The Safety and Efficacy Study of ISU302 in Patient With Type I Gaucher Disease

A Multi-national Randomized Double Blinded Phase III Study to Evaluate the Safety and Efficacy of ISU302(Imiglucerase for Injection) or Cerezyme in Patient With Type I Gaucher Disease

The purpose of this study is to compare and evaluate the efficacy and safety of ISU302, an investigational product, and Cerezyme®, comparator, for Type 1 Gaucher Disease patients

Study Overview

• Status: Withdrawn
• Conditions: Gaucher Disease
• Intervention / Treatment: Drug: Cerezyme®; Drug: ISU302

• Study Type: Interventional
• Phase: Phase 3

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 75 years (ADULT, OLDER_ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• The subjects have a diagnosis of Type I Gaucher Disease
• Subjects between 2 years old and 75 years old
• Subjects documented with glucocerebrosidase deficiency
• Subjects with splenomegaly (as indicated by CT volumetric analysis as 5 times over than the standard size (0.2% of total body weight in kilograms))
• A hemoglobin concentration level:Male > 12 years of age <12.0 g/dL Female>12 years of age<11.0 g/dL Child > 2 years of age and <12 years of age <10.5 g/dL
• Platelet count of < 120,000 / ㎣
• Treatment-naive to enzyme replacement therapy (ERT) or treatment- experienced subjects who have not received ERT in the 12 months before screening and antibody test result is negative
• Treatment naive to substrate reduction therapy (SRT) or treatment- experienced subjects who have not received SRT in the 12 months before screening
• Subjects or their spouses who provide consent to use one of following contraception methods, or women in menopause. (In this case, menopause is defined as a period after 12 months from the last menstruation)
• Condoms, sponge, foams, jellies, diaphragm, or intrauterine device (Spouse or patient who had vasectomy or tubal ligation/ hysterectomy)
• Total abstinence from sexual intercourse: Female patient using oral contraceptives must use other contraception method (barrier method) also during the trial period and after the completion of trial as well as up to 90 days from the conclusion of trial.
• The subject or their legal representative has signed the informed consent.

Exclusion Criteria:

• Treatment with any investigational product in 90 days before study entry
• Partial or total splenectomy
• Subjects who have a serious concurrent disease like infection or who abuse addictive drug and substances.
• Pregnant and/or breast-feeding women
• Presence of Hepatitis B surface antigen or Hepatitis C or the patients show positive reaction to human immunodeficiency virus (HIV) type1
• Subjects with a history of allergic reaction to Imiglucerase
• Subjects with a history of severe pulmonary hypertension caused by Gaucher Disease
• Any subject whom the investigator or the sub investigator considers as inad equate for this trial

Study Plan

How is the study designed?

Design Details

• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: DOUBLE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
ACTIVE_COMPARATOR: Cerezyme®; 60 U/kg infusion (every 2 weeks for 24 weeks)	Drug: Cerezyme®; administered by IV infusion for a dose of 60 U/kg
EXPERIMENTAL: ISU302; 60 U/kg infusion (every 2 weeks for 24 weeks)	Drug: ISU302; administered by IV infusion for a dose of 60 U/kg

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
change in splenic volume compared to baseline	Primary endpoints to demonstrate non-inferiority of ISU302 against Cerezyme® for change in splenic volume compared to baseline.	one year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
change in organ parameters and biochemical value compared to baseline	Change in splenic volume compared to baseline for all subjects enrolled; Change in hemoglobin concentration; Change in platelet counts; Change in liver volume compared to baseline; Change in range variation of liver function test (ALT/AST); Change in skeletal status improvement level; Change in bone mineral density; Change in biomarkers (acid phosphatase, angiotensin-converting enzyme (ACE) and chitotriosidase); After the 1st administration, to evaluate pharmacokinetic profile by glucocerebrosidase activity assay	one year

Collaborators and Investigators

• Sponsor: ISU Abxis Co., Ltd.

Study record dates

Study Major Dates

• Study Start: January 1, 2011
• Primary Completion (ANTICIPATED): August 1, 2011

Study Registration Dates

• First Submitted: July 11, 2010
• First Submitted That Met QC Criteria: July 13, 2010
• First Posted (ESTIMATE): July 14, 2010

Study Record Updates

• Last Update Posted (ESTIMATE): August 23, 2016
• Last Update Submitted That Met QC Criteria: August 22, 2016
• Last Verified: July 1, 2010

More Information

Terms related to this study

• Keywords: 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: ISU302-2008