TerminatedNot Applicable

MPS II Immunophenotyping

Stopped: Low Enrollment

United States6 participantsStarted 2022-04-01

Plain-language 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.

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

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

Timeframe: baseline, 1, 3, 6 months

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

Timeframe: baseline, 1, 3, 6 months

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

Timeframe: baseline, 1, 3, 6 months

Changes in distribution of helper T cells measured by flow cytometry

Timeframe: baseline, 1, 3, 6 months

Trial details

NCT IDNCT04976231

SponsorDuke University

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2024-04-26

View on ClinicalTrials.gov More MPS II trials

Plain-language summary

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

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

Timeframe: baseline, 1, 3, 6 months

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

Timeframe: baseline, 1, 3, 6 months

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

Timeframe: baseline, 1, 3, 6 months

Changes in distribution of helper T cells measured by flow cytometry

Timeframe: baseline, 1, 3, 6 months