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

Gaucher Disease Clinical Trial

Official 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

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT01161914
• Other study ID #: ISU302-2008
• Status: Withdrawn
• Phase: Phase 3
• First received: July 11, 2010
• Last updated: August 22, 2016
• Start date: January 2011

Study information

• Verified date: July 2010
• Source: ISU Abxis Co., Ltd.
• Contact: n/a
• Is FDA regulated: No
• Health authority: Korea: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. primary completion date: August 2011
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 2 Years to 75 Years

Eligibility

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 Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator)

Related Conditions & MeSH terms

• Gaucher Disease

Intervention

Drug:
• Cerezyme®
administered by IV infusion for a dose of 60 U/kg
• ISU302
administered by IV infusion for a dose of 60 U/kg

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
ISU Abxis Co., Ltd.

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	No
Secondary	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	No