MPS II Siblings Study
Research type
Research Study
Full title
Retrospective chart review analysis of pairs of siblings with Mucopolysaccharidosis type II to evaluate the effectiveness of idursulfase started at 12 months of age and younger
IRAS ID
308730
Contact name
James Davison
Contact email
Sponsor organisation
Takeda Pharmaceutical Company Limited
Duration of Study in the UK
0 years, 5 months, 20 days
Research summary
Hunter syndrome is an inherited genetic disorder. Enzyme replacement therapy with IV idursulfase (Elaprase®) has shown clinical benefits in MPS II patients within both clinical trials and post marketing observational studies. Despite the clinical benefits of Elaprase in MPS II patients demonstrated by studies conducted to date, no evidence has so far been generated in very young patients who started ERT at an age of 12 months or less.
Conducting a retrospective study, collecting data from patients who started ERT at an age of 12 months or less and describing their outcomes compared to their older siblings who started treatment at an age of 36 months or more, will help to address the current lack of scientific evidence on the effectiveness of Elaprase in this patient population and the impact of early initiation of ERT on clinical outcomes.Data will be collected retrospectively from patients’ medical charts from the date of diagnosis of MPS II to the last post Elaprase treatment initiation follow-up data available (i.e. minimum follow up time is 2 years).
REC name
South West - Cornwall & Plymouth Research Ethics Committee
REC reference
22/SW/0032
Date of REC Opinion
10 Mar 2022
REC opinion
Favourable Opinion