MPS II (Hunter Syndrome) Natural History study
Research type
Research Study
Full title
A Retrospective and Cross-sectional Study to Evaluate Neurodevelopmental Status in Pediatric Subjects with Severe Mucopolysaccharidosis Type II (Hunter Syndrome).
IRAS ID
242237
Contact name
Jacob Wesley, Pharm.D, MS
Contact email
Duration of Study in the UK
years, 4 months, days
Research summary
The purpose of this study is to learn more about the natural history of the severe form of Hunter syndrome (Mucopolysaccharides Type II - MPS II). There is currently limited information on the natural history of the disease, i.e. as to when the symptoms develop and change during an individual's life, especially with respect to neurocognitive decline in patients with the more severe form of the disease.
This is planned to be a medical records review study in severe MPS II subjects where data will be collected retrospectively. As part of an optional sub-study, additional information will be collected from parent(s)/legal guardian(s) in a subset of the main study subjects using questionnaires.
The study is observational. There is no drug treatment being given, and there are no procedures associated with subject participation.
Collectively the data may inform the design of future MPS II gene therapy treatment studies and may be utilized as historical comparative control data.
This study may also further help characterize the burden of the disease on subjects and families.REC name
London - Harrow Research Ethics Committee
REC reference
18/LO/0716
Date of REC Opinion
11 Jun 2018
REC opinion
Further Information Favourable Opinion