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

The recruitment status of this study is unknown because the information has not been verified recently.
Verified July 2010 by ISU Abxis Co., Ltd..
Recruitment status was  Not yet recruiting
Sponsor:
Information provided by:
ISU Abxis Co., Ltd.
ClinicalTrials.gov Identifier:
NCT01161914
First received: July 11, 2010
Last updated: July 13, 2010
Last verified: July 2010
History of Changes
  Purpose

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


Condition Intervention Phase
Gaucher Disease
 Drug: Cerezyme®
Drug: ISU302
 Phase 3

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

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by ISU Abxis Co., Ltd.:

Primary Outcome Measures:
  • change in splenic volume compared to baseline [ Time Frame: one year ] [ Designated as safety issue: No ]
    Primary endpoints to demonstrate non-inferiority of ISU302 against Cerezyme® for change in splenic volume compared to baseline.


Secondary Outcome Measures:
  • change in organ parameters and biochemical value compared to baseline [ Time Frame: one year ] [ Designated as safety issue: No ]
    • 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


Estimated Enrollment: 18
Study Start Date: January 2011
Estimated Primary Completion Date: August 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

  Eligibility

Ages Eligible for Study:   2 Years to 75 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
No Contacts or Locations Provided
  More Information

No publications provided

Responsible Party: Lee, JaeHeung, ISU ABXIS CO., LTD
ClinicalTrials.gov Identifier: NCT01161914     History of Changes
Other Study ID Numbers: ISU302-2008
Study First Received: July 11, 2010
Last Updated: July 13, 2010
Health Authority: Korea: Food and Drug Administration

Keywords provided by ISU Abxis Co., Ltd.:
Type I Gaucher

Additional relevant MeSH terms:
Gaucher Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
 Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders

ClinicalTrials.gov processed this record on May 16, 2013