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Study identifier: NCT05371613 Synced from ClinicalTrials.gov · July 29, 2026
● Recruiting

A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

Condition: Mucopolysaccharidosis II  ·  Sponsor: Denali Therapeutics Inc.

PhasePhase 2/Phase 3
Planned participants63
Who can joinAll sexes, 2 Years to 25 Years
Healthy volunteersNo

About this study

This is a Phase 2/3, multiregional, two-arm, double-blind, randomized, active (standard-of-care)-controlled study of the efficacy and safety of tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant enzyme-replacement therapy (ERT) for mucopolysaccharidosis type II (MPS II). Participants may also qualify to enter an open-label treatment phase with DNL310 or idursulfase based on pre-specified criteria.

This description comes directly from the study's public registry record.

Talk to the study team

Clinical Trials at Denali Therapeutics  ·  clinical-trials@dnli.com

Always discuss trial participation with your own doctor first.

Locations (32)

UCSF Benioff Children's Hospital OaklandOakland, California, United StatesRecruiting
Ann and Robert H Lurie Children's Hospital of ChicagoChicago, Illinois, United StatesRecruiting
Hackensack University Medical CenterHackensack, New Jersey, United StatesRecruiting
UNC Children's Research InstituteChapel Hill, North Carolina, United StatesRecruiting
Cincinnati Children's Hospital Medical CenterCincinnati, Ohio, United StatesRecruiting
Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania, United StatesRecruiting
The University of Texas Medical School at HoustonHouston, Texas, United StatesRecruiting
University of Utah, PPDSSalt Lake City, Utah, United StatesRecruiting
Sanatorio Mater DeiBuenos Aires, ArgentinaRecruiting
Women's and Children's HospitalNorth Adelaide, AustraliaWithdrawn
UZ AntwerpenAntwerp, Antwerpen, BelgiumRecruiting
Universitair Ziekenhuis BrusselJette, Brussels Capital, BelgiumRecruiting

+ 20 more locations — full list on the registry record.

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Source record: clinicaltrials.gov/study/NCT05371613