Qualitative Analysis of Niemann-Pick Type C 5-Domain NPCCSS v1.0
Research type
Research Study
Full title
Qualitative Analysis of Niemann-Pick Type C Clinical Severity 5-Domain Score (5-Domain NPCCSS)
IRAS ID
257087
Contact name
Claire Burbridge
Contact email
Sponsor organisation
Clinical Outcomes Solutions
Duration of Study in the UK
0 years, 7 months, 23 days
Research summary
The purpose of conducting this participant research is to explore the patient and caregiver journey of Niemann-Pick Disease Type C (NPC)from first symptoms through to diagnosis and present day and to evaluate the importance and relevance of each domain of the 5-domain NPCCSS (a clinician-reported outcome measure). This is a non-drug, non-interventional study.
There are two parts to this study, approximately 60 participants (a mixture of caregivers and patients) will firstly take part in a web-based survey. Survey questions will include demographics, patient journey, severity of NPC, relevancy and impact of symptoms and estimated costs of NPC.
A follow-up telephone interview will then be conducted with a subset of approximately 30 participants (caregivers and patients) that completed the survey. The interview is expected to take approximately 60 minutes and will probe further about the impacts of NPC identified in the web-based survey. The interview will review and probe further about: (1) the impact of the symptoms of importance and the 5-domain NPCCSS symptoms on their day-to-day lives and health-related quality of life (HRQOL); (2) meaningful change - what a decline or no change in each of these individual symptoms would mean to them; (3) the patient journey - major events pre- and post-diagnosis, and (4) costs of NPC - the out-of-pocket expenses associated with their NPC over time.
Prior to the interview part of the study, participants will be asked to create a timeline of major events from the onset of NPC symptoms to diagnosis of NPC and since the patient’s diagnosis.
REC name
London - Hampstead Research Ethics Committee
REC reference
19/LO/0769
Date of REC Opinion
5 Jun 2019
REC opinion
Further Information Favourable Opinion