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

  • End date
    Jan 1, 2024
  • participants needed
  • sponsor
    University Children's Hospital, Zurich
Updated on 13 June 2022


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.

Condition Pompe Disease
Treatment Testing of reliability / validity of new questionnaire
Clinical Study IdentifierNCT04724213
SponsorUniversity Children's Hospital, Zurich
Last Modified on13 June 2022


Yes No Not Sure

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
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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