MPS II Immunophenotyping
Recruiting
- Conditions
- MPS IIHunter SyndromeMucopolysaccharidosis 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
Name Time Method Changes in distribution of helper T cells measured by flow cytometry baseline, 1, 3, 6 months Changes in level of T follicular helper cells measured by flow cytometry baseline, 1, 3, 6 months Changes in level of circulation memory B cells measured by flow cytometry baseline, 1, 3, 6 months Changes in the frequency of natural killer cells measured by flow cytometry baseline, 1, 3, 6 months
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (1)
Duke University
🇺🇸Durham, North Carolina, United States