MPS II Immunophenotyping

Immunophenotyping of Patients With MPS II Treated With Enzyme Replacement Therapy

Status

Terminated

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT04976231

Enrollment

Registered

2021-07-26

Start date

2022-04-01

Completion date

2024-04-26

Last updated

2024-09-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hunter Syndrome, MPS II, Mucopolysaccharidosis II

Keywords

MPS II, Mucopolysaccharidosis II, Hunter Syndrome

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.

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame
Changes in the frequency of natural killer 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 level of T follicular helper cells measured by flow cytometry	baseline, 1, 3, 6 months
Changes in distribution of helper T cells measured by flow cytometry	baseline, 1, 3, 6 months

Countries

United States

Outcome results