The Long-term Safety Study of Idursulfase-beta in Hunter Syndrome(Mucopolysaccharidosis II) Patients

NCT02044692 | Unknown

• 1 other identifier: GC1111_OS
• Study Type: observational
• Target: 34
• Locations: 1 country
• Sites: 1

Brief Summary

The objective of this study is to evaluate the long term safety and efficacy of once weekly dosing of idurasulfase-beta 0.5mg/kg administered in Hunter Syndrome(Mucopolysaccharidosis II) Patients

Conditions

Hunter Syndrome

Keywords

Hunter syndrome; Mucopolysaccharidosis II; Hunterase

Outcome Measures

Primary Outcomes (1)

• Incidence of adverse event and adverse drug reaction.

  Once a week up to 5years

Secondary Outcomes (1)

• Change of vital sign, physical and clinical examination, anti-idurasulfase-beta antibody status.

  Base line and every three months up to 5years.(exception: clinical examination-> baseline and every six months up to 5years)

Other Outcomes (2)

• Percent change of urine GAG.

  Baseline and every three months up to 5years.

• Percent change of 6 minute walking test.

  Baseline and every 6 months up to 5years.

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Hunter Syndrome(Mucopolysaccharidosis II) Patients

You may qualify if:

• Patients who have a diagnosis of Hunter syndrome(Mucopolysaccharidosis II).
• Patients who are administered idurasulfase-beta or willing to be administered idurasulfase-beta.
• Patient's parent(s), or patient's legal guardian must have given voluntary written consent to participate in the study.

You may not qualify if:

• Patients who have participated in any other blind clinical trials.
• Patient who cannot be tracked about safety.
• Patients who are judged disqualified to participate clinical trials by investigator for other causes.

Sponsors & Collaborators

• Green Cross Corporation: lead

Study Sites (1)

Samsung Medical Center

Seoul, South Korea

RECRUITING

MeSH Terms

Conditions

Mucopolysaccharidosis II

Condition Hierarchy (Ancestors)

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

Study Officials

• Dong-Kyu Jin

  Samsung medical center, Seoul, Republic of Korea

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Dong-Kyu Jin

CONTACT

jindk.jin@samsung.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 19, 2014
• First Posted: January 24, 2014
• Study Start: January 1, 2014
• Primary Completion: January 1, 2020
• Study Completion: January 1, 2020
• Last Updated: June 20, 2014

Record last verified: 2014-06

Locations

KR (1)