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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT02053896
Other study ID # ISU302-003
Secondary ID
Status Completed
Phase Phase 2
First received January 26, 2014
Last updated February 3, 2014
Start date May 2011
Est. completion date February 2012

Study information

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

Clinical Trial 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.


Recruitment information / eligibility

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

Study Design

Observational Model: Case-Only, Time Perspective: Prospective


Related Conditions & MeSH terms


Locations

Country Name City State
n/a

Sponsors (1)

Lead Sponsor Collaborator
ISU Abxis Co., Ltd.

Outcome

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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