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

Study Timeline

• Study First Submitted: 2021-07-19
• Study First Submitted QC: 2021-07-19
• Study First Posted: 2021-07-26
• Study Start: 2022-04-01
• Status Verified: 2024-04
• Last Update Submitted: 2024-04-19
• Last Update Posted: 2024-04-22
• Primary Completion: 2025-04
• Study Completion: 2025-04

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.

Exclusion Criteria

• None

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

Trial Locations

Locations (1)

Duke University; 🇺🇸 Durham, North Carolina, United States