World Health Organization site
Skip Navigation Links

Please fill this short user satisfaction survey


Main
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
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
Study design:   
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
Address: 
Telephone:
Email:
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 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]
Total T-Scores for Bone Mineral Density [Time Frame: Baseline]
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]
Percent Change From Baseline in Liver Volume (in MN) at Week 208 [Time Frame: Baseline, Week 208]
Hemoglobin Level [Time Frame: Baseline]
Percent Change From Baseline in Platelet Counts at Week 208 [Time Frame: Baseline, Week 208]
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]
Percent Change From Baseline in Spleen Volume (in MN) at Week 208 [Time Frame: Baseline, Week 208]
Secondary ID(s)
GZGD02607
EFC12812
2008-005223-28
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results
Results available: Yes
Date Posted: 04/09/2014
Date Completed:
URL: https://clinicaltrials.gov/ct2/show/results/NCT00943111
Disclaimer: Trials posted on this search portal are not endorsed by WHO, but are provided as a service to our users. In no event shall the World Health Organization be liable for any damages arising from the use of the information linked to in this section. None of the information obtained through use of the search portal should in any way be used in clinical care without consulting a physician or licensed health professional. WHO is not responsible for the accuracy, completeness and/or use made of the content displayed for any trial record.
Copyright - World Health Organization - Version 3.6 - Version history Please fill this short user satisfaction survey