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

• Status: Approved for marketing
• Trial end date: 1969-12-31
• Target enrollment: 0
• Participant gender: All

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.

Details

• Lead Sponsor: Shire

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