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Safety and Efficacy of Hunterase (GC1111)

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Green Cross Corporation
ClinicalTrials.gov Identifier:
NCT01645189
First received: July 16, 2012
Last updated: April 12, 2013
Last verified: April 2013
History of Changes
  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:

Drug Information available for: Idursulfase
U.S. FDA Resources

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
Estimated Study Completion Date: September 2013
Estimated 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
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: GC1111_C_P3b
Study First Received: July 16, 2012
Last Updated: April 12, 2013
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
Mental Retardation
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 May 19, 2013