Gaucher Disease Clinical Trial
Official title:
A Phase 2 Multi-Center, Open-Label, Switch-Over Trial to Evaluate the Safety and Efficacy of ISU302 in Patients With Type 1 Gaucher Disease Previously Treated With Imiglucerase
Verified date | February 2014 |
Source | ISU Abxis Co., Ltd. |
Contact | n/a |
Is FDA regulated | No |
Health authority | Korea: Food and Drug Administration |
Study type | Observational |
The purpose of this study is to evaluate the safety and efficacy of ISU302 in patients with Type 1 Gaucher disease previously treated with Imiglucerase.
Status | Completed |
Enrollment | 5 |
Est. completion date | February 2012 |
Est. primary completion date | February 2012 |
Accepts healthy volunteers | No |
Gender | Both |
Age group | 8 Years to 29 Years |
Eligibility |
Inclusion Criteria: - Patient diagnosed with type-1 Gaucher disease - Patient who was stably treating Gaucher disease with Cerezyme® and who was maintaining the usage and dosage of Cerezyme® for at least 6 months prior to study drug administration - Patient aged 2 years or higher - Female patient with contraception during the study period (oral or injectable contraceptive hormones, intrauterine device, physical devices using condom, sponge form, jelly, and femidom, and abstinence) - Patient who signed the informed consent form after hearing the detailed explanation about this study - Definition of the stable treatment of type-1 Gaucher disease: - No neurologic deficit - Normal hemoglobin concentration, and platelet count that has increased to =100,000/?, or maintained to 100,000/? - Normal or no deteriorated bone mineral density - Normal or no deteriorated splenomegaly or hepatomegaly Exclusion Criteria: - Patient who participated in other clinical studies within 90 days before study drug administration - Patient with unstable hemoglobin and platelet counts for at least 6 months before study drug administration - Patient with hypersensitivity to Cerezyme® - Patient positive to HIV antibody, hepatitis B antigen, and hepatitis C antibody - Patient with Fe, folic acid, or vitamin B12-deficcient anemia - Patient who received miglustat within 6 months before study drug administration - Patient who received erythrocyte growth factor or chronic systemic corticosteroids within 6 months before study drug administration - Patient who had clinically significant splenic obstruction within 12 months before study drug administration - Pregnant or lactating patient - Patient who had serious concurrent diseases such as infectious diseases or drug-addicted patient - Patient who was considered inappropriate for this study by the investigators or sub-investigators |
Observational Model: Case-Only, Time Perspective: Prospective
Country | Name | City | State |
---|---|---|---|
n/a |
Lead Sponsor | Collaborator |
---|---|
ISU Abxis Co., Ltd. |
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Number of participants with Adverse Events | To evaluate the safety of ISU302 in patients with Type 1 Gaucher disease previously treated with Cerezyme® after administering ISU302 alternative to Cerezyme® | From Screening to Week 24 | No |
Secondary | Pharmacokinetics | Measurement of blood glucocerebrosidase activity for pharmacokinetic assessment after initial study drug administration | From Screening to Week 24 | No |
Secondary | hemoglobin concentration | Change in hemoglobin concentration | From Screening to Week 24 | No |
Secondary | platelet count | Change in platelet count | From Screening to Week 24 | No |
Secondary | liver and spleen volumes and liver function | Changes in liver and spleen volumes and liver function | From Screening to Week 24 | No |
Secondary | biomarker levels | Changes in biomarker levels (Acid Phosphatase, Angiotensin Converting Enzyme, and Chitotriosidase) | From Screening to Week 24 | No |
Secondary | skeletal status and bone mineral density | Changes in skeletal status and bone mineral density | From Screening to Week 24 | No |
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