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NCT04724213 Clinical Trial

Development of an Assessment Tool for Health-related Quality of Life in Children and Adolescents With Pompe Disease

Pompe Disease Clinical Trial

— Pompe_HrQol  

Official title:
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

Clinical Trial Details — Status: Completed

Administrative data
NCT number NCT04724213
Other study ID # Pompe_HrQol
Secondary ID
Status Completed
Phase N/A
First received
Last updated
Start date July 1, 2021
Est. completion date December 1, 2023

Study information
Verified date December 2023
Source University Children's Hospital, Zurich
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility
Status Completed
Enrollment 101
Est. completion date December 1, 2023
Est. primary completion date December 1, 2023
Accepts healthy volunteers No
Gender All
Age group 8 Years and older
Eligibility Inclusion Criteria: - 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 Exclusion Criteria: - - Inability to follow the procedures of the study, e.g. due to language problems or severely reduced health status

Study Design

Related Conditions & MeSH terms

Intervention

Other:
• Testing of reliability / validity of new questionnaire
Participants will answer new questionnaire and other, established, generic and chronic generic HrQol instruments

Locations
Country Name City State
Switzerland University Childrens Hospital Zürich Zürich

Sponsors (2)
Lead Sponsor Collaborator
University Children's Hospital, Zurich Sanofi

Country where clinical trial is conducted

Switzerland, 

Outcome
Type Measure Description Time frame Safety issue
Primary Reliability/ validity of questionnaire 15 months
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