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Completed

NCT Number: NCT01301898

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

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

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Key information

Conditions

Mucopolysaccharidosis II Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Death Death, Sudden Genetic Diseases, Inborn Genetic Diseases, X-Linked Heredodegenerative Disorders, Nervous System Infant Death Intellectual Disability Lysosomal Storage Diseases Metabolic Diseases Metabolism, Inborn Errors Mucinoses Mucopolysaccharidoses Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations Nutritional and Metabolic Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Skin and Connective Tissue Diseases Sudden Infant Death X-Linked Intellectual Disability

Age range

6 year–35 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Samsung Medical Center

Seoul, Irwon-dong, Gangnam-Gu, 135-710, South Korea

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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

Exclusion criteria

  • History of a tracheostomy or a bone marrow transplant
  • Known hypersensitivity to idursulfase
  • Known shock to idursulfase
  • History of receiving treatment with another investigational therapy within the past 30 days
  • History of a stem cell transplant
  • Known hypersensitivity to any of the components of idursulfase
  • Female

Treatment and study plan

GC1111_0.5mg/kg

Drug

GC1111_1.0mg/kg

Drug

Elaprase_0.5mg/kg

Drug

Primary outcomes

  1. Primary Outcome

    Time frame: baseline, every 4 weeks

    measurement of % change of Urine GAG

Secondary outcomes

  1. Secondary Outcome

    Time frame: baseline, every 12 weeks

    • 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

Sponsors and collaborators

Lead sponsor

Green Cross Corporation

Industry

Registry information

Official study 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

Important dates

Study start
2010
Primary completion
2011
Study completion
2011
First posted
Feb 23, 2011
Registry last updated
Feb 14, 2012

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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