Hunter Syndrome
Conditions
Keywords
Hunter syndrome, idursulfase-beta, Hunterase, GC1111
Brief summary
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.
Interventions
BIOLOGICALHunterase
once weekly, 0.5mg/kg IV infusion
Sponsors
Green Cross Corporation
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
No minimum to 5 Years
Healthy volunteers
No
Inclusion criteria
1. 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 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
Exclusion criteria
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)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of adverse events | One year |
Secondary
| Measure | Time frame |
|---|---|
| change of anti-idursulfase-beta antibody status | baseline and one year |
| Percent Change of Urine GAG | baseline to 53 weeks |
Countries
South Korea
Outcome results
None listed