Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase
Public ClinicalTrials.gov record NCT00478647. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Multicenter Open-Label Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients With Type 1 Gaucher Disease Previously Treated With Imiglucerase
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT00478647
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 2, Phase 3
- Enrollment
- 40 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 2 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jul 24, 2007
- Primary completion
- Jun 25, 2009
- Completion
- Jun 25, 2009
- Last update posted
- Jun 9, 2021
2007 – 2009
United States locations
- U.S. sites
- 11
- U.S. states
- 10
- U.S. cities
- 11
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Regional Metabolic Center | Los Angeles | California | 90027 | — |
| Children's Hospital Oakland | Oakland | California | 94609 | — |
| Emory University | Decatur | Georgia | 30033 | — |
| Feinberg School of Medicine | Chicago | Illinois | 60614 | — |
| Children's of Minnesota | Minneapolis | Minnesota | 55404 | — |
| Children's Mercy Hospital and Clinic | Kansas City | Missouri | 64108 | — |
| NYU School of Medicine | New York | New York | 10016 | — |
| Cincinatti Children's Hospital | Cincinnati | Ohio | 45229 | — |
| Texas Children's Hospital | Houston | Texas | 77030 | — |
| Medical Genetics/Pediatrics | Salt Lake City | Utah | 84132 | — |
| Children's Hospital of Wisconsin | Milwaukee | Wisconsin | 53226 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 4 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT00478647, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jun 9, 2021 · Synced Sep 6, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT00478647 live on ClinicalTrials.gov.