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

Launched by SHIRE · Aug 6, 2009

Keywords

ClinConnect Summary

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 occurri...

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Eligibility criteria

• Inclusion Criteria:
• • 1. The patient has a documented diagnosis of type 1 Gaucher disease
• • 2. The patient is \> 2 years of age
• • 3. The patient has NOT previously experienced an anaphylactic or anaphylactoid reaction to another ERT including imiglucerase
• • 4. 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.
• • 5. The patient is sufficiently cooperative to participate in this treatment plan as judged by the Investigator
• 6. 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