Development of Health-related Quality of Life Instrument for Patients With Cystinosis
- Conditions
- Cystinosis
- Registration Number
- NCT05959668
- Lead Sponsor
- Cystinose Stiftung
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- Not specified
In all study phases, patient recruitment follows these inclusion criteria:<br><br>Patients will be asked to participate in the study if they meet the following inclusion<br>criteria:<br><br> - Children, adolescents, and young adults aged 8-26 years (and at least one of their<br> parents) and further parents only of children aged 0-7<br><br> - patients have a confirmed diagnosis of cystinosis<br><br> - patients have a sufficient knowledge of the German/ English/ French or Spanish<br> language to participate in focus interviews and complete questionnaires<br><br> - the informed consent of legal guardian and assent from the patient (if older than<br> eight years) was given<br><br>Exclusion criteria:<br><br> - severe cognitive impairment<br><br> - other severe illnesses that strongly determine everyday life
Not provided
Study & Design
- Study Type
- Observational
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Semi-structured interviews with young patients with cystinosis and their parents to identify relevant aspects of disease-specific HrQoL;Pilot-test version including a cognitive debriefing to assess the preliminary disease-specific HrQoL tool (QUALIFY) for young people with cystinosis;Field-test and of the final version of the disease-specific HrQoL tool (QUALIFY) for young people with cystinosis
- Secondary Outcome Measures
Name Time Method Health related quality of life measured by the chronic-generic DISABKIDS Questionnaire;Health related quality of life measured by the generic PedsQL™ Generic Core Scales;Health related quality of life measured by the generic PedsQL™ Infant Scales;Impact of pediatric chronic health condition on parents and the family by the PedsQL™ Family Impact Modules;Health related quality of life measured by the newly developed (disease-specific) PROM