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MPS II Immunophenotyping

Recruiting
Conditions
MPS II
Hunter Syndrome
Mucopolysaccharidosis II
Registration Number
NCT04976231
Lead Sponsor
Duke University
Brief Summary

The purpose of this study is to investigate how participant's body's immune system responds to idursulfase, an enzyme replacement therapy (ERT) and find out which types of immune cells are involved in causing untoward responses to the ERT so that the investigators can relate the level of immune response to the treatment.

Detailed Description

Not available

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
25
Inclusion Criteria
  • Subject has a confirmed diagnosis of Mucopolysaccharidosis type II (MPS II) or Hunter Syndrome based on enzyme activity and variant analysis.
  • Subject who plan to receive or have received enzyme replacement therapy with idursulfase.
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Exclusion Criteria
  • None
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Study & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Changes in distribution of helper T cells measured by flow cytometrybaseline, 1, 3, 6 months
Changes in level of T follicular helper cells measured by flow cytometrybaseline, 1, 3, 6 months
Changes in level of circulation memory B cells measured by flow cytometrybaseline, 1, 3, 6 months
Changes in the frequency of natural killer cells measured by flow cytometrybaseline, 1, 3, 6 months
Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (1)

Duke University

🇺🇸

Durham, North Carolina, United States

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