NCT04724213Completed
Development of an Assessment Tool for Health-related Quality of Life in Children and Adolescents With Pompe Disease
Development and Validation of a Standardized Assessment Instrument for Health-related Quality of Life (HrQoL) in Children and Adolescents With Pompe Disease (PD): a Joint Effort of Patients, Caregivers and Metabolic Experts
Source: ClinicalTrials.gov · Last updated 5 Dec 2023
Brief summary (registry, in English)
Health related quality of life (HrQoL), is "the patient's subjective perception of the impact of his disease and its treatment on his daily life, physical, psychological and social functioning and well-being" and thus constitutes a patient reported outcome (PRO) of utmost importance. Generic HrQoL instruments can by definition not capture disease-specific parameters nor are they sensitive enough to detect their changes. In this study, a disease-specific HrQoL questionnaire for children and adolescents will be developed. Patients and parents will be involved in focus groups and interviews to identify relevant contents. The instrument will be tested for validity and reliability.
- Status (registry)
- Completed
- Study type
- Interventional
- Enrollment
- 101 (actual)
- First submitted
- 20 Jan 2021
- Start date
- Jul 2021
- Primary completion date
- Dec 2023
- Lead sponsor
- University Children's Hospital, Zurich
- Diseases
- Pompe disease
- NCT number
- NCT04724213
What you commit to
- Study type: Interventional
- Participants enrolled: 101
- From Jul 2021 to the primary completion date, Dec 2023
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other
- \- Inability to follow the procedures of the study, e.g. due to language problems or severely reduced health status
Who the study is looking for
- Patients with PD from age 8 years
- Parent of patient(s) \< 18 years with PD
- Able to give informed consent as documented by signature
- Sufficient command of the German language
Age in the registry: 8 years to - · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| University Childrens Hospital Zürich Coordinator of this site? Confirm its status | Zurich | Switzerland | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.