This is a multicenter, multiregional, open-label study to assess the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of DNL310, an investigational central nervous system (CNS)-penetrant enzyme replacement therapy (ERT), designed to treat both the peripheral and CNS manifestations of Mucopolysaccharidosis type II (MPS II; Hunter syndrome). The study has two staggered cohorts: the first will enroll subjects with neuronopathic MPS II aged 5 to 10 years; and the second will enroll subjects with MPS II, either neuronopathic or non-neuronopathic, aged 2 to 18 years. Subjects, whose physicians feel they are deriving benefit, will have the opportunity to be reconsented into a safety extension for continued evaluation, if implemented.
A Phase 1/2, Multicenter, Open-Label Study to Determine the Safety, Pharmacokinetics, and Pharmacodynamics of DNL310 in Pediatric Subjects With Hunter Syndrome