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

NCT04724213 · Status: COMPLETED · Phase: NA · Type: INTERVENTIONAL · Enrollment: 101

Last updated 2023-12-05

No results posted yet for this study

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.

Conditions

  • Pompe Disease

Interventions

OTHER

Testing of reliability / validity of new questionnaire

Participants will answer new questionnaire and other, established, generic and chronic generic HrQol instruments

Sponsors & Collaborators

  • Sanofi

    collaborator INDUSTRY

  • University Children's Hospital, Zurich

    lead OTHER

Principal Investigators

  • Martina Huemer, Prof dr · University Childrens Hospital Zurich

Study Design

Allocation
NA
Purpose
OTHER
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
8 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-07-01
Primary Completion
2023-12-01
Completion
2023-12-01

Countries

  • Switzerland

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04724213 on ClinicalTrials.gov