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

NCT01161914 | Withdrawn Phase 3

• 1 other identifier: ISU302-2008
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief 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

Conditions

Gaucher Disease

Keywords

Type I Gaucher

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• change in organ parameters and biochemical value compared to baseline

  one year

Study Arms (2)

Cerezyme®

ACTIVE COMPARATOR

60 U/kg infusion (every 2 weeks for 24 weeks)

Drug: Cerezyme®

ISU302

EXPERIMENTAL

60 U/kg infusion (every 2 weeks for 24 weeks)

Drug: ISU302

Interventions

Cerezyme® DRUG

administered by IV infusion for a dose of 60 U/kg

Cerezyme®

ISU302 DRUG

administered by IV infusion for a dose of 60 U/kg

ISU302

Eligibility Criteria

• Age: 2 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• ISU Abxis Co., Ltd.: lead

MeSH Terms

Conditions

Gaucher Disease

Interventions

imiglucerase

Condition Hierarchy (Ancestors)

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; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY

Study Record Dates

• First Submitted: July 11, 2010
• First Posted: July 14, 2010
• Study Start: January 1, 2011
• Primary Completion: August 1, 2011
• Last Updated: August 23, 2016

Record last verified: 2010-07