A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

a study on Hunter Syndrome Mucopolysaccharidosis

Summary

Eligibility
for people ages 2-16 (full criteria)
Location
at UCSF
Dates
study started
completion around
Principal Investigator
by Paul Harmatz, MD (ucsf)
Headshot of Paul Harmatz
Paul Harmatz

Description

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.

Official Title

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

Keywords

Mucopolysaccharidosis II, Hunter Syndrome, MPS II, nMPS II, nnMPS II, Mucopolysaccharidoses, tividenofusp alfa, idursulfase

Eligibility

You can join if…

Open to people ages 2-16

  • Participants aged ≥2 to <6 years (Cohort A) or ≥6 to <17 years (Cohort B)
  • Confirmed diagnosis of MPS II (for Cohort A, nMPS II; for Cohort B, nnMPS II)
  • Be on maintenance enzyme replacement therapy (ERT) and have tolerated idursulfase for a minimum of 4 months prior to screening

You CAN'T join if...

  • 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 imagings (MRIs)
  • 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

Locations

  • UCSF Benioff Children's Hospital Oakland accepting new patients
    Oakland California 94609 United States
  • University of Utah, PPDS accepting new patients
    Salt Lake City Utah 84132 United States

Lead Scientist at University of California Health

  • Paul Harmatz, MD (ucsf)
    Dr. Paul Harmatz is a gastroenterologist who specializes in mucopolysaccharidoses (MPS) and other lysosomal storage diseases (genetic disorders in which a lack of certain enzymes results in progressive damage to cells and organ systems). He leads a team of specialists who diagnose and care for patients with these rare diseases, offering therapies such as weekly enzyme infusion.

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Denali Therapeutics Inc.
Links
Engage Hunter Website
ID
NCT05371613
Phase
Phase 2/3 research study
Study Type
Interventional
Participants
Expecting 54 study participants
Last Updated