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
• Status: Completed
• Phase: N/A
• 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
• 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

Related Conditions & MeSH terms

• Glycogen Storage Disease Type II
• Pompe Disease

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