Phase 3 N=160 Randomized Treatment

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

Gaucher Disease, Type 1

Bottom Line

View on ClinicalTrials.gov: NCT00943111 ↗

Enrolled (actual)

160

Serious AEs

15.7%

Results posted

Sep 2014

Primary outcome: Primary: Percentage of Participants Who Remained Stable for 52 Weeks During the Primary Analysis Period — 84.8; 93.6 percentage of participants

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Eliglustat tartrate (Drug); Imiglucerase (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Genzyme, a Sanofi Company
Primary completion: Nov 2012

Outcome Measures

Outcome	Result	p-value
PRIMARY Percentage of Participants Who Remained Stable for 52 Weeks During the Primary Analysis Period	84.8; 93.6	—
PRIMARY Percentage of Participants Who Remained Stable Annually for 4 Years During the LTTP	83.6; 75.65; 60.53; 26.97	—
SECONDARY Total T-Scores for Bone Mineral Density	-0.56; -0.33; -0.11; -0.47	—
SECONDARY Absolute Change From Baseline in Total T-Scores for Bone Mineral Density at Week 52	0.04; 0.03; 0.00; -0.03	—
SECONDARY Total Z-Scores for Bone Mineral Density	-0.35; -0.14; 0.09; -0.18	—
SECONDARY Absolute Change From Baseline in Total Z-Scores for Bone Mineral Density at Week 52	0.06; 0.06; 0.03; 0.02	—
SECONDARY Hemoglobin Level	13.592; 13.797	—
SECONDARY Absolute Change From Baseline in Hemoglobin Levels at Week 52	-0.22; 0.05	—
SECONDARY Percent Change From Baseline in Platelet Counts at Week 52	3.93; 2.63	—
SECONDARY Percent Change From Baseline in Spleen Volume (MN) at Week 52	-6.05; -3.22	—
SECONDARY Percent Change From Baseline in Liver Volume (in MN) at Week 52	1.99; 3.13	—
SECONDARY Absolute Change From Baseline in Total T-Scores for Bone Mineral Density at Week 208	0.22; -0.03	—
SECONDARY Absolute Change From Baseline in Total Z-Scores for Bone Mineral Density at Week 208	0.29; 0.03	—
SECONDARY Absolute Change From Baseline in Hemoglobin Levels at Week 208	0.297	—
SECONDARY Percent Change From Baseline in Platelet Counts at Week 208	6.990	—
SECONDARY Percent Change From Baseline in Spleen Volume (in MN) at Week 208	-14.768	—
SECONDARY Percent Change From Baseline in Liver Volume (in MN) at Week 208	-2.345	—

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).

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

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Data sourced from ClinicalTrials.gov (NCT00943111). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.