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OpenTrials
Completed

NCT Number: NCT01645189

Safety and Efficacy of Hunterase

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.

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Key information

Conditions

Hunter Syndrome Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked Heredodegenerative Disorders, Nervous System Intellectual Disability Lysosomal Storage Diseases Metabolic Diseases Metabolism, Inborn Errors Mucinoses Mucopolysaccharidoses Mucopolysaccharidosis II Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations Nutritional and Metabolic Diseases Skin and Connective Tissue Diseases X-Linked Intellectual Disability

Age range

Up to 5 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 3

Primary location

Samsug Medical Center

Seoul, South Korea

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • 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
  • < 6 years old and male
  • Patients who are able to comply with the study requirements
  • The patient's parent(s), or patient's legal guardian must have given voluntary written consent to participate in the study

Exclusion criteria

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

Treatment and study plan

Hunterase

Biological

once weekly, 0.5mg/kg IV infusion

Primary outcomes

  1. Incidence of adverse events

    Time frame: One year

Secondary outcomes

  1. change of anti-idursulfase-beta antibody status

    Time frame: baseline and one year

  2. Percent Change of Urine GAG

    Time frame: baseline to 53 weeks

Sponsors and collaborators

Lead sponsor

Green Cross Corporation

Industry

Registry information

Official study title

To Evaluate the Safety and Efficacy of Hunterase(Idursulfase-beta) in Hunter Syndrome Patients < 6 Years of Age Receiving Idursulfase Enzyme Replacement Therapy

Acronym: GC1111

Important dates

Study start
2012
Primary completion
2013
Study completion
2013
First posted
Jul 20, 2012
Registry last updated
Jul 8, 2014

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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