A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome

a study on Mucopolysaccharidosis Hunter Syndrome

Summary

Eligibility
for people ages up to 30 years (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 four-part prospective, multicenter, multiregional observational study of patients with mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, to assess biomarkers potentially related to disease severity and/or treatment response and prospectively assess the progression of disease in participants with MPS II who are aged 2 through 10 years (Part 1), 2 through 30 years (Part 2), < 8 years (Part 3), and 6 to < 17 years (Part 4) at the time of enrollment.

Official Title

A Prospective, Longitudinal Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome

Details

This clinical trial information was submitted voluntarily under the applicable law and, therefore, certain submission deadlines may not apply. (That is, clinical trial information for this applicable clinical trial was submitted under section 402(j)(4)(A) of the Public Health Service Act and 42 CFR 11.60 and is not subject to the deadlines established by sections 402(j)(2) and (3) of the Public Health Service Act or 42 CFR 11.24 and 11.44.).

Keywords

Mucopolysaccharidosis II MPS-II Hunter Syndrome Lysosomal Storage Disease nMPS II nnMPS II Mucopolysaccharidoses

Eligibility

You can join if…

Open to people ages up to 30 years

(Part 1):

  • Participants aged 2 through 10 years
  • nMPS II subgroup: participants with a development quotient (DQ) <85 and/or a decline of at least 7.5 points in DQ, assessed at least 6 months apart, or with the same genetic mutation as a blood relative with confirmed nMPS II

Key Inclusion Criteria (Part 2):

  • Participants aged 2 through 30 years
  • nMPS II subgroup: patients with an age-adjusted DQ <85 and/or a decline of 10 points or more in DQ in the previous 6 months or more, or with the same genetic mutation as a blood relative with confirmed nMPS II
  • Scheduled to undergo general anesthesia or CSF sampling for non-study-related medical reasons and parent(s)/legally authorized representative consent to donate CSF for research purposes during that procedure, or an adult patient is able to provide consent and agrees to participation in the study for CSF collection/donation

Key Inclusion Criteria (Part 3):

  • nMPS II participants aged <8 years

Key Inclusion Criteria (Part 4):

  • nnMPS II participants aged 6 to 17 years

You CAN'T join if...

(All Parts):

  • Have unstable medical condition that would make participation in the study unsafe or would interfere with necessary medical care
  • Have received any central nervous system (CNS)-targeted MPS II investigational therapy within the previous 6 months

Locations

  • UCSF Benioff Children's Hospital accepting new patients
    Oakland California 94609 United States
  • UPMC | Children's Hospital of Pittsburgh accepting new patients
    Pittsburgh Pennsylvania 15224 United States

Lead Scientist at UCSF

  • Paul Harmatz, MD
    Professor, Pediatrics, School of Medicine. Authored (or co-authored) 205 research publications

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Denali Therapeutics Inc.
ID
NCT04007536
Study Type
Observational
Participants
Expecting 37 study participants
Last Updated
Contact us about this trial