Creating a quality of life tool for cystinosis patients
Development of a Patient-reported Outcome to Measure the Health-related Quality of Life of Children, Adolescents and Young Adults With Cystinosis.
Cystinose Stiftung · NCT05959668
This study is trying to create a new tool to help understand how cystinosis affects the quality of life for patients and their families.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 300 (estimated) |
| Ages | 8 Years to 26 Years |
| Sex | All |
| Sponsor | Cystinose Stiftung (other) |
| Locations | 1 site (Hamburg) |
| Trial ID | NCT05959668 on ClinicalTrials.gov |
What this trial studies
This study aims to develop a disease-specific patient-reported outcome measure (PROM) to assess the health-related quality of life for patients with cystinosis. It will involve three phases: conducting focus interviews with parents and young patients to identify relevant quality of life topics, pilot testing the instrument for comprehensibility and cultural relevance, and finally administering a refined questionnaire to a larger group for validation. The study will take place across multiple countries including Germany, France, Spain, and the USA, ensuring a diverse participant pool.
Who should consider this trial
Good fit: Ideal candidates include children, adolescents, and young adults aged 8-26 with a confirmed diagnosis of cystinosis, along with their parents.
Not a fit: Patients with severe cognitive impairment or other severe illnesses that significantly affect their daily life may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide a validated tool to better understand and improve the quality of life for patients with cystinosis.
How similar studies have performed: While there have been studies on quality of life in other conditions, the development of a specific PROM for cystinosis is a novel approach.
Eligibility criteria
Show full inclusion / exclusion criteria
In all study phases, patient recruitment follows these inclusion criteria: Patients will be asked to participate in the study if they meet the following inclusion criteria: * Children, adolescents, and young adults aged 8-26 years (and at least one of their parents) and further parents only of children aged 0-7 * patients have a confirmed diagnosis of cystinosis * patients have a sufficient knowledge of the German/ English/ French or Spanish language to participate in focus interviews and complete questionnaires * the informed consent of legal guardian and assent from the patient (if older than eight years) was given Exclusion criteria: * severe cognitive impairment * other severe illnesses that strongly determine everyday life
Where this trial is running
Hamburg
- University Medical Center Hamburg-Eppendorf | Center for Psychosocial Medicine | Department of Medical Psychology — Hamburg, Germany (RECRUITING)
Study contacts
- Study coordinator: Julia Hannah Quitmann, PD Dr.
- Email: j.quitmann@uke.de
- Phone: +49 (0) 40 7410 52789
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Cystinosis, Health-related quality of life, Patient-reported outcome measure