clinical trial · NCT04251026
A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome
Mucopolysaccharidosis IItividenofusp alfa
brief summary
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.
started
Jul 16, 2020
primary completion
Feb 1, 2031
completion
Feb 1, 2031
last updated
Aug 7, 2025
official title
A Phase 1/2, Multicenter, Open-Label Study to Determine the Safety, Pharmacokinetics, and Pharmacodynamics of DNL310 in Pediatric Participants With Hunter Syndrome
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol