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NCT00478647 · ClinicalTrials.gov registry record · Phase 2
Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase
A Phase 2 study of Gaucher Disease, sponsored by Shire.
- Completed
- Registry status
- Phase 2
- Development phase
- 40
- Enrollment target
- 15
- Study locations
NCT00478647: Completed Phase 2 study of Gaucher Disease, sponsored by Shire.
NCT00478647 is a Phase 2 study of Gaucher Disease that has completed, run by Shire. The registered enrollment target is 40 participants, below the 1,056-participant average among 13 other Gaucher Disease trials with a reported enrollment target (96% lower). The trial reports 15 study locations across 11 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00478647, a Phase 2 study of Gaucher Disease, has completed, sponsored by Shire.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 40 participants
- Enrollment target
- 15
- Study locations
Study Summary
Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular engorgement, organomegaly, and organ system dysfunction. The purpose of this study is to evaluate the safety and efficacy of every other week dosing of GA-GCB (velaglucerase alfa) in participants with type 1 Gaucher disease who were previously treated with imiglucerase.
Primary Outcome
Safety was assessed throughout the study by assessments including adverse events, concomitant medication use, and vital signs. Additional safety assessments, including 12-lead ECGs, physical examinations, clinical laboratory tests and determination of the presence of anti-velaglucerase alfa antibodies. Refer to Adverse event section for further details.
Conditions Studied
Interventions
- BIOLOGICAL GA-GCB (velaglucerase alfa)
Study Locations (15)
Other
- Shaare Zedek Medical Center - Jerusalem
- Children's Memorial Health Institute - Warsaw
- Hospital Universitario Miguel Servet - Zaragoza
- The Royal Free Hospital - London
California
- Regional Metabolic Center - Los Angeles
- Children's Hospital Oakland - Oakland
Georgia
- Emory University - Decatur
Illinois
- Feinberg School of Medicine - Chicago
Minnesota
- Children's of Minnesota - Minneapolis
Missouri
- Children's Mercy Hospital and Clinic - Kansas City
New York
- NYU School of Medicine - New York
Ohio
- Cincinatti Children's Hospital - Cincinnati
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 40 participants |
| Start Date | 2007-07-25 |
| Est. Completion | 2009-06-26 |
| Phase | Phase 2 |
What the finished NCT00478647 record still lists
NCT00478647 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 40 participants, a relatively small participant target, below the 1,056-participant average among 13 other Gaucher Disease trials with a reported enrollment target (96% lower).
The record links to 1 condition, with Gaucher Disease appearing as the primary indexed condition, and to 1 intervention - of which GA-GCB (velaglucerase alfa) is the first listed.
NCT00478647 lists 15 locations in 11 states (Other, California, Georgia).
Frequently Asked Questions
What is clinical trial NCT00478647 about?
NCT00478647 is a clinical study titled "Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase". Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular engorgement, organomegaly, and organ system dysfunction. The purpose of t...
What is the current status of trial NCT00478647?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 40 participants. The study started on 2007-07-25. Estimated completion is 2009-06-26.
What conditions does trial NCT00478647 study?
This clinical trial studies the following conditions: Gaucher Disease.
What interventions are being tested in trial NCT00478647?
The interventions under investigation include: GA-GCB (velaglucerase alfa) (BIOLOGICAL).
Who is sponsoring clinical trial NCT00478647?
This trial is sponsored by Shire, which has 245 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT00478647 being conducted?
This trial has 15 study locations across California, Georgia, Illinois, Minnesota, Missouri. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Gaucher Disease
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 NCT00478647's enrollment target sits among peer trials
40 5th of 13 higher than 9 of 13 other Gaucher Disease trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Gaucher Disease 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
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