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

Gaucher Disease Clinical Trial

Official title:

A Phase 2 Multi-Center, Open-Label, Switch-Over Trial to Evaluate the Safety and Efficacy of ISU302 in Patients With Type 1 Gaucher Disease Previously Treated With Imiglucerase

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02053896
• Other study ID #: ISU302-003
• 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

• Gaucher Disease

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