Summary

Eligibility
for people ages 2-16 (full criteria)
Location
at Oakland, California and other locations
Dates
study started
estimated completion
Principal Investigator
by Paul Harmatz, MD

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 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 DNL310 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
  • UNC Children's Research Institute accepting new patients
    Chapel Hill North Carolina 27514 United States

Lead Scientist at UCSF

  • Paul Harmatz, MD
    Professor, Pediatrics, School of Medicine. Authored (or co-authored) 203 research publications

Details

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