NCT02770625

Phase III Study of ISU302 in Patients With Type 1 Gaucher Disease

A Multicenter, Open-Label Phase III Study to Evaluate the Safety and Efficacy of ISU302 (Imiglucerase for Injection) in Patients With Type 1 Gaucher Disease

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 8 (actual)
• Sponsor: ISU Abxis Co., Ltd. · Industry
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the safety and efficacy of ISU302 in patients with Type 1 Gaucher disease.

Detailed description

The objectives of this clinical study were to evaluate the efficacy and safety of every other week (EOW) dosing of ISU302 at a dose of 60 U/kg as an effective glucocerebrosidase enzyme replacement therapeutic product in patients with Type 1 Gaucher disease (GD). Primary efficacy endpoint was the difference in hemoglobin concentration between baseline and Week 24. Secondary efficacy endpoints included assessment of platelet counts, spleen and liver volume, and biomarker levels in plasma at Week 24 compared to baseline; skeletal change and bone mineral density (BMD); and single-dose pharmacokinetic (PK) analysis. Secondary safety endpoints included the assessment of adverse events (AEs), vital signs, physical examination, and electrocardiogram (ECG); clinical safety laboratory analyses included serum chemistry, urinalysis, hematology and coagulation, and the measurement of anti-ISU302 antibodies.

Conditions

• Gaucher Disease, Type 1

Interventions

Type	Name	Description
DRUG	ISU302	60 U/kg given intravenously

Timeline

• Start date: 2011-09-01
• Primary completion: 2013-03-01
• Completion: 2014-08-01
• First posted: 2016-05-12
• Last updated: 2017-07-12
• Results posted: 2017-07-12