Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease
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Purpose
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.
| Condition | Intervention |
|---|---|
|
Gaucher Disease, Type 1 |
Drug: velaglucerase alfa |
| Study Type: | Expanded Access What is Expanded Access? |
| 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 |
-
Drug: velaglucerase alfa
- VPRIV
- Gene activated human glucocerebrosidase
- GA-GCB
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-Activated™ 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.
Eligibility| Ages Eligible for Study: | 3 Years and older |
| Genders Eligible for Study: | Both |
Inclusion Criteria:
- 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
Exclusion Criteria: None
Contacts and Locations
Hide Study Locations| United States, Arizona | |
| St Joseph's Hospital & Medical Center | |
| Phoenix, Arizona, United States, 85013 | |
| United States, California | |
| Tower Hematology Oncology | |
| Beverly Hills, California, United States, 90211-1850 | |
| Rady's Children's Hospital of San Diego | |
| La Jolla, California, United States, 92093 | |
| Southern California Permanente Medical Group | |
| Los Angeles, California, United States, 90027 | |
| The Permanente Medical Group | |
| Sacramento, California, United States, 95815 | |
| Stanford University Medical Genetics | |
| Stanford, California, United States, 94305-5208 | |
| United States, Colorado | |
| Rocky Mountain Cancer Centers | |
| Denver, Colorado, United States, 80218 | |
| United States, Connecticut | |
| Yale University | |
| New Haven, Connecticut, United States, 06510 | |
| United States, Florida | |
| University Research Foundation for Lysosomal Storage Diseases | |
| Coral Springs, Florida, United States, 33065 | |
| Gainesville Hematology Oncology Associates | |
| Gainesville, Florida, United States, 32605-4218 | |
| Adventis Healthcare System dba Florida Hospital | |
| Orlando, Florida, United States, 32804-4603 | |
| East Lake Oncology | |
| Palm Harbor, Florida, United States, 34685 | |
| United States, Georgia | |
| Emory Genetics | |
| Decatur, Georgia, United States, 30033 | |
| United States, Illinois | |
| Children's Memorial Hospital | |
| Chicago, Illinois, United States, 60614 | |
| United States, Iowa | |
| University of Iowa Hospitals and Clinics | |
| Iowa City, Iowa, United States, 52242 | |
| United States, Maryland | |
| Annapolis Oncology Center | |
| Annapolis, Maryland, United States, 21401 | |
| Sinai Hospital of Baltimore | |
| Baltimore, Maryland, United States, 21215 | |
| United States, Massachusetts | |
| University of Massachusetts | |
| Shrewsbury, Massachusetts, United States, 01545 | |
| United States, Minnesota | |
| Children's Hospitals and Clinics of Minnesota | |
| Minneapolis, Minnesota, United States, 55404 | |
| United States, Missouri | |
| The University Research Foundation for Lysosomal Storage Diseases | |
| Kansas City, Missouri, United States, 64108-4619 | |
| United States, New Jersey | |
| St. Joseph's | |
| Patterson, New Jersey, United States, 07503 | |
| United States, New York | |
| Hemophilia Center of Western New York Incorporated | |
| Buffalo, New York, United States, 14215 | |
| North Shore Hematology/Oncology - Manhasset | |
| Manhasset, New York, United States, 11030 | |
| New York University School of Medicine | |
| New York, New York, United States, 10016 | |
| Mount Sinai School of Medicine | |
| New York, New York, United States, 10029-6500 | |
| United States, North Carolina | |
| Fullerton Genetic | |
| Ashville, North Carolina, United States, 28801-4420 | |
| Duke Medical Center | |
| Durham, North Carolina, United States, 27710 | |
| United States, Ohio | |
| Akron Children's Hospital | |
| Akron, Ohio, United States, 44308 | |
| Cincinnati Children's Hospital Medical Center | |
| Cincinnati, Ohio, United States, 45229 | |
| United States, Pennsylvania | |
| Children's Hospital of Philadelphia | |
| Philadelphia, Pennsylvania, United States, 19104 | |
| United States, Virginia | |
| University of Virginia Health Systems | |
| Charlottesville, Virginia, United States, 22908-0386 | |
| O & O Alpan, LLC | |
| Springfield, Virginia, United States, 22152 | |
| Study Director: | Gabriel M. Cohn, M.D. | Shire Human Genetic Therapies, Inc. |
More Information
No publications provided
| Responsible Party: | Shire Human Genetic Therapies, Inc. |
| ClinicalTrials.gov Identifier: | NCT00954460 History of Changes |
| Other Study ID Numbers: | HGT-GCB-058 |
| Study First Received: | August 5, 2009 |
| Last Updated: | June 26, 2012 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by Shire Human Genetic Therapies, Inc.:
|
VPRIV Enzyme Replacement Therapy Gaucher disease glucocerebrosidase beta-glucocerebrosidase |
Acid beta-glucocerebrosidase glucosylceramidase D-glucosyl-N-acylsphingosine glucohydrolase gene activation human |
Additional relevant MeSH terms:
|
Gaucher Disease Sphingolipidoses Lysosomal Storage Diseases, Nervous System Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases |
Metabolism, Inborn Errors Genetic Diseases, Inborn Lipidoses Lipid Metabolism, Inborn Errors Lysosomal Storage Diseases Metabolic Diseases Lipid Metabolism Disorders |
ClinicalTrials.gov processed this record on May 21, 2013