Trial record 10 of 21 for:    "Gaucher disease type 1"

A Switch-Over Study of the Safety and Efficacy of ISU302 in Patients With Type 1 Gaucher Disease

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
ISU Abxis Co., Ltd.
ClinicalTrials.gov Identifier:
NCT02053896
First received: January 26, 2014
Last updated: February 3, 2014
Last verified: February 2014
  Purpose

The purpose of this study is to evaluate the safety and efficacy of ISU302 in patients with Type 1 Gaucher disease previously treated with Imiglucerase.


Condition Phase
Gaucher Disease
Phase 2

Study Type: Observational
Study Design: Observational Model: Case-Only
Time Perspective: Prospective
Official Title: A Phase 2 Multi-Center, Open-Label, Switch-Over Trial to Evaluate the Safety and Efficacy of ISU302 in Patients With Type 1 Gaucher Disease Previously Treated With Imiglucerase

Resource links provided by NLM:


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

Primary Outcome Measures:
  • Number of participants with Adverse Events [ Time Frame: From Screening to Week 24 ] [ Designated as safety issue: No ]
    To evaluate the safety of ISU302 in patients with Type 1 Gaucher disease previously treated with Cerezyme® after administering ISU302 alternative to Cerezyme®


Secondary Outcome Measures:
  • Pharmacokinetics [ Time Frame: From Screening to Week 24 ] [ Designated as safety issue: No ]
    Measurement of blood glucocerebrosidase activity for pharmacokinetic assessment after initial study drug administration

  • hemoglobin concentration [ Time Frame: From Screening to Week 24 ] [ Designated as safety issue: No ]
    Change in hemoglobin concentration

  • platelet count [ Time Frame: From Screening to Week 24 ] [ Designated as safety issue: No ]
    Change in platelet count

  • liver and spleen volumes and liver function [ Time Frame: From Screening to Week 24 ] [ Designated as safety issue: No ]
    Changes in liver and spleen volumes and liver function

  • biomarker levels [ Time Frame: From Screening to Week 24 ] [ Designated as safety issue: No ]
    Changes in biomarker levels (Acid Phosphatase, Angiotensin Converting Enzyme, and Chitotriosidase)

  • skeletal status and bone mineral density [ Time Frame: From Screening to Week 24 ] [ Designated as safety issue: No ]
    Changes in skeletal status and bone mineral density


Enrollment: 5
Study Start Date: May 2011
Study Completion Date: February 2012
Primary Completion Date: February 2012 (Final data collection date for primary outcome measure)
Groups/Cohorts
ISU302
15~60U/kg (once every 2 weeks for 6 months)

  Eligibility

Ages Eligible for Study:   8 Years to 29 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population

Patient diagnosed with type-1 Gaucher disease

Criteria

Inclusion Criteria:

  • Patient diagnosed with type-1 Gaucher disease
  • Patient who was stably treating Gaucher disease with Cerezyme® and who was maintaining the usage and dosage of Cerezyme® for at least 6 months prior to study drug administration
  • Patient aged 2 years or higher
  • Female patient with contraception during the study period (oral or injectable contraceptive hormones, intrauterine device, physical devices using condom, sponge form, jelly, and femidom, and abstinence)
  • Patient who signed the informed consent form after hearing the detailed explanation about this study

    • Definition of the stable treatment of type-1 Gaucher disease:
  • No neurologic deficit
  • Normal hemoglobin concentration, and platelet count that has increased to ≥100,000/㎣, or maintained to 100,000/㎣
  • Normal or no deteriorated bone mineral density
  • Normal or no deteriorated splenomegaly or hepatomegaly

Exclusion Criteria:

  • Patient who participated in other clinical studies within 90 days before study drug administration
  • Patient with unstable hemoglobin and platelet counts for at least 6 months before study drug administration
  • Patient with hypersensitivity to Cerezyme®
  • Patient positive to HIV antibody, hepatitis B antigen, and hepatitis C antibody
  • Patient with Fe, folic acid, or vitamin B12-deficcient anemia
  • Patient who received miglustat within 6 months before study drug administration
  • Patient who received erythrocyte growth factor or chronic systemic corticosteroids within 6 months before study drug administration
  • Patient who had clinically significant splenic obstruction within 12 months before study drug administration
  • Pregnant or lactating patient
  • Patient who had serious concurrent diseases such as infectious diseases or drug-addicted patient
  • Patient who was considered inappropriate for this study by the investigators or sub-investigators
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

No Contacts or Locations Provided
  More Information

No publications provided

Responsible Party: ISU Abxis Co., Ltd.
ClinicalTrials.gov Identifier: NCT02053896     History of Changes
Other Study ID Numbers: ISU302-003
Study First Received: January 26, 2014
Last Updated: February 3, 2014
Health Authority: Korea: Food and Drug Administration

Keywords provided by ISU Abxis Co., Ltd.:
Type I Gaucher
ISU302
Imiglucerase

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 September 18, 2014