Safety and Efficacy of Hunterase (GC1111)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Green Cross Corporation
ClinicalTrials.gov Identifier:
NCT01645189
First received: July 16, 2012
Last updated: July 7, 2014
Last verified: July 2014
  Purpose

The objective of this study is to determine the safety and efficacy of once weekly dosing of idursulfase-beta 0.5mg/kg administered by intravenous(IV) infusion for Hunter syndrome patients < 6 years old.


Condition Intervention Phase
Hunter Syndrome
Biological: Hunterase
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: To Evaluate the Safety and Efficacy of Hunterase(Idursulfase-beta) in Hunter Syndrome Patients < 6 Years of Age Receiving Idursulfase Enzyme Replacement Therapy

Resource links provided by NLM:


Further study details as provided by Green Cross Corporation:

Primary Outcome Measures:
  • Incidence of adverse events [ Time Frame: One year ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • change of anti-idursulfase-beta antibody status [ Time Frame: baseline and one year ] [ Designated as safety issue: Yes ]
  • Percent Change of Urine GAG [ Time Frame: baseline to 53 weeks ] [ Designated as safety issue: No ]

Enrollment: 6
Study Start Date: July 2012
Study Completion Date: September 2013
Primary Completion Date: September 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Test drug
Idursulfase-beta
Biological: Hunterase
once weekly, 0.5mg/kg IV infusion

  Eligibility

Ages Eligible for Study:   up to 5 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. The patient has a diagnosis of Hunter syndrome based upon biochemical criteria:

    • as measured in plasma, leukocytes, or fibroblasts,

      • a deficiency in iduronate-2-sulfatase (I2S) enzyme activity of ≤ 10 % of the lower limit of the normal range
    • That corresponds to one or more of the following:

      • a normal enzyme activity level of one other sulfatase
      • Confirmed as MPS2 by genetic test results
      • shows clinical symptoms/ visible signs of MPS2
  2. < 6 years old and male
  3. Patients who are able to comply with the study requirements
  4. The patient's parent(s), or patient's legal guardian must have given voluntary written consent to participate in the study

Exclusion Criteria:

  1. The patient has had a tracheostomy
  2. The patient has known severe hypersensitivity or shock to any of the components of idursulfase
  3. The patient has received treatment with another investigational therapy within 30 days prior to enrollment
  4. History of a stem cell transplant
  5. The patient has known severe hypersensitivity or shock to any of the components of test drug(excipient etc)
  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: NCT01645189

Locations
Korea, Republic of
Samsug Medical Center
Seoul, Korea, Republic of
Sponsors and Collaborators
Green Cross Corporation
Investigators
Principal Investigator: Dong-Kyu Jin Samsung medical center, Seoul, Republic of Korea
  More Information

No publications provided

Responsible Party: Green Cross Corporation
ClinicalTrials.gov Identifier: NCT01645189     History of Changes
Other Study ID Numbers: GC1111C
Study First Received: July 16, 2012
Last Updated: July 7, 2014
Health Authority: Korea: Food and Drug Administration

Keywords provided by Green Cross Corporation:
Hunter syndrome
idursulfase-beta
Hunterase
GC1111

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

ClinicalTrials.gov processed this record on August 01, 2014