Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease
- Conditions
- Gaucher Disease, Type 1
- Registration Number
- NCT00954460
- Lead Sponsor
- Shire
- 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.
- Detailed Description
Type 1 Gaucher disease, the most common form, accounts for more than 90% of all cases of Gaucher disease and does not involve the CNS. Typical manifestations of type 1 Gaucher disease include hepatomegaly, splenomegaly, thrombocytopenia, bleeding tendencies, anemia, hypermetabolism, skeletal pathology, growth retardation, pulmonary disease, and decreased quality of life. Velaglucerase alfa (Gene-Activatedâ„¢ human glucocerebrosidase;GA-GCB) is produced in a continuous human cell line using proprietary gene-activation technology and has an identical amino acid sequence to the naturally occurring human enzyme. Velaglucerase alfa contains terminal mannose residues that target the enzyme to the macrophages-the primary target cells in Gaucher disease. This treatment protocol will 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. Patients currently being treated with ERT for their Gaucher disease will receive the same number of units of velaglucerase alfa per month as their imiglucerase dose for doses between 30-120 U/kg/month. For patients who experienced dose reductions in their imiglucerase treatment due to supply constraints the pre-reduction monthly dose may be used to determine the monthly dose of velaglucerase alfa.
Recruitment & Eligibility
- Status
- APPROVED_FOR_MARKETING
- Sex
- All
- Target Recruitment
- Not specified
-
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
None
Study & Design
- Study Type
- EXPANDED_ACCESS
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (32)
Children's Hospitals and Clinics of Minnesota
🇺🇸Minneapolis, Minnesota, United States
Sinai Hospital of Baltimore
🇺🇸Baltimore, Maryland, United States
North Shore Hematology/Oncology - Manhasset
🇺🇸Manhasset, New York, United States
Cincinnati Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States
Southern California Permanente Medical Group
🇺🇸Los Angeles, California, United States
Emory Genetics
🇺🇸Decatur, Georgia, United States
Fullerton Genetic
🇺🇸Asheville, North Carolina, United States
Children's Memorial Hospital
🇺🇸Chicago, Illinois, United States
Duke Medical Center
🇺🇸Durham, North Carolina, United States
Akron Children's Hospital
🇺🇸Akron, Ohio, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
St Joseph's Hospital & Medical Center
🇺🇸Phoenix, Arizona, United States
Rocky Mountain Cancer Centers
🇺🇸Denver, Colorado, United States
Rady's Children's Hospital of San Diego
🇺🇸La Jolla, California, United States
Stanford University Medical Genetics
🇺🇸Stanford, California, United States
Tower Hematology Oncology
🇺🇸Beverly Hills, California, United States
University Research Foundation for Lysosomal Storage Diseases
🇺🇸Coral Springs, Florida, United States
East Lake Oncology
🇺🇸Palm Harbor, Florida, United States
Gainesville Hematology Oncology Associates
🇺🇸Gainesville, Florida, United States
University of Iowa Hospitals and Clinics
🇺🇸Iowa City, Iowa, United States
Annapolis Oncology Center
🇺🇸Annapolis, Maryland, United States
University of Massachusetts
🇺🇸Shrewsbury, Massachusetts, United States
Hemophilia Center of Western New York Incorporated
🇺🇸Buffalo, New York, United States
New York University School of Medicine
🇺🇸New York, New York, United States
Mount Sinai School of Medicine
🇺🇸New York, New York, United States
St. Joseph's
🇺🇸Paterson, New Jersey, United States
University of Virginia Health Systems
🇺🇸Charlottesville, Virginia, United States
O & O Alpan, LLC
🇺🇸Springfield, Virginia, United States
The Permanente Medical Group
🇺🇸Sacramento, California, United States
Adventis Healthcare System dba Florida Hospital
🇺🇸Orlando, Florida, United States
The University Research Foundation for Lysosomal Storage Diseases
🇺🇸Kansas City, Missouri, United States
Yale University
🇺🇸New Haven, Connecticut, United States