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

ISU Abxis

Status and phase

Withdrawn

Phase 3

Conditions

Gaucher Disease

Treatments

Drug: ISU302

Drug: Cerezyme®

Study type

Interventional

Funder types

Industry

Identifiers

NCT01161914

ISU302-2008

Details and patient eligibility

About

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

Sex

All

Ages

2 to 75 years old

Volunteers

No Healthy Volunteers

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