Status:
COMPLETED
A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease
Lead Sponsor:
Shire
Conditions:
Gaucher Disease, Type 1
Eligibility:
All Genders
2+ years
Phase:
PHASE3
Brief Summary
Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to this deficiency of functional GCB, glucocerebroside accumulates within macr...
Detailed Description
Type 1 Gaucher disease, the most common form, accounts for more than 90% of all cases and does not involve the Central Nervous System (CNS). Typical manifestations of type 1 Gaucher disease include he...
Eligibility Criteria
Inclusion
- Patient has a documented diagnosis of type 1 Gaucher disease, as determined by deficient glucocerebrosidase (GCB) activity relative to normal as measured in leukocytes or by genotype analysis and is willing and able to provide written informed consent prior to initiating any study-related procedures
- Patient is at least 2 years of age
- Patient has Gaucher disease-related anemia and
- Patient has at least moderate splenomegaly or
- Patient has Gaucher disease-related thrombocytopenia or
- Patient has a readily palpable enlarged liver
- Patient has not received treatment for Gaucher disease within 30 months prior to study entry
- Female patients of child-bearing potential agree to use a medically acceptable method of contraception. Male patients must agree to use a medically acceptable method of birth control.
- Patient must be sufficiently cooperative to participate in the study as judged by the Investigator.
Exclusion
- Includes:
- Patient has type 2 or 3 Gaucher disease or is suspected of having type 3 Gaucher disease
- Patient is antibody-positive to imiglucerase during screening or has experienced an anaphylactic reaction to imiglucerase
- Patient has received treatment with any investigational drug or device within the 30 days prior to study entry
- Patient is Human immunodeficiency virus (HIV) positive
- Patient is hepatitis positive
- Patient presents with iron, folic acid and/or vitamin B12 deficiency sustained anemia during screening
- 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
- Patient has a significant comorbidity(ies)that might affect study data or confound the study results
- Patient is a pregnant and/or lactating female
- Patient is unable to comply with the protocol or is unlikely to complete the study, as determined by the Investigator
Key Trial Info
Start Date :
February 15 2007
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
April 1 2009
Estimated Enrollment :
25 Patients enrolled
Trial Details
Trial ID
NCT00430625
Start Date
February 15 2007
End Date
April 1 2009
Last Update
June 29 2021
Active Locations (5)
Enter a location and click search to find clinical trials sorted by distance.
1
Hipolito Yrigoyen
Buenos Aires, Argentina
2
Shaare Zedek Medical Center
Jerusalem, Israel
3
Sociedad Espanola de Socorros Mutuos
Asunción, Paraguay
4
National Research Center for Haematology
Moscow, Russia