A Switch-Over Study of the Safety and Efficacy of ISU302 in Patients With Type 1 Gaucher Disease

Completed

• Conditions: Gaucher Disease

• Registration Number: NCT02053896
• Lead Sponsor: ISU Abxis Co., Ltd.

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2011-05
• Primary Completion: 2012-02
• Study Completion: 2012-02
• Study First Submitted: 2014-01-26
• Status Verified: 2014-02
• Study First Submitted QC: 2014-02-03
• Last Update Submitted: 2014-02-03
• Study First Posted: 2014-02-04
• Last Update Posted: 2014-02-04

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 5

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Number of participants with Adverse Events	From Screening to Week 24	To evaluate the safety of ISU302 in patients with Type 1 Gaucher disease previously treated with Cerezyme® after administering ISU302 alternative to Cerezyme®

Secondary Outcome Measures

Name	Time	Method
skeletal status and bone mineral density	From Screening to Week 24	Changes in skeletal status and bone mineral density
platelet count	From Screening to Week 24	Change in platelet count
liver and spleen volumes and liver function	From Screening to Week 24	Changes in liver and spleen volumes and liver function
Pharmacokinetics	From Screening to Week 24	Measurement of blood glucocerebrosidase activity for pharmacokinetic assessment after initial study drug administration
hemoglobin concentration	From Screening to Week 24	Change in hemoglobin concentration
biomarker levels	From Screening to Week 24	Changes in biomarker levels (Acid Phosphatase, Angiotensin Converting Enzyme, and Chitotriosidase)