QoL and resource use in Arginase-1 deficiency patients and carers
Research type
Research Study
Full title
A European Survey of Resource Use and Health-Related Quality of Life in Patients with Arginase 1 Deficiency and their Caregivers
IRAS ID
316363
Contact name
Karolina Stepien
Contact email
Sponsor organisation
Immedica Pharma AB Sweden
Duration of Study in the UK
0 years, 4 months, 31 days
Research summary
This study aims to collect disease specific data to assess the burden of illness of Arginase-1 deficiency disease on patients and their caregivers, taking into account specific patient characteristics and treatment patterns. For the individual with Arginase-1 Deficiency which is a subtype of a Urea Cycle Disorder (ARG1-D), the study will collect data on background information (age, gender, education etc), diagnosis and treatment, symptoms and disease severity, direct medical resource use (health care, medications), non-medical resource use (caregiving, devices, specialised school etc.), indirect cost (production loss), and intangible costs (like Health related quality of Life).
For the primary caregiver to the individual with ARG1-D (likely the parent), the study will collect data on background information, caregiving, work and productivity, and health related quality of life.
Data on resource use is collected in detail for the last 12 months. Data on health related quality of life is collected in detail for the time of survey completion.REC name
Yorkshire & The Humber - Leeds East Research Ethics Committee
REC reference
23/YH/0023
Date of REC Opinion
24 Apr 2023
REC opinion
Further Information Favourable Opinion