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
Phase 2
Phase 3
Interventional Study
Mucopolysaccharidosis II
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Pivotal Trial
This treatment is in the last trial phase before FDA approval.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 2 – 25
- Sex
- Any
- Trial phase
- Phase 2/3
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 63 (estimated)
- Sponsor
- Denali Therapeutics Inc. · Industry
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About This Trial
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 idursulf…
Trial Locations
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Eligibility Criteria
Key Inclusion Criteria:
* 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…
Contacts