit production of this storage material in Gaucher cells. This study was designed to determine the efficacy, safety, and PK of eliglustat tartrate in adult participants with Gaucher disease type 1 who had been stabilized on ERT.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Gaucher Disease, Type 1

Keywords

Gaucher disease,, Genz-112638,, beta-glucosidase,, acid ß-glucosidase,, glucocerebrosidase,, glucosylceramide,, D-glucosyl-N-acylsphingosine glucohydrolase,, substrate reduction therapy

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Randomized

Enrollment

160 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Investigational

Arm Type

Experimental

Arm Description

Eliglustat tartrate

Arm Title

Imiglucerase

Arm Type

Active Comparator

Intervention Type

Drug

Intervention Name(s)

Eliglustat tartrate

Other Intervention Name(s)

Genz-112638

Intervention Description

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.

Intervention Type

Drug

Intervention Name(s)

Imiglucerase

Other Intervention Name(s)

Cerezyme®

Intervention Description

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.

Primary Outcome Measure Information:

Title

Percentage of Participants Who Remained Stable for 52 Weeks During the Primary Analysis Period

Description

Time Frame

Baseline up to Week 52

Title

Percentage of Participants Who Remained Stable Annually for 4 Years During the LTTP

Description

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.

Time Frame

Week 52 up to week 208

Secondary Outcome Measure Information:

Title

Total T-Scores for Bone Mineral Density

Description

Time Frame

Baseline

Title

Absolute Change From Baseline in Total T-Scores for Bone Mineral Density at Week 52

Description

Time Frame

Baseline, Week 52

Title

Total Z-Scores for Bone Mineral Density

Description

Time Frame

Baseline

Title

Absolute Change From Baseline in Total Z-Scores for Bone Mineral Density at Week 52

Description

Time Frame

Baseline, Week 52

Title

Hemoglobin Level

Time Frame

Baseline

Title

Absolute Change From Baseline in Hemoglobin Levels at Week 52

Description

Absolute change = hemoglobin level at Week 52 minus hemoglobin level at baseline.

Time Frame

Baseline, Week 52

Title

Percent Change From Baseline in Platelet Counts at Week 52

Description

Percent change in platelet counts = ([platelet count at Week 52 minus platelet count at baseline] divided by [platelet count at baseline]) multiplied by 100.

Time Frame

Baseline, Week 52

Title

Percent Change From Baseline in Spleen Volume (MN) at Week 52

Description

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.

Time Frame

Baseline, Week 52

Title

Percent Change From Baseline in Liver Volume (in MN) at Week 52

Description

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.

Time Frame

Baseline, Week 52

Title

Absolute Change From Baseline in Total T-Scores for Bone Mineral Density at Week 208

Description

Time Frame

Baseline, Week 208

Title

Absolute Change From Baseline in Total Z-Scores for Bone Mineral Density at Week 208

Description

Time Frame

Baseline, Week 208

Title

Absolute Change From Baseline in Hemoglobin Levels at Week 208

Description

Absolute change = hemoglobin level at Week 208 minus hemoglobin level at baseline.

Time Frame

Baseline, Week 208

Title

Percent Change From Baseline in Platelet Counts at Week 208

Description

Percent change in platelet counts = ([platelet count at Week 208 minus platelet count at baseline] divided by [platelet count at baseline]) multiplied by 100.

Time Frame

Baseline, Week 208

Title

Percent Change From Baseline in Spleen Volume (in MN) at Week 208

Description

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.

Time Frame

Baseline, Week 208

Title

Percent Change From Baseline in Liver Volume (in MN) at Week 208

Description

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.

Time Frame

Baseline, Week 208

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

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 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Medical Monitor

Organizational Affiliation

Genzyme, a Sanofi Company

Official's Role

Study Director

Facility Information:

Facility Name

Tower Hematology Oncology Medical Group

City

Beverly Hills

State/Province

California

Country

United States

Facility Name

UCSF MS Center

City

San Francisco

State/Province

California

Country

United States

Facility Name

University of Colorado Health Science Center - Aurora

City

Aurora

State/Province

Colorado

Country

United States

Facility Name

Yale University School of Medicine

City

New Haven

State/Province

Connecticut

Country

United States

Facility Name

Northwest Oncology Hematology Associates PA

City

Coral Springs

State/Province

Florida

Country

United States

Facility Name

Emory University Medical Genetics

City

Decatur

State/Province

Georgia

Country

United States

Facility Name

Children's Memorial Hospital

City

Chicago

State/Province

Illinois

Country

United States

Facility Name

University of Iowa Hospitals and Clinics

City

Iowa City

State/Province

Iowa

Country

United States

Facility Name

Albany Medical Center

City

Albany

State/Province

New York

Country

United States

Facility Name

North Shore University Medical Center

City

Manhasset

State/Province

New York

ZIP/Postal Code

11030

Country

United States

Facility Name

Mount Sinai School of Medicine

City

New York

State/Province

New York

Country

United States

Facility Name

New York University School of Medicine

City

New York

State/Province

New York

Country

United States

Facility Name

Duke University Medical Center

City

Durham

State/Province

North Carolina

Country

United States

Facility Name

Cincinnati Children's Hospital Medical Center

City

Cincinnati

State/Province

Ohio

Country

United States

Facility Name

Children's Hospital of Philadelphia

City

Philadelphia

State/Province

Pennsylvania

Country

United States

Facility Name

University of Pittsburgh Medical Center

City

Pittsburgh

State/Province

Pennsylvania

Country

United States

Facility Name

O and O Alpan LLC

City

Springfield

State/Province

Virginia

Country

United States

Facility Name

Hospital General de Agudos J.M Ramos Mejia

City

Buenos Aires

Country

Argentina

Facility Name

Hospital General de Ninos Dr. Ricardo Gutierrez

City

Buenos Aires

Country

Argentina

Facility Name

Royal Perth Hospital

City

Perth, WA

Country

Australia

Facility Name

Hospital de Clinicas da Universidade Federal do Parana

City

Curitiba

Country

Brazil

Facility Name

Instituto de Estadual de Hematologia Arthur de Siqueria Cavalcanti

City

Rio de Janeiro

Country

Brazil

Facility Name

IGEIM

City

São Paulo

Country

Brazil

Facility Name

Mount Sinai Hospital and the Samuel Lunenfeld Research Institute

City

Toronto Ontario

Country

Canada

Facility Name

Abou El Reesh Children's University Hospital (El Mounira), Faculty of Medicine (Kasr Al-Aini), Cairo University Hospitals, El Mounira, Cairo, Egypt

City

Cairo

Country

Egypt

Facility Name

Hôpital Beaujon

City

Clichy

Country

France

Facility Name

Charité Universitätsmedizin Berlin

City

Berlin

Country

Germany

Facility Name

Asklepios Klinik St. Georg

City

Hamburg

Country

Germany

Facility Name

Katholische Kliniken Oberhausen gem. GmbH

City

Oberhausen

Country

Germany

Facility Name

Azienda Ospedaliero Universitaria Careggi

City

Firenze

Country

Italy

Facility Name

Azienda Ospedialiero-Universitaria S. Maria Della Misericordia

City

Udine

Country

Italy

Facility Name

Hematology Research Center of Ministry of Healthcare of the Russian Federation

City

Moscow

Country

Russian Federation

Facility Name

Hospital University Miguel Servet

City

Zaragoza

Country

Spain

Facility Name

Cambridge University Hosptials, Addenbrookes Hospital

City

Cambridge

Country

United Kingdom

12. IPD Sharing Statement

Citations:

PubMed Identifier

20439622

Citation

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.

Results Reference

background

PubMed Identifier

20713962

Citation

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.

Results Reference

background

PubMed Identifier

17509920

Citation

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.

Results Reference

background

PubMed Identifier

24816856

Citation

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.

Results Reference

background

PubMed Identifier

24835462

Citation

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.

Results Reference

background

PubMed Identifier

28167660

Citation

Cox TM, Drelichman G, Cravo R, Balwani M, Burrow TA, Martins AM, Lukina E, Rosenbloom B, Goker-Alpan O, Watman N, El-Beshlawy A, Kishnani PS, Pedroso ML, Gaemers SJM, Tayag R, Peterschmitt MJ. Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy. Blood. 2017 Apr 27;129(17):2375-2383. doi: 10.1182/blood-2016-12-758409. Epub 2017 Feb 6.

Results Reference

derived

PubMed Identifier

25819691

Citation

Cox TM, Drelichman G, Cravo R, Balwani M, Burrow TA, Martins AM, Lukina E, Rosenbloom B, Ross L, Angell J, Puga AC. Eliglustat compared with imiglucerase in patients with Gaucher's disease type 1 stabilised on enzyme replacement therapy: a phase 3, randomised, open-label, non-inferiority trial. Lancet. 2015 Jun 13;385(9985):2355-62. doi: 10.1016/S0140-6736(14)61841-9. Epub 2015 Mar 26. Erratum In: Lancet. 2015 Jun 13;385(9985):2354.

Results Reference

derived

Learn more about this trial

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)

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