To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Green Cross Corporation
ClinicalTrials.gov Identifier:
NCT01301898
First received: February 22, 2011
Last updated: February 12, 2012
Last verified: February 2012
  Purpose

The purpose of this study is to evaluate the safety and efficacy of GC1111 (recombinant human iduronate-w-sulfatase) in Hunter Syndrome (Mucopolysaccharidosis II) patients


Condition Intervention Phase
Mucopolysaccharidosis II
Drug: GC1111_0.5mg/kg
Drug: GC1111_1.0mg/kg
Drug: Elaprase_0.5mg/kg
Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Single Blind (Subject)
Primary Purpose: Treatment
Official Title: Randomized, Single-blind, Active-controlled, Phase 1/2 Study to Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome (Mucopolysaccharidosis II) Patients

Resource links provided by NLM:


Further study details as provided by Green Cross Corporation:

Primary Outcome Measures:
  • Primary Outcome [ Time Frame: baseline, every 4 weeks ] [ Designated as safety issue: No ]
    measurement of % change of Urine GAG


Secondary Outcome Measures:
  • Secondary Outcome [ Time Frame: baseline, every 12 weeks ] [ Designated as safety issue: Yes ]
    • Measurements of the six-minute-walk test (6-MWT)
    • Measurements of liver volume
    • Measurements of heart size and heart function
    • Measurements of joint range of motion
    • Measurements of urine GAG levels
    • Measurements of pulmonary function
    • Evaluate the safety: vital signs, physical examination, laboratory tests, adverse events, immunogenecity


Enrollment: 31
Study Start Date: May 2010
Study Completion Date: March 2011
Primary Completion Date: March 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: GC1111_0.5mg/kg Drug: GC1111_0.5mg/kg
Experimental: GC1111_1.0mg/kg Drug: GC1111_1.0mg/kg
Active Comparator: Elaprase_0.5mg/kg Drug: Elaprase_0.5mg/kg

  Eligibility

Ages Eligible for Study:   6 Years to 35 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Patients with diagnosis of MPS II based on both clinical and biochemical criteria
  2. Male, ages 6 to 35 years old
  3. Patients who are able to comply with the study requirements
  4. Patients who have given voluntary written consent to participate in the study
  5. Patients who is acceptable for using an appropriate method of contraception

Exclusion Criteria:

  1. History of a tracheostomy or a bone marrow transplant
  2. Known hypersensitivity to idursulfase
  3. Known shock to idursulfase
  4. History of receiving treatment with another investigational therapy within the past 30 days
  5. History of a stem cell transplant
  6. Known hypersensitivity to any of the components of idursulfase
  7. Female
  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.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01301898

Locations
Korea, Republic of
Samsung Medical Center
Seoul, Irwon-dong, Gangnam-Gu, Korea, Republic of, 135-710
Sponsors and Collaborators
Green Cross Corporation
  More Information

No publications provided by Green Cross Corporation

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Green Cross Corporation
ClinicalTrials.gov Identifier: NCT01301898     History of Changes
Other Study ID Numbers: GC1111_P1/2
Study First Received: February 22, 2011
Last Updated: February 12, 2012
Health Authority: Korea: Food and Drug Administration

Keywords provided by Green Cross Corporation:
Hunter syndrome
MPS II
Idursulfase

Additional relevant MeSH terms:
Syndrome
Mucopolysaccharidoses
Mucopolysaccharidosis II
Disease
Pathologic Processes
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Genetic Diseases, X-Linked
Heredodegenerative Disorders, Nervous System

ClinicalTrials.gov processed this record on October 01, 2014