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)
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Source updated2026-09-09Last update listed in the registry record
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Main study measure wording changed
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− Before
Measure: Percentchange from baseline in cerebrospinal fluid (CSF) heparan sulfate (HS) concentration (Cohort A)
Measure: Change from baseline in the Vineland Adaptive Behavior Scale, Third Edition (Vineland-3)(Cohort A)
+ After
Measure: Ratio to baseline in cerebrospinal fluid (CSF) heparan sulfate (HS) concentration (Cohort A)
Measure: Change from baseline in the Vineland Adaptive Behavior Scale, Third Edition (Vineland-3)(Cohort A)
Trial