Gaucher Disease
Conditions
Keywords
Type I Gaucher, ISU302, Imiglucerase
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.
Interventions
None listed
Sponsors
ISU Abxis Co., Ltd.
Study design
Observational model
CASE_ONLY
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
8 Years to 29 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| hemoglobin concentration | From Screening to Week 24 | Change in hemoglobin concentration |
| platelet count | From Screening to Week 24 | Change in platelet count |
| Pharmacokinetics | From Screening to Week 24 | Measurement of blood glucocerebrosidase activity for pharmacokinetic assessment after initial study drug administration |
| biomarker levels | From Screening to Week 24 | Changes in biomarker levels (Acid Phosphatase, Angiotensin Converting Enzyme, and Chitotriosidase) |
| skeletal status and bone mineral density | From Screening to Week 24 | Changes in skeletal status and bone mineral density |
| liver and spleen volumes and liver function | From Screening to Week 24 | Changes in liver and spleen volumes and liver function |
Outcome results
None listed