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

University of Zurich (UZH)

Status: Completed

Conditions: Pompe Disease

Treatments: Other: Testing of reliability / validity of new questionnaire

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT04724213

Pompe_HrQol

Details and patient eligibility

About

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.

Enrollment: 101 patients

Sex: All

Ages: 8+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Other

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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101 participants in 1 patient group

Testing of reliability / validity of new questionnaire

Other group

Treatment:

Other: Testing of reliability / validity of new questionnaire