Status:
COMPLETED
An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease
Lead Sponsor:
Shire
Conditions:
Gaucher Disease, Type 1
Eligibility:
All Genders
2+ years
Phase:
PHASE3
Brief Summary
The purpose of this study is to evaluate the long-term safety of every other week dosing of Gene-Activated® human glucocerebrosidase (GA-GCB, velaglucerase alfa) intravenously in patients with type 1 ...
Detailed Description
Type 1 Gaucher disease, the most common form,accounts for more than 90% of all cases and does not involve the CNS. Typical manifestations of type 1 Gaucher disease include hepatomegaly, splenomegaly, ...
Eligibility Criteria
Inclusion
- The patient has completed study TKT032 or TKT034, or study HGT-GCB-039.
- Female patients of child-bearing potential must agree to use a medically acceptable method of contraception at all times during the study and must have negative results to a pregnancy test performed at the time of enrollment and as required throughout their participation in the study.
- Male patients must agree to use a medically acceptable method of contraception at all times during the study and report a partner's pregnancy to the investigator.
- The patient, the patient's parent(s) or legal guardian(s) has provided written informed consent that has been approved by the Institutional Review Board/Independent Ethics Committee (IRB/IEC).
- The patient must be sufficiently cooperative to participate in this clinical study as judged by the Investigator
Exclusion
- The patient has received treatment with any non-Gaucher disease-related investigational drug or device within the 30 days prior to study entry; such use during the study is not permitted.
- The patient is pregnant or lactating.
- The patient, patient's parent(s), or patient's legal guardian(s) is/are unable to understand the nature, scope, and possible consequences of the study.
- The patient has a significant comorbidity(ies) that might affect study data or confound the study results (e.g., malignancies, primary biliary cirrhosis, autoimmune liver disease, etc.).
- The patient is unable to comply with the protocol, e.g., has a clinically relevant medical condition making implementation of the protocol difficult, has an uncooperative attitude, is unable to return for safety evaluations, or is otherwise unlikely to complete the study, as determined by the Investigator
Key Trial Info
Start Date :
March 13 2008
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 28 2012
Estimated Enrollment :
95 Patients enrolled
Trial Details
Trial ID
NCT00635427
Start Date
March 13 2008
End Date
December 28 2012
Last Update
June 10 2021
Active Locations (21)
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1
Los Angeles Medical Center
Los Angeles, California, United States, 90027
2
Children's Hospital Oakland
Oakland, California, United States, 94609
3
Emory University School of Medicine
Atlanta, Georgia, United States, 30322
4
Children's Memorial Hospital
Chicago, Illinois, United States, 60614