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

Sponsor
University Children's Hospital, Zurich (Other)
Overall Status
Recruiting
CT.gov ID
NCT04724213
Collaborator
Sanofi (Industry)
80
Enrollment
1
Location
1
Arm
30
Anticipated Duration (Months)
2.7
Patients Per Site Per Month

Study Details

Study Description

Brief 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.

Condition or Disease Intervention/Treatment Phase
  • Other: Testing of reliability / validity of new questionnaire
 N/A

Study Design

Study Type:
Interventional
Anticipated Enrollment :
80 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Other
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
Actual Study Start Date :
Jul 1, 2021
Anticipated Primary Completion Date :
Dec 1, 2023
Anticipated Study Completion Date :
Jan 1, 2024

Arms and Interventions

Arm Intervention/Treatment
Other: Testing of reliability / validity of new questionnaire

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

Outcome Measures

Primary Outcome Measures

  1. Reliability/ validity of questionnaire [15 months]

Eligibility Criteria

Criteria

Ages Eligible for Study:
8 Years and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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

Contacts and Locations

Locations

Site City State Country Postal Code
1 University Childrens Hospital Zürich Zürich Switzerland 8032

Sponsors and Collaborators

  • University Children's Hospital, Zurich
  • Sanofi

Investigators

  • Principal Investigator: Martina Huemer, Prof dr, University Childrens Hospital Zurich

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
University Children's Hospital, Zurich
ClinicalTrials.gov Identifier:
NCT04724213
Other Study ID Numbers:
  • Pompe_HrQol
First Posted:
Jan 26, 2021
Last Update Posted:
May 31, 2022
Last Verified:
May 1, 2022
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:
Glycogen Storage Disease Type II

Study Results

No Results Posted as of May 31, 2022