A Phase II open-label, parallel group, 2 sites (Brazil), designed to evaluate the long term safety and efficacy of study drug for the treatment of the MPS II.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
19
IV infusion (lyophilized powder)
Grupo de Pesquisa Clínica em Genética Médica - HCPA
Porto Alegre, Brazil
Igeim - Unifesp
São Paulo, Brazil
Number of participants with Adverse Events
Time frame: From screening up to the end of study, up to approximately 5 years
Urinary heparan sulfate concentrations
Time frame: through study completion, an average of 52 weeks, up to approximately 5 years
Urinary dermatan sulfate concentrations
Time frame: through study completion, an average of 52 weeks, up to approximately 5 years
Serum heparan sulfate concentrations
Time frame: through study completion, an average of 52 weeks, up to approximately 5 years
Serum dermatan sulfate concentrations
Time frame: through study completion, an average of 52 weeks, up to approximately 5 years
Liver and spleen volumes (MRI)
Time frame: through study completion, an average of 52 weeks, up to approximately 5 years
Echocardiography
Time frame: through study completion, an average of 52 weeks, up to approximately 5 years
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