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

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

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.

Study Overview

• Status: Completed
• Conditions: Gaucher Disease

• Study Type: Observational
• Enrollment (Actual): 5

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 8 years to 29 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Probability Sample

Study Population

Patient diagnosed with type-1 Gaucher disease

Description

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 Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
ISU302; 15~60U/kg (once every 2 weeks for 6 months)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Pharmacokinetics	Measurement of blood glucocerebrosidase activity for pharmacokinetic assessment after initial study drug administration	From Screening to Week 24
hemoglobin concentration	Change in hemoglobin concentration	From Screening to Week 24
platelet count	Change in platelet count	From Screening to Week 24
liver and spleen volumes and liver function	Changes in liver and spleen volumes and liver function	From Screening to Week 24
biomarker levels	Changes in biomarker levels (Acid Phosphatase, Angiotensin Converting Enzyme, and Chitotriosidase)	From Screening to Week 24
skeletal status and bone mineral density	Changes in skeletal status and bone mineral density	From Screening to Week 24

Collaborators and Investigators

• Sponsor: ISU Abxis Co., Ltd.

Publications and helpful links

General Publications

• 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. Erratum In: J Korean Med Sci. 2015 Sep;30(9):1373.

Study record dates

Study Major Dates

• Study Start: May 1, 2011
• Primary Completion (Actual): February 1, 2012
• Study Completion (Actual): February 1, 2012

Study Registration Dates

• First Submitted: January 26, 2014
• First Submitted That Met QC Criteria: February 3, 2014
• First Posted (Estimate): February 4, 2014

Study Record Updates

• Last Update Posted (Estimate): February 4, 2014
• Last Update Submitted That Met QC Criteria: February 3, 2014
• Last Verified: February 1, 2014

More Information

Terms related to this study

• Keywords: Imiglucerase; ISU302; Type I Gaucher
• Additional Relevant MeSH Terms: Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Lipid Metabolism Disorders; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Lipidoses; Lipid Metabolism, Inborn Errors; Gaucher Disease
• Other Study ID Numbers: ISU302-003