The purpose of this study is to evaluate the safety and efficacy of GC1111 (recombinant human iduronate-w-sulfatase) in Hunter Syndrome (Mucopolysaccharidosis II) patients
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Enrollment
31
Samsung Medical Center
Seoul, Irwon-dong, Gangnam-Gu, South Korea
Primary Outcome
measurement of % change of Urine GAG
Time frame: baseline, every 4 weeks
Secondary Outcome
* Measurements of the six-minute-walk test (6-MWT) * Measurements of liver volume * Measurements of heart size and heart function * Measurements of joint range of motion * Measurements of urine GAG levels * Measurements of pulmonary function * Evaluate the safety: vital signs, physical examination, laboratory tests, adverse events, immunogenecity
Time frame: baseline, every 12 weeks
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