To evaluate the Safety and Efficacy of Hunterase in Pediatric Hunter Syndrome
- Conditions
- Congenital malformations, deformations and chromosomal abnormalities
- Registration Number
- KCT0000567
- Lead Sponsor
- Green Cross
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Male
- Target Recruitment
- 5
1) The patient has a diagnosis of Hunter syndrome based upon biochemical criteria:
? as measured in plasma, leukocytes, or fibroblasts, Iduronate-2-sulfatase enzyme activity of = 10 % of the lower limit of the normal range AND
? at least one of the following items
- a normal enzyme activity level of one other sulfatase
- MPS ll is confirmed by gene analysis
- subjects who are showing signs/clinical symptoms of MPS ll
2) < 6 years old and male
3) Patients who are able to comply with the study requirements
4) The patient's parent(s), or patient's legal guardian must have given voluntary written consent to participate in the study
1) The patient has had a tracheostomy
2) The patient has known severe hypersensitivity or shock to any of the components of idursulfase
3) The patient has received treatment with another investigational therapy within 30 days prior to enrollment
4) History of a stem cell transplant
5) The patient has known severe hypersensitivity or shock to any of the components of test drug(excipient etc)
Study & Design
- Study Type
- Interventional Study
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Incidence of adverse events
- Secondary Outcome Measures
Name Time Method Change of laboratory test;Change of physical examination;change of anti-idursulfase-beta antibody status;Percent Change of Urine GAG;Change of ecocardiogram;Change of vital sign