A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome

This is a multicenter, multiregional, open-label study to assess the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of tividenofusp alfa (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). Participants, whose physicians feel they are deriving benefit, will have the opportunity to be reconsented into a safety extension and then an open-label extension for continued evaluation.

NCT Number	NCT04251026
Lead Sponsor	Denali Therapeutics Inc.
Conditions	Mucopolysaccharidosis II
Enrollment	47 participants
Start Date	2020-07-16
Primary Completion	2031-02 (estimated)
Study Completion	2031-02 (estimated)
Updated on ClinicalTrials.gov	2025-08-07