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)

A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic or Non-Neuronopathic Mucopolysaccharidosis Type II

• Status: Recruiting
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 63 (estimated)
• Sponsor: Denali Therapeutics Inc. · Industry
• Sex: All
• Age: 2 Years – 25 Years
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• Mucopolysaccharidosis II

Interventions

Type	Name	Description
DRUG	tividenofusp alfa	Intravenous repeating dose
DRUG	idursulfase	Intravenous repeating dose

Timeline

• Start date: 2022-07-21
• Primary completion: 2027-12-01
• Completion: 2027-12-01
• First posted: 2022-05-12
• Last updated: 2025-08-05

Locations

32 sites across 15 countries: United States, Argentina, Australia, Belgium, Brazil, Canada, Czechia, France, Germany, Italy, Netherlands, Spain, Sweden, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study