Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease

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Conditions studied: Gaucher Disease, Type 1

In brief

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.

Key facts

Study ID

NCT00954460

Run by

Shire

Last updated by the study team

2021-05-21

Who can join

Age: 3 and older. Sex: any. Healthy volunteers: not accepted.

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
• Exclusion Criteria: None

Where it is running

• St Joseph's Hospital & Medical Center — Phoenix, Arizona, United States
• Tower Hematology Oncology — Beverly Hills, California, United States
• Rady's Children's Hospital of San Diego — La Jolla, California, United States
• Southern California Permanente Medical Group — Los Angeles, California, United States
• The Permanente Medical Group — Sacramento, California, United States
• Stanford University Medical Genetics — Stanford, California, United States
• Rocky Mountain Cancer Centers — Denver, Colorado, United States
• Yale University — New Haven, Connecticut, United States
• University Research Foundation for Lysosomal Storage Diseases — Coral Springs, Florida, United States
• Gainesville Hematology Oncology Associates — Gainesville, Florida, United States
• Adventis Healthcare System dba Florida Hospital — Orlando, Florida, United States
• East Lake Oncology — Palm Harbor, Florida, United States
• Emory Genetics — Decatur, Georgia, United States
• Children's Memorial Hospital — Chicago, Illinois, United States
• University of Iowa Hospitals and Clinics — Iowa City, Iowa, United States
• Annapolis Oncology Center — Annapolis, Maryland, United States
• Sinai Hospital of Baltimore — Baltimore, Maryland, United States
• University of Massachusetts — Shrewsbury, Massachusetts, United States
• Children's Hospitals and Clinics of Minnesota — Minneapolis, Minnesota, United States
• The University Research Foundation for Lysosomal Storage Diseases — Kansas City, Missouri, United States
• St. Joseph's — Paterson, New Jersey, United States
• Hemophilia Center of Western New York Incorporated — Buffalo, New York, United States
• North Shore Hematology/Oncology - Manhasset — Manhasset, 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

Full record on ClinicalTrials.gov

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