Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease
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
1 other identifier
expanded_access
N/A
1 country
32
Brief 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 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.
Trial Health
Trial Health Score
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32 active sites
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Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
August 5, 2009
CompletedFirst Posted
Study publicly available on registry
August 7, 2009
CompletedMay 21, 2021
May 1, 2021
August 5, 2009
May 18, 2021
Conditions
Keywords
Interventions
up to 60 U/kg, every other week via intravenous infusion
Eligibility Criteria
You may qualify if:
- The patient has a documented diagnosis of type 1 Gaucher disease
- The patient is \> 2 years of age
- The patient has NOT previously experienced an anaphylactic or anaphylactoid reaction to another ERT including imiglucerase
- Women of child-bearing potential must agree to use a medically acceptable method of contraception at all times during the study; and must have a negative result to a pregnancy test as required throughout their participation in the study. Male patients must use a medically acceptable method of birth control throughout their participation in the study and must report their partner's pregnancy.
- The patient is sufficiently cooperative to participate in this treatment plan as judged by the Investigator
- If the patient is naïve or new to treatment, the patient has one or more of the following (in absence of the following criteria, please call the sponsor for treatment justification):
- Gaucher disease-related anemia
- Moderate splenomegaly (2 to 3 cm below the left costal margin), by palpation
- Gaucher disease-related thrombocytopenia
- Gaucher disease-related palpable enlarged liver
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Shirelead
Study Sites (32)
St Joseph's Hospital & Medical Center
Phoenix, Arizona, 85013, United States
Tower Hematology Oncology
Beverly Hills, California, 90211-1850, United States
Rady's Children's Hospital of San Diego
La Jolla, California, 92093, United States
Southern California Permanente Medical Group
Los Angeles, California, 90027, United States
The Permanente Medical Group
Sacramento, California, 95815, United States
Stanford University Medical Genetics
Stanford, California, 94305-5208, United States
Rocky Mountain Cancer Centers
Denver, Colorado, 80218, United States
Yale University
New Haven, Connecticut, 06510, United States
University Research Foundation for Lysosomal Storage Diseases
Coral Springs, Florida, 33065, United States
Gainesville Hematology Oncology Associates
Gainesville, Florida, 32605-4218, United States
Adventis Healthcare System dba Florida Hospital
Orlando, Florida, 32804-4603, United States
East Lake Oncology
Palm Harbor, Florida, 34685, United States
Emory Genetics
Decatur, Georgia, 30033, United States
Children's Memorial Hospital
Chicago, Illinois, 60614, United States
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242, United States
Annapolis Oncology Center
Annapolis, Maryland, 21401, United States
Sinai Hospital of Baltimore
Baltimore, Maryland, 21215, United States
University of Massachusetts
Shrewsbury, Massachusetts, 01545, United States
Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota, 55404, United States
The University Research Foundation for Lysosomal Storage Diseases
Kansas City, Missouri, 64108-4619, United States
St. Joseph's
Paterson, New Jersey, 07503, United States
Hemophilia Center of Western New York Incorporated
Buffalo, New York, 14215, United States
North Shore Hematology/Oncology - Manhasset
Manhasset, New York, 11030, United States
New York University School of Medicine
New York, New York, 10016, United States
Mount Sinai School of Medicine
New York, New York, 10029-6500, United States
Fullerton Genetic
Asheville, North Carolina, 28801-4420, United States
Duke Medical Center
Durham, North Carolina, 27710, United States
Akron Children's Hospital
Akron, Ohio, 44308, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
University of Virginia Health Systems
Charlottesville, Virginia, 22908-0386, United States
O & O Alpan, LLC
Springfield, Virginia, 22152, United States
Related Publications (1)
Pastores GM, Rosenbloom B, Weinreb N, Goker-Alpan O, Grabowski G, Cohn GM, Zahrieh D. A multicenter open-label treatment protocol (HGT-GCB-058) of velaglucerase alfa enzyme replacement therapy in patients with Gaucher disease type 1: safety and tolerability. Genet Med. 2014 May;16(5):359-66. doi: 10.1038/gim.2013.154. Epub 2013 Nov 21.
PMID: 24263462DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Study Director
Takeda
Study Design
- Study Type
- expanded access
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 5, 2009
First Posted
August 7, 2009
Last Updated
May 21, 2021
Record last verified: 2021-05