Status:
APPROVED_FOR_MARKETING
Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease
Lead Sponsor:
Shire
Conditions:
Gaucher Disease, Type 1
Eligibility:
All Genders
3+ years
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 macro...
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 hepatome...
Eligibility Criteria
Inclusion
- 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
- None
Key Trial Info
Start Date :
Trial Type :
EXPANDED_ACCESS
End Date :
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT00954460
Last Update
May 21 2021
Active Locations (32)
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1
St Joseph's Hospital & Medical Center
Phoenix, Arizona, United States, 85013
2
Tower Hematology Oncology
Beverly Hills, California, United States, 90211-1850
3
Rady's Children's Hospital of San Diego
La Jolla, California, United States, 92093
4
Southern California Permanente Medical Group
Los Angeles, California, United States, 90027