A Phase 2/3 interventional study of tividenofusp alfa in Mucopolysaccharidosis II, sponsored by Denali Therapeutics Inc.. Enrolling by invitation at 31 sites in 14 countries. Open to participants aged Up to 18 Years. Per ClinicalTrials.gov, last updated 2026-11-09.
Sponsored by Denali Therapeutics Inc. · Phase 2/3, Interventional, and Treatment
This is a multiregional open-label extension (OLE) to assess the safety, tolerability, and efficacy of long-term treatment with tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant intravenous (IV) enzyme replacement therapy (ERT) for Hunter syndrome (MPS II). Participants who complete at least through the Week 49 visit in Study DNLI-E-0002 and do not discontinue study intervention early and participants who complete Study DNLI-E-0007 will be enrolled in this OLE. All participants will receive DNL310 for up to 5 years from the time of entry in this OLE. Participants, site staff, and the Sponsor will remain blinded to the original treatment assignment for participants entering this OLE from Study DNLI-E-0007.
77 studies on the registry are indexed under Mucopolysaccharidosis II; 8 are open to participants now.
This study's planned enrollment of 99 is above the median of 22 across 48 interventional studies indexed under Mucopolysaccharidosis II.
Browse Mucopolysaccharidosis II studies →Denali Therapeutics Inc. is the lead sponsor of 19 studies on the registry; 5 are open to participants now.
Of its 6 completed or terminated interventional studies of FDA-regulated products, 0 (0%) have results posted.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Key Exclusion Criteria:
Participants with nMPS II, aged ≥5 to ≤10 years
Drug: tividenofusp alfa
Participants with nMPS II or nnMPS II, aged ≥1 to ≤18 years
Drug: tividenofusp alfa
Participants with nMPS II, aged \<4 years
Drug: tividenofusp alfa
Participants with nMPS II or nnMPS II, aged ≤18 years with preexisting hepatomegaly who have never taken standard-of-care ERT
Drug: tividenofusp alfa
Participants with nMPS II, aged ≥6 years; participants with nnMPS II, aged \<6 or ≥17 years; or participants with nMPS II, aged ≥1 to ≤18 years, with a history of prior HSCT or gene therapy and have completed at least 48 weeks in Study DNLI-E-0001
Drug: tividenofusp alfa
Participants with nMPS II, aged ≥2 to \<6 years
Drug: tividenofusp alfa
Participants with nnMPS II, aged ≥6 to \<17 years
Drug: tividenofusp alfa
Intravenous repeating dose
Incidence and intensity of treatment-emergent adverse events (TEAEs)
Time frame: 5 years
Clinically significant changes in urine total glycosaminoglycan (GAG) concentrations throughout the treatment period
Time frame: 5 years
Incidence and intensity of infusion-related reactions (IRRs)
The intensity of IRRs will be assessed following each infusion of DNL310 using the categories of Mild, Moderate and Severe. IRRs will be summarized overall as well as stratified by intensity.
Time frame: 5 years
Percentage change from baseline in cerebrospinal fluid (CSF) heparan sulfate (HS) concentration
Time frame: 5 years
Change from baseline in the Vineland-3 Adaptive Behavior Scale
Time frame: 5 years
Change from baseline in the Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III) cognitive raw score
Time frame: 5 years
Change from baseline in distance walked (meters) in the Six-Minute Walk Test (6MWT)
Time frame: 5 years
Percent change from baseline in the sum of urine HS and dermatan sulfate (DS) concentrations
Time frame: 5 years
Liver volume within the normal range (normal vs abnormal) as measured by MRI
Time frame: 5 years
Spleen volume within the normal range (normal vs abnormal) as measured by MRI
Time frame: 5 years
Improvement in the Parent/Caregiver Global Impression of Change (CaGI-C) Overall MPS II
Time frame: 5 years
Plan to share: No
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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Studies of the drug tested here: Tividenofusp alfa
Denali Therapeutics Inc.