MPS II Immunophenotyping

NCT ID: NCT04976231

Last Updated: 2024-09-19

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Total Enrollment

6 participants

Study Classification

OBSERVATIONAL

Study Start Date

2022-04-01

Study Completion Date

2024-04-26

Brief Summary

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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

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Conditions

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MPS II Mucopolysaccharidosis II Hunter Syndrome

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Treatment Naive

Subjects who are naïve to ERT and start treatment with idursulfase

No interventions assigned to this group

Treatment less than 3 years

Subjects who have received ERT for \<3 years

No interventions assigned to this group

Treatment over 3 years

Subjects who have received ERT for 3 or more years

No interventions assigned to this group

Eligibility Criteria

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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
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Duke University

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Priya Kishnani, MD

Role: PRINCIPAL_INVESTIGATOR

Duke University

Locations

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Duke University

Durham, North Carolina, United States

Site Status

Countries

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United States

Other Identifiers

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Pro00108243

Identifier Type: -

Identifier Source: org_study_id

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