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

ISU Abxis

Status: Completed

Conditions: Gaucher Disease

Study type: Observational

Funder types: Industry

Identifiers

NCT02053896

ISU302-003

Details and patient eligibility

About

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.

Enrollment: 5 patients

Sex: All

Ages: 8 to 29 years old

Volunteers: No Healthy Volunteers

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

Trial design

5 participants in 1 patient group

ISU302

Description:

15\~60U/kg (once every 2 weeks for 6 months)