A Study of Potential Treatment-Responsive Biomarkers in Hunter Syndrome

  • End date
    Feb 8, 2024
  • participants needed
  • sponsor
    Denali Therapeutics Inc.
Updated on 12 September 2021
general anesthesia
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This is an observational, Pre-phase 1 study of biomarkers in patients with mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome.


This is a 2-part, prospective, multicenter, observational study of patients with MPS II to characterize disease-related biomarkers in urine, blood and cerebrospinal fluid (CSF). Part 1 will include MPS II patients who are 2 -10 years and Part 2 will include MPS II patients who are 2 -30 years old at the time of enrollment. All patients may be on approved therapies for MPS II; no investigational treatment will be administered.

Condition Hunter's Syndrome, Hunter Syndrome (MPS II), hunter syndrome
Treatment No intervention
Clinical Study IdentifierNCT04007536
SponsorDenali Therapeutics Inc.
Last Modified on12 September 2021


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