A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome
Running, not enrolling · Phase 1/Phase 2
Conditions studied: Mucopolysaccharidosis II
In brief
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.
Key facts
- Study ID
- NCT04251026
- Run by
- Denali Therapeutics Inc.
- People needed
- 47
- Starts
- 2020-07-16
- Expected to finish
- 2031-02-01
- Last updated by the study team
- 2025-08-07
Who can join
Age: any, up to 18. Sex: male. Healthy volunteers: not accepted.
Where it is running
- UCSF Benioff Children's Hospital — Oakland, California, United States
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
- UNC Children's Research Institute — Chapel Hill, North Carolina, United States
- UPMC | Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
- McGill University Health Centre - Royal Victoria Hospital — Montreal, Quebec, Canada
- Erasmus Medical Center — Rotterdam, South Holland, Netherlands
- St Mary's Hospital, Manchester Academic Health Science Centre — Manchester, United Kingdom
Full record on ClinicalTrials.gov
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