Hunter Syndrome Clinical Trial
— GC1111Official title:
To Evaluate the Safety and Efficacy of Hunterase(Idursulfase-beta) in Hunter Syndrome Patients < 6 Years of Age Receiving Idursulfase Enzyme Replacement Therapy
Verified date | July 2014 |
Source | Green Cross Corporation |
Contact | n/a |
Is FDA regulated | No |
Health authority | Korea: Food and Drug Administration |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 6 |
Est. completion date | September 2013 |
Est. primary completion date | September 2013 |
Accepts healthy volunteers | No |
Gender | Male |
Age group | N/A to 5 Years |
Eligibility |
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) |
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
Korea, Republic of | Samsug Medical Center | Seoul |
Lead Sponsor | Collaborator |
---|---|
Green Cross Corporation |
Korea, Republic of,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Incidence of adverse events | One year | Yes | |
Secondary | change of anti-idursulfase-beta antibody status | baseline and one year | Yes | |
Secondary | Percent Change of Urine GAG | baseline to 53 weeks | No |
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