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MPS II Immunophenotyping

Sponsor
Duke University (Other)
Overall Status
Recruiting
CT.gov ID
NCT04976231
Collaborator
(none)
25
Enrollment
1
Location
16
Anticipated Duration (Months)
1.6
Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase

    Study Design

    Study Type:
    Observational
    Anticipated Enrollment :
    25 participants
    Observational Model:
    Cohort
    Time Perspective:
    Prospective
    Official Title:
    Immunophenotyping of Patients With MPS II Treated With Enzyme Replacement Therapy
    Actual Study Start Date :
    Apr 1, 2022
    Anticipated Primary Completion Date :
    Jul 31, 2023
    Anticipated Study Completion Date :
    Jul 31, 2023

    Arms and Interventions

    Arm Intervention/Treatment
    Treatment Naive

    subjects who are naïve to ERT and start treatment with idursulfase
    Treatment less than 3 years

    Subjects who have received ERT for <3 years
    Treatment over 3 years

    Subjects who have received ERT for 3 or more years

    Outcome Measures

    Primary Outcome Measures

    1. Changes in the frequency of natural killer cells measured by flow cytometry [baseline, 1, 3, 6 months]

    2. Changes in level of circulation memory B cells measured by flow cytometry [baseline, 1, 3, 6 months]

    3. Changes in level of T follicular helper cells measured by flow cytometry [baseline, 1, 3, 6 months]

    4. Changes in distribution of helper T cells measured by flow cytometry [baseline, 1, 3, 6 months]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    N/A and Older
    Sexes Eligible for Study:
    All
    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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Duke University Durham North Carolina United States 27710

    Sponsors and Collaborators

    • Duke University

    Investigators

    • Principal Investigator: Priya Kishnani, MD, Duke University

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Duke University
    ClinicalTrials.gov Identifier:
    NCT04976231
    Other Study ID Numbers:
    • Pro00108243
    First Posted:
    Jul 26, 2021
    Last Update Posted:
    Jun 6, 2022
    Last Verified:
    Apr 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by Duke University
    MPS II
    Mucopolysaccharidosis II
    Hunter Syndrome
    Additional relevant MeSH terms:
    Mucopolysaccharidosis II
    Mucopolysaccharidoses

    Study Results

    No Results Posted as of Jun 6, 2022