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NCT00943111 · ClinicalTrials.gov registry record · Phase 3
A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)
A Phase 3 study of Gaucher Disease, Type 1, sponsored by Genzyme, a Sanofi Company.
- Completed
- Registry status
- Phase 3
- Development phase
- 160
- Enrollment target
- 20
- Study locations
NCT00943111: Completed Phase 3 study of Gaucher Disease, Type 1, sponsored by Genzyme, a Sanofi Company.
NCT00943111 is a Phase 3 study of Gaucher Disease, Type 1 that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 160 participants, above the 39-participant average among 8 other Gaucher Disease, Type 1 trials with a reported enrollment target (310% higher). The trial reports 20 study locations across 13 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00943111, a Phase 3 study of Gaucher Disease, Type 1, has completed, sponsored by Genzyme, a Sanofi Company.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 160 participants
- Enrollment target
- 20
- Study locations
Study 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).
Primary Outcome
For a participant to be classified as stable, the participant must have remained stable in hematological parameters (hemoglobin levels and platelet counts) and organ volumes (spleen, when applicable, and liver volumes in multiples of normal \[MN\]). Stable hematological parameters were defined as hemoglobin level did not decrease more than (\>) 1.5 gram per deciliter (g/dL) from baseline and platelet count did not decrease \>25% from baseline. Stable organ volumes were defined as spleen volume (
Conditions Studied
Interventions
- DRUG Eliglustat tartrate
- DRUG Imiglucerase
Study Locations (20)
New York
- Albany Medical Center - Albany
- North Shore University Medical Center - Manhasset
- Mount Sinai School of Medicine - New York
- New York University School of Medicine - New York
Other
- Hospital General de Agudos J.M Ramos Mejia - Buenos Aires
- Hospital General de Ninos Dr. Ricardo Gutierrez - Buenos Aires
- Royal Perth Hospital - Perth, WA
California
- Tower Hematology Oncology Medical Group - Beverly Hills
- UCSF MS Center - San Francisco
Pennsylvania
- Children's Hospital of Philadelphia - Philadelphia
- University of Pittsburgh Medical Center - Pittsburgh
Colorado
- University of Colorado Health Science Center - Aurora - Aurora
Connecticut
- Yale University School of Medicine - New Haven
Florida
- Northwest Oncology Hematology Associates PA - Coral Springs
Georgia
- Emory University Medical Genetics - Decatur
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 160 participants |
| Start Date | 2009-09 |
| Est. Completion | 2015-06 |
| Phase | Phase 3 |
What the finished NCT00943111 record still lists
NCT00943111 is an interventional study that assigns participants to a tested intervention. The registered 160 participants enrollment target is mid-sized for trials with a published cap, above the 39-participant average among 8 other Gaucher Disease, Type 1 trials with a reported enrollment target (310% higher).
The record links to 1 condition, with Gaucher Disease, Type 1 appearing as the primary indexed condition, and to 2 interventions - of which Eliglustat tartrate is the first listed.
NCT00943111 names 20 study sites across 13 states, led by New York, Other, California.
Frequently Asked Questions
What is clinical trial NCT00943111 about?
NCT00943111 is a clinical study titled "A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)". 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).
What is the current status of trial NCT00943111?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 160 participants. The study started on 2009-09. Estimated completion is 2015-06.
What conditions does trial NCT00943111 study?
This clinical trial studies the following conditions: Gaucher Disease, Type 1.
What interventions are being tested in trial NCT00943111?
The interventions under investigation include: Eliglustat tartrate (DRUG), Imiglucerase (DRUG).
Who is sponsoring clinical trial NCT00943111?
This trial is sponsored by Genzyme, a Sanofi Company, which has 196 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT00943111 being conducted?
This trial has 20 study locations across California, Colorado, Connecticut, Florida, Georgia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Gaucher Disease, Type 1
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT00943111's enrollment target sits among peer trials
160 1st of 8 higher than 8 of 8 other Gaucher Disease, Type 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Gaucher Disease, Type 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
-
A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)
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-
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A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)
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Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2)
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A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients
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