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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00943111
• Date of registration: 20/07/2009
• Prospective Registration: Yes
• Primary sponsor: Genzyme, a Sanofi Company
• Public title: A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE) 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)
• Date of first enrolment: September 2009
• Target sample size: 160
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00943111
• Study type: Interventional
• Phase: Phase 3

Countries of recruitment

Argentina, Australia, Brazil, Canada, Czech Republic, Egypt, France, Germany, Italy, Netherlands, Russian Federation, Spain, Turkey, United Kingdom, United States

Contacts

• Name: Medical Monitor
• Affiliation: Genzyme, a Sanofi Company

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Gaucher Disease, Type 1

Intervention(s)

Drug: Imiglucerase

Drug: Eliglustat tartrate

Primary Outcome(s)

Percentage of Participants Who Remained Stable Annually for 4 Years During the LTTP [Time Frame: Week 52 up to week 208]

Percentage of Participants Who Remained Stable for 52 Weeks During the Primary Analysis Period [Time Frame: Baseline up to Week 52]

Secondary Outcome(s)

Absolute Change From Baseline in Hemoglobin Levels at Week 208 [Time Frame: Baseline, Week 208]

Absolute Change From Baseline in Total T-Scores for Bone Mineral Density at Week 208 [Time Frame: Baseline, Week 208]

Percent Change From Baseline in Platelet Counts at Week 52 [Time Frame: Baseline, Week 52]

Hemoglobin Level [Time Frame: Baseline]

Percent Change From Baseline in Platelet Counts at Week 208 [Time Frame: Baseline, Week 208]

Percent Change From Baseline in Liver Volume (in MN) at Week 208 [Time Frame: Baseline, Week 208]

Absolute Change From Baseline in Total Z-Scores for Bone Mineral Density at Week 52 [Time Frame: Baseline, Week 52]

Percent Change From Baseline in Liver Volume (in MN) at Week 52 [Time Frame: Baseline, Week 52]

Total Z-Scores for Bone Mineral Density [Time Frame: Baseline]

Absolute Change From Baseline in Hemoglobin Levels at Week 52 [Time Frame: Baseline, Week 52]

Percent Change From Baseline in Spleen Volume (MN) at Week 52 [Time Frame: Baseline, Week 52]

Absolute Change From Baseline in Total T-Scores for Bone Mineral Density at Week 52 [Time Frame: Baseline, Week 52]

Absolute Change From Baseline in Total Z-Scores for Bone Mineral Density at Week 208 [Time Frame: Baseline, Week 208]

Total T-Scores for Bone Mineral Density [Time Frame: Baseline]

Percent Change From Baseline in Spleen Volume (in MN) at Week 208 [Time Frame: Baseline, Week 208]

Secondary ID(s)

EFC12812

2008-005223-28

GZGD02607

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 04/09/2014
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00943111