CLN2 standard care costing study
Research type
Research Study
Full title
CLN2 Disease Standard Care Costing Study at GOSH London (UK)
IRAS ID
242694
Contact name
Paul Gissen
Contact email
Sponsor organisation
BioMarin Europe Ltd.
Duration of Study in the UK
0 years, 11 months, 30 days
Research summary
CLN2 Disease Standard Care Costing Study at Great Ormond Street Hospital (GOSH) London
Classic neuronal ceroid lipofuscinosis type 2 (CLN2) disease is a rare genetic disorder that affects the body’s nervous system, leading to problems with movement and brain function. CLN2 disease progresses extremely quickly and leads to death by approximately 10 years of age. There are very few guidelines on how CLN2 disease patients should be treated, and about the costs associated with treating and caring for patients.
The aim of this study is to calculate the cost of treating and caring for CLN2 disease patients with standard care in the UK. Whilst there are no treatment benefits for study participants, it is hoped the data collected will provide information about the costs of treating and caring for patients with CLN2 disease in the UK.
Data from alive or deceased CLN2 disease patients can be included in the study. Patients will have been treated with standard care at GOSH within the last 15 years. Parents/legal guardians are required to have given informed consent for their child’s data to be used in the study.
Study participants will have their medical records accessed by a study researcher at GOSH, who will record details associated with treatment and care received for CLN2 disease. Personal details, such as names, will not be collected to prevent participants from being identified. The study researcher at GOSH will then share the data with the wider study research team in an aggregated form. The data collection period is expected to take between 6 to 12 months. The total cost of treating and caring for CLN2 disease patients will then be calculated.
The study is being funded by the pharmaceutical company BioMarin Europe Ltd and Costello Medical will be conducting the statistical analyses on the final data.
REC name
London - Central Research Ethics Committee
REC reference
19/LO/1637
Date of REC Opinion
15 May 2020
REC opinion
Further Information Favourable Opinion