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Study of Tividenofusp Alfa (DNL310) for Mucopolysaccharidosis Type II

This study is testing an investigational medicine called tividenofusp alfa (DNL310) against a standard treatment, idursulfase, for children and young adults with Mucopolysaccharidosis Type II (MPS II). MPS II is a rare genetic condition. Researchers want to see how safe and effective tividenofusp alfa is. They will measure changes in a substance called heparan sulfate (HS) in the brain fluid after 24 weeks, and look at adaptive behaviors (like communication and daily living skills) after 96 weeks. You might be able to join if you are between 2 and 25 years old and have been diagnosed with MPS II. The study is currently recruiting about 63 participants, but its overall status is unclear.

Study design
This is a Phase 2/3 study that will randomly assign participants to receive either tividenofusp alfa or idursulfase. It is a double-blind study, meaning neither you nor your doctor will know which treatment you are receiving.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
The study will measure some effects for up to 96 weeks (about 1 year and 10 months).

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT05371613

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)

Recruiting
PHASE2Ages 2–25InterventionalTreatment
Denali Therapeutics Inc.
~63 participants
Updated 2025-08-05 on ClinicalTrials.gov
What's tested:tividenofusp alfaidursulfase

At a glance

Recruiting sites
30 of 32 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Percent change from baseline in cerebrospinal fluid (CSF) heparan sulfate (HS) concentration (Cohort A)
Measured over 24 weeks
+1 more outcome measured

Conditions

Mucopolysaccharidosis II

Explore related trials

Hunter Syndrome

Where it's being run

32 sites across 28 states
United Kingdom3
Brazil2
Turkey (Türkiye)2
California1
Illinois1
New Jersey1
North Carolina1
Ohio1

Study leadership

  • Jose Alcantara Rodriguez, PharmD · STUDY_DIRECTOR · Denali Therapeutics Inc.

Who to contact

Clinical Trials at Denali Therapeutics
[email protected]
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Participants aged ≥2 to \<6 years (Cohort A) or ≥6 to \<26 years (Cohort B)
Confirmed diagnosis of MPS II (for Cohort A, nMPS II; for Cohort B, nnMPS II)
Have no history of treatment with enzyme replacement therapy (ERT) OR not have received continuous ERT for 4 months prior to screening OR be on maintenance ERT and have tolerated idursulfase for a minimum of 4 months prior to screening

Exclusion

Have a documented mutation of other genes or genetic diagnosis accounting for developmental delay
Previously received an iduronate 2-sulfatase (IDS) gene therapy or stem cell therapy
Received any CNS-targeted MPS ERT within 6 months prior to screening
Have a contraindication for lumbar punctures and/or magnetic resonance imaging (MRI)
Participated in any other investigational drug study or used an investigational drug within 60 days prior to screening or intend to receive another investigational drug during the study

What this trial measures

  • Percent change from baseline in cerebrospinal fluid (CSF) heparan sulfate (HS) concentration (Cohort A)24 weeks
  • Change from baseline in the Vineland Adaptive Behavior Scale, Third Edition (Vineland-3)(Cohort A)96 weeks