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Completed

NCT Number: NCT07344376

An Extension Study to Assess the Long-term Safety and Efficacy of Hunterase (Idursulfase Beta)

The objective of this study is to assess the long-term safety and efficacy of Hunterase for approximately 1 year in subjects who completed the end of study (EOS) visit (Visit 54) tests in the Phase 3 Hunterase study and patients who received Hunterase for more than 6 months

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Key information

Conditions

MPS II Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Death Death, Sudden GC1111 Genetic Diseases, Inborn Genetic Diseases, X-Linked Heredodegenerative Disorders, Nervous System Hunter Syndrome Hunter Syndrome (MPS II) Hunterase Infant Death 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 Pathologic Processes Pathological Conditions, Signs and Symptoms Skin and Connective Tissue Diseases Sudden Infant Death X-Linked Intellectual Disability

Sex eligibility

Male

Study type

Interventional

Phase

Phase 3

Primary location

Samsung 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

  • Subject diagnosed with Hunter syndrome (MPS II) who completed activities at the EOS visit (Visit 54) in the GC1111_P3 study
  • (Subject who did not participate in the GC1111_P3 study) Subject diagnosed with Hunter syndrome (MPS II) who received Hunterase for more than 6 months
  • (Subjects who did not participate in the GC1111_P3 study) Males aged ≥ 5
  • Informed consent form voluntarily signed by the subject or by a legally acceptable representative
  • Subject who agrees to use contraception

Exclusion criteria

  • Subject with hypersensitivity to any of the ingredients of the investigational product
  • Subject impossible to perform follow-up observation of the safety
  • Subject who received the treatment with another investigational product within 14 days prior to the start of study drug
  • Subject who plans to be treated with another investigational product during the study period
  • Subject who has history of tracheostomy, bone marrow transplant, or cord blood transplant
  • Any other inappropriate conditions for study participation at the investigator's discretion

Treatment and study plan

Hunterase

Drug

0.5mg/kg weekly IV administration

Other names: GC1111

Primary outcomes

  1. Laboratory tests (CBC, Chemistry, Urinanalysis)

    Time frame: Every visit until 12months

    Abnormality of Laboratory tests results

  2. Physical examination

    Time frame: Every visit until 12months

    Abnormality of physical examination

  3. Adverse Event

    Time frame: Every visit until 12months

    occurence, grade

  4. Electrocardiography

    Time frame: Every 6 months until 12months

    Abnormality of ECG results

Secondary outcomes

  1. 6-minute walk test

    Time frame: Every 4months until 12months

    Mean, Change and percent change of 6-minute walk test

  2. Urine GAG (Heparan sulfate, Dermatan sulfate)

    Time frame: Every visit until 12months

    Mean, Change and percent change of Urine HS/DS

Sponsors and collaborators

Lead sponsor

GC Biopharma Corp

Industry

Collaborators

  • Samsung Medical Center

Registry information

Official study title

An Extension Study to Assess the Long-term Safety and Efficacy of Hunterase (Idursulfase Beta) in Patients With Hunter Syndrome

Important dates

Study start
2018
Primary completion
2022
Study completion
2022
First posted
Jan 15, 2026
Registry last updated
Jan 15, 2026

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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