A Study of Potential Treatment-Responsive Biomarkers in Hunter Syndrome

  • STATUS
    Recruiting
  • End date
    Feb 8, 2024
  • participants needed
    28
  • sponsor
    Denali Therapeutics Inc.
Updated on 12 September 2021
anesthesia
general anesthesia
mucopolysaccharidosis
ids gene

Summary

This is an observational, Pre-phase 1 study of biomarkers in patients with mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome.

Description

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.

Details
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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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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