MPS II Immunophenotyping

Duke University6 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

MPS II Mucopolysaccharidosis II Hunter Syndrome

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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. Exclusion Criteria: * None

Locations (1)

Duke University

Durham, North Carolina, United States

Outcomes

Primary Outcomes

Changes in the frequency of natural killer cells measured by flow cytometry

Time frame: baseline, 1, 3, 6 months

Changes in level of circulation memory B cells measured by flow cytometry

Time frame: baseline, 1, 3, 6 months

Changes in level of T follicular helper cells measured by flow cytometry

Time frame: baseline, 1, 3, 6 months

Changes in distribution of helper T cells measured by flow cytometry

Time frame: baseline, 1, 3, 6 months

Data from ClinicalTrials.gov

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