A Study of DNL310 in Pediatric Participants With Hunter Syndrome
Status:
Recruiting
Trial end date:
2024-03-01
Target enrollment:
Participant gender:
Summary
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 three cohorts:Cohort A will enroll participants with neuronopathic MPS II aged
5 to 10 years; Cohort B will enroll participants with MPS II, either neuronopathic or
non-neuronopathic, aged 2 to 18 years; and Cohort C will enroll participants with
neuronopathic MPS II aged ≥2 and <4 (Cohort C can include nMPS II participants ≥4 if the
participant is a sibling of a participant aged ≥2 and <4).
Participants, whose physicians feel they are deriving benefit, will have the opportunity to
be reconsented into a safety extension for continued evaluation.