Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease
Public ClinicalTrials.gov record NCT00954460. 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
Multicenter Open-Label Treatment Protocol to Observe the Safety of Gene-Activated™ Human Glucocerebrosidase (GA-GCB, Velaglucerase Alfa) ERT in Newly Diagnosed or Previously Treated (With Imiglucerase) Patients With Type 1 Gaucher Disease
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 treatment protocol is to observe the safety of velaglucerase alfa in patients with type 1 Gaucher disease who are either treatment naive (newly diagnosed) or who are currently being treated with the Enzyme Replacement Therapy (ERT) imiglucerase.
Study identification
- NCT ID
- NCT00954460
- Recruitment status
- Approved For Marketing
- Study type
- Expanded access
- Phase
- Not listed
- Enrollment
- Not listed
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 3 Years and older
- Sex
- All
- Healthy volunteers
- Not listed
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Not listed
- Primary completion
- Not listed
- Completion
- Not listed
- Last update posted
- May 21, 2021
United States locations
- U.S. sites
- 32
- U.S. states
- 18
- U.S. cities
- 31
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| St Joseph's Hospital & Medical Center | Phoenix | Arizona | 85013 | — |
| Tower Hematology Oncology | Beverly Hills | California | 90211-1850 | — |
| Rady's Children's Hospital of San Diego | La Jolla | California | 92093 | — |
| Southern California Permanente Medical Group | Los Angeles | California | 90027 | — |
| The Permanente Medical Group | Sacramento | California | 95815 | — |
| Stanford University Medical Genetics | Stanford | California | 94305-5208 | — |
| Rocky Mountain Cancer Centers | Denver | Colorado | 80218 | — |
| Yale University | New Haven | Connecticut | 06510 | — |
| University Research Foundation for Lysosomal Storage Diseases | Coral Springs | Florida | 33065 | — |
| Gainesville Hematology Oncology Associates | Gainesville | Florida | 32605-4218 | — |
| Adventis Healthcare System dba Florida Hospital | Orlando | Florida | 32804-4603 | — |
| East Lake Oncology | Palm Harbor | Florida | 34685 | — |
| Emory Genetics | Decatur | Georgia | 30033 | — |
| Children's Memorial Hospital | Chicago | Illinois | 60614 | — |
| University of Iowa Hospitals and Clinics | Iowa City | Iowa | 52242 | — |
| Annapolis Oncology Center | Annapolis | Maryland | 21401 | — |
| Sinai Hospital of Baltimore | Baltimore | Maryland | 21215 | — |
| University of Massachusetts | Shrewsbury | Massachusetts | 01545 | — |
| Children's Hospitals and Clinics of Minnesota | Minneapolis | Minnesota | 55404 | — |
| The University Research Foundation for Lysosomal Storage Diseases | Kansas City | Missouri | 64108-4619 | — |
| St. Joseph's | Paterson | New Jersey | 07503 | — |
| Hemophilia Center of Western New York Incorporated | Buffalo | New York | 14215 | — |
| North Shore Hematology/Oncology - Manhasset | Manhasset | New York | 11030 | — |
| New York University School of Medicine | New York | New York | 10016 | — |
| Mount Sinai School of Medicine | New York | New York | 10029-6500 | — |
| Fullerton Genetic | Asheville | North Carolina | 28801-4420 | — |
| Duke Medical Center | Durham | North Carolina | 27710 | — |
| Akron Children's Hospital | Akron | Ohio | 44308 | — |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| University of Virginia Health Systems | Charlottesville | Virginia | 22908-0386 | — |
| O & O Alpan, LLC | Springfield | Virginia | 22152 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT00954460, 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 May 21, 2021 · Synced Oct 26, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT00954460 live on ClinicalTrials.gov.