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

Hunter Syndrome Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT02044692
• Other study ID #: GC1111_OS
• Status: Recruiting
• Phase: N/A
• First received: January 19, 2014
• Last updated: June 19, 2014
• Start date: January 2014
• Est. completion date: January 2020

Study information

• Verified date: June 2014
• Source: Green Cross Corporation
• Contact: Dong-Kyu Jin
• Email: jindk.jin[@]samsung.com
• Is FDA regulated: No
• Health authority: Korea: Ministry of Food and Drug Safety
• Study type: Observational

Clinical Trial 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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 34
• Est. completion date: January 2020
• Est. primary completion date: January 2020
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• 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.

Exclusion Criteria:

• 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.

Study Design

Observational Model: Cohort, Time Perspective: Prospective

Related Conditions & MeSH terms

• Hunter Syndrome
• Mucopolysaccharidoses
• Mucopolysaccharidosis II
• Syndrome

Locations

Country	Name	City	State
Korea, Republic of	Samsung Medical Center	Seoul

Sponsors (1)

Lead Sponsor	Collaborator
Green Cross Corporation

Country where clinical trial is conducted

Korea, Republic of, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Percent change of urine GAG.		Baseline and every three months up to 5years.	No
Other	Percent change of 6 minute walking test.		Baseline and every 6 months up to 5years.	No
Primary	Incidence of adverse event and adverse drug reaction.		Once a week up to 5years	Yes
Secondary	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)	Yes