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

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT00891202
• Date of registration: 30/04/2009
• Prospective Registration: Yes
• Primary sponsor: Genzyme, a Sanofi Company
• Public title: A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)
• Scientific title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study Confirming the Efficacy and Safety of Genz-112638 in Patients With Gaucher Disease Type 1 (ENGAGE)
• Date of first enrolment: November 2009
• Target sample size: 40
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00891202
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

Bulgaria, Canada, Chile, Colombia, Georgia, India, Israel, Jordan, Lebanon, Mexico, Netherlands, Russian Federation, Saudi Arabia, Serbia, Tunisia, 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 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.



Age minimum: 16 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Gaucher Disease, Type 1

Intervention(s)

Drug: Placebo

Drug: Eliglustat tartrate

Primary Outcome(s)

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 [Time Frame: PAP Baseline (Day 1), Week 39]

Secondary Outcome(s)

LTTP: Absolute Change From Baseline in Hemoglobin Level at Week 234 [Time Frame: PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234]

LTTP: Percent Change From Baseline in Platelet Counts at Week 234 [Time Frame: PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234]

LTTP: Percent Change From Baseline in Spleen Volume (in MN) at Week 234 [Time Frame: PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234]

LTTP: Percent Change From Baseline in Liver Volume (in MN) at Week 234 [Time Frame: PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234]

PAP: Percent Change From Baseline in Liver Volume (in MN) at Week 39 [Time Frame: PAP Baseline (Day 1), Week 39]

PAP: Absolute Change From Baseline in Hemoglobin Level at Week 39 [Time Frame: PAP Baseline (Day 1), Week 39]

PAP: Hemoglobin Level [Time Frame: PAP Baseline (Day 1)]

PAP: Percent Change From Baseline in Platelet Counts at Week 39 [Time Frame: PAP Baseline (Day 1), Week 39]

Secondary ID(s)

2008-005222-37

EFC12813

GZGD02507

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 03/09/2014
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00891202