Status:
RECRUITING
Development of Health-related Quality of Life Instrument for Patients With Cystinosis
Lead Sponsor:
Cystinose Stiftung
Collaborating Sponsors:
Cystinosis Research Network
Leben eben! Cystinose-Selbsthilfe e.V.
Conditions:
Cystinosis
Eligibility:
All Genders
8-26 years
Brief Summary
Cystinosis is a rare congenital, inherited metabolic disorder that results in the storage of cystine in the cells of many organs of the body. In the infantile nephropathic form of the disease, only th...
Detailed Description
First, a literature review was conducted to identify relevant quality of life topics for the focus interviews. The development of the cystinosis-specific PROM will include three phases with patient re...
Eligibility 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
Key Trial Info
Start Date :
May 1 2022
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
May 31 2025
Estimated Enrollment :
300 Patients enrolled
Trial Details
Trial ID
NCT05959668
Start Date
May 1 2022
End Date
May 31 2025
Last Update
August 22 2024
Active Locations (1)
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1
University Medical Center Hamburg-Eppendorf | Center for Psychosocial Medicine | Department of Medical Psychology
Hamburg, Germany, 20246