Phase 3 N=40 Randomized Quadruple-blind Treatment

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)

Gaucher Disease, Type 1

Bottom Line

View on ClinicalTrials.gov: NCT00891202 ↗

Enrolled (actual)

Serious AEs

12.5%

Results posted

Sep 2014

Primary outcome: Primary: PAP: Percent Change From Baseline in Spleen Volume (in Multiples of Normal [MN]) at Week 39 of the Primary Analysis Period With Eliglustat Tartrate Treatment as Compared to Placebo — -27.77; 2.26 percent change — p=<0.0001

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Eliglustat tartrate (Drug); Placebo (Drug)
Age: Pediatric, Adult, Older Adult · 16+ yrs
Sex: All
Sponsor: Genzyme, a Sanofi Company
Primary completion: Jul 2012

Outcome Measures

Outcome	Result	p-value
PRIMARY PAP: Percent Change From Baseline in Spleen Volume (in Multiples of Normal [MN]) at Week 39 of the Primary Analysis Period With Eliglustat Tartrate Treatment as Compared to Placebo	-27.77; 2.26	<0.0001 sig
SECONDARY PAP: Hemoglobin Level	12.05; 12.75	—
SECONDARY PAP: Absolute Change From Baseline in Hemoglobin Level at Week 39	0.69; -0.54	—
SECONDARY PAP: Percent Change From Baseline in Liver Volume (in MN) at Week 39	-5.20; 1.44	—
SECONDARY PAP: Percent Change From Baseline in Platelet Counts at Week 39	32.00; -9.06	—
SECONDARY LTTP: Percent Change From Baseline in Spleen Volume (in MN) at Week 234	-66.9; -64.0	—
SECONDARY LTTP: Absolute Change From Baseline in Hemoglobin Level at Week 234	1.1; 1.9	—
SECONDARY LTTP: Percent Change From Baseline in Liver Volume (in MN) at Week 234	-24.3; -22.4	—
SECONDARY LTTP: Percent Change From Baseline in Platelet Counts at Week 234	77.3; 100.1	—

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.

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 16 years old at the time of randomization;
The participant had a confirmed diagnosis of Gaucher disease Type 1;
Female participants of childbearing potential must 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 has had a partial or total splenectomy;
The participant had received pharmacological chaperones or miglustat within 6 months prior to randomization;
The participant had received enzyme replacement therapy within 9 months prior to randomization;
The participant had Type 2 or 3 Gaucher disease or was suspected of having Type 3 Gaucher disease;
The participant had any clinically significant disease, other than Gaucher disease, including cardiovascular, renal, hepatic, gastrointestinal (GI), pulmonary, neurologic, endocrine, metabolic, (for example, hypokalemia, hypomagnesemia), or psychiatric disease, other medical conditions, or serious intercurrent illness that might confound the study results, or, on the opinion of the investigator, might 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 (NCT00891202). 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.