A Phase 1/2, Multicenter, Open-Label Study to Determine the Safety, Pharmacokinetics, and Pharmacodynamics of DNL310 in Pediatric Participants with Hunter Syndrome

Hunter Syndrome (Mucopolysaccharidosis Type II [MPS II])

Incidence and severity of treatment-emergent adverse events (TEAEs) throughout the 24-week study period, the safety extension (Week 104), the open-label extension (Week 261), and across the study, Incidence and severity of infusion-related reactions (IRRs) throughout the 24-week study period, the safety extension (Week 104), the open-label extension (Week 261), and across the study, Change from baseline in urine total glycosaminoglycan (GAG) concentration throughout the 24-week study period, the safety extension (Week 104), the open-label extension (Week 261), and across the study, Change from baseline in concomitant medication throughout the 24-week study period, the safety extension (Week 104), the open-label extension (Week 261), and across the study

Percent change from baseline in cerebrospinal fluid (CSF) of heparan sulfate [Time Frame:24 weeks], Participants with improvement in individual disease progression in the Vineland Adaptive Behavior Scale Adaptive Behavior Composite (ABC) score [Time Frame: 49 weeks], Participants with improvement in individual disease progression in the Vineland Adaptive Behavior Scale subdomain scores [Time Frame: 49 weeks], PK parameter: Maximum observed concentration (Cmax) of DNL310 in serum [Time Frame: 24 weeks], PK parameter: Trough concentration (Cmin) of DNL310 in serum [Time Frame: 24 weeks], PK parameter: Time to maximum observed concentration (tmax) of DNL310 in serum [Time Frame: 24 weeks], PK parameter: Area under the concentration-time curve from time zero to the time of last quantifiable concentration (AUClast) of DNL310 in serum [Time Frame: 24 weeks], PK parameter: Area under the concentration-time curve from time zero to infinity (AUC∞) of DNL310 in serum [Time Frame: 24 weeks], PK parameter: Area under the concentration-time curve over a dosing interval (AUCτ) of DNL310 in serum [Time Frame: 24 weeks], PK parameter: Apparent terminal elimination half-life (t½) of DNL310 in serum [Time Frame: 24 weeks], Characterization of immunogenicity of DNL310 in serum, as measured by the incidence of anti-drug antibodies (ADAs) relative to baseline [Time Frame: 24 weeks], Percent change from baseline in urine concentration of heparan sulfate (HS) [Time Frame: 24 weeks], Participants with liver volume in the normal range [Time Frame: 24 weeks and 49 weeks], Percentage change form baseline in liver volume [Time Frame: 24 weeks and 49 weeks]

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