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

NCT02053896 | Completed

• 1 other identifier: ISU302-003
• Study Type: observational
• Target: 5
• Locations: 0 countries
• Sites: N/A

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

Keywords

Type I Gaucher; ISU302; Imiglucerase

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (6)

• Pharmacokinetics

  From Screening to Week 24

• hemoglobin concentration

  From Screening to Week 24

• platelet count

  From Screening to Week 24

• liver and spleen volumes and liver function

  From Screening to Week 24

• biomarker levels

  From Screening to Week 24

Study Arms (1)

ISU302

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

Eligibility Criteria

• Age: 8 Years - 29 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Probability Sample

Study Population

Patient diagnosed with type-1 Gaucher disease

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• ISU Abxis Co., Ltd.: lead

Related Publications (1)

• 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.

  PMID: 25829804 DERIVED

MeSH Terms

Conditions

Gaucher Disease

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 26, 2014
• First Posted: February 4, 2014
• Study Start: May 1, 2011
• Primary Completion: February 1, 2012
• Study Completion: February 1, 2012
• Last Updated: February 4, 2014

Record last verified: 2014-02