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

• Register: ClinicalTrials.gov
• Last refreshed on: 5 September 2016
• Main ID: NCT01161914
• Date of registration: 11/07/2010
• Prospective Registration: Yes
• Primary sponsor: ISU Abxis Co., Ltd.
• Public title: The Safety and Efficacy Study of ISU302 in Patient With Type I Gaucher Disease
• Scientific title: 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
• Date of first enrolment: January 2011
• Target sample size: 0
• Recruitment status: Withdrawn
• URL: https://clinicaltrials.gov/show/NCT01161914
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator)
• Phase: Phase 3

Key inclusion & exclusion criteria

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



Age minimum: 2 Years
Age maximum: 75 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Gaucher Disease

Intervention(s)

Drug: ISU302

Drug: Cerezyme®

Primary Outcome(s)

change in splenic volume compared to baseline [Time Frame: one year]

Secondary Outcome(s)

change in organ parameters and biochemical value compared to baseline [Time Frame: one year]

Secondary ID(s)

ISU302-2008

Source(s) of Monetary Support

Please refer to primary and secondary sponsors