MPS II Immunophenotyping

Duke University

Status: Terminated

Conditions: Hunter Syndrome; MPS II; Mucopolysaccharidosis II

Study type: Observational

Funder types: Other

Identifiers

NCT04976231

Pro00108243

Details and patient eligibility

About

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.

Enrollment: 6 patients

Sex: All

Volunteers: No 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

Trial design

6 participants in 3 patient groups

Treatment Naive

Description:

Subjects who are naïve to ERT and start treatment with idursulfase

Treatment less than 3 years

Description:

Subjects who have received ERT for \<3 years

Treatment over 3 years

Description:

Subjects who have received ERT for 3 or more years