MedTrace Logo

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)

NCT05371613 · Status: RECRUITING · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 63

Last updated 2025-08-05

No results posted yet for this study

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

DRUG

tividenofusp alfa

Intravenous repeating dose

DRUG

idursulfase

Intravenous repeating dose

Sponsors & Collaborators

  • Denali Therapeutics Inc.

    lead INDUSTRY

Principal Investigators

  • Jose Alcantara Rodriguez, PharmD · Denali Therapeutics Inc.

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
PARALLEL

Eligibility

Min Age
2 Years
Max Age
25 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-07-21
Primary Completion
2027-12-31
Completion
2027-12-31
FDA Drug
Yes

Countries

  • United States
  • Argentina
  • Australia
  • Belgium
  • Brazil
  • Canada
  • Czechia
  • France
  • Germany
  • Italy
  • Netherlands
  • Spain
  • Sweden
  • Turkey (Türkiye)
  • United Kingdom

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05371613 on ClinicalTrials.gov