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

NCT ID: NCT02053896

Last Updated: 2014-02-04

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 5 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2011-05-31
• Study Completion Date: 2012-02-29

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.

Conditions

Gaucher Disease

Study Design

• Observational Model Type: CASE_ONLY
• Study Time Perspective: PROSPECTIVE

Study Groups

ISU302

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

No interventions assigned to this group

Eligibility Criteria

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

• Minimum Eligible Age: 8 Years
• Maximum Eligible Age: 29 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

ISU Abxis Co., Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

References

Choi JH, Lee BH, Ko JM, Sohn YB, Lee JS, Kim GH, Heo SH, Park JY, Kim YM, Kim JH, Yoo HW. A phase 2 multi-center, open-label, switch-over trial to evaluate the safety and efficacy of Abcertin(R) in patients with type 1 Gaucher disease. J Korean Med Sci. 2015 Apr;30(4):378-84. doi: 10.3346/jkms.2015.30.4.378. Epub 2015 Mar 19.

Reference Type: DERIVED

PMID: 25829804 (View on PubMed)

Other Identifiers

ISU302-003

Identifier Type: -

Identifier Source: org_study_id