• Home
  • Study Details
By physician referral or invitation only

Denali Interventional Study

This is a multicenter, open-label study to study the safety and characteristics of DNL310, an investigational drug designed to treat both the body and brain manifestations of Mucopolysaccharidosis type II (MPS II; Hunter syndrome). The plan is for each participant to start at a low dose and increase over time until a final dose is achieved. Blood, urine and CSF samples will be taken, and tests and assessments will be done to see how your child is doing in the study.

Age & Gender

  • 2 years ~ 18 years
  • Male

Contact the Team

Location

Thank you for your interest, but this study is recruiting by invitation only.

United States (Nationwide)

Additional Study Information

Principal Investigator

Joseph Muenzer
Pediatrics - Genetics and Metabolism

Study Type

Clinical or Medical
Interventional

Study Topics

Genetics and Genetic Disorders
Rare Diseases

IRB Number

20-1303

ClinicalTrials.gov

NCT04251026

Research for Me logo

Copyright © 2013-2022 The NC TraCS Institute, the integrated home of the NIH Clinical and Translational Science Awards (CTSA) Program at UNC-CH.  This website is made possible by CTSA Grant UL1TR002489 and the National Center for Advancing Translational Sciences.

Questions?

  • This email address is being protected from spambots. You need JavaScript enabled to view it.
logo for the North Carolina Translational and Clinical Sciences Institute
logo for UNC Health
logo for UNC School of Medicine
logo for UNC Research