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

Primary Purpose

Pompe Disease

Status
Recruiting
Phase
Not Applicable
Locations
Switzerland
Study Type
Interventional
Intervention
Testing of reliability / validity of new questionnaire
Sponsored by
University Children's Hospital, Zurich
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional other trial for Pompe Disease

Eligibility Criteria

8 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

  • University Childrens Hospital ZürichRecruiting

Arms of the Study

Arm 1

Arm Type

Other

Arm Label

Testing of reliability / validity of new questionnaire

Arm Description

Outcomes

Primary Outcome Measures

Reliability/ validity of questionnaire

Secondary Outcome Measures

Full Information

First Posted
January 20, 2021
Last Updated
May 25, 2022
Sponsor
University Children's Hospital, Zurich
Collaborators
Sanofi
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1. Study Identification

Unique Protocol Identification Number
NCT04724213
Brief Title
Development of an Assessment Tool for Health-related Quality of Life in Children and Adolescents With Pompe Disease
Acronym
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
Study Type
Interventional

2. Study Status

Record Verification Date
May 2022
Overall Recruitment Status
Recruiting
Study Start Date
July 1, 2021 (Actual)
Primary Completion Date
December 1, 2023 (Anticipated)
Study Completion Date
January 1, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University Children's Hospital, Zurich
Collaborators
Sanofi

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No

5. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pompe Disease

7. Study Design

Primary Purpose
Other
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
80 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Testing of reliability / validity of new questionnaire
Arm Type
Other
Intervention Type
Other
Intervention Name(s)
Testing of reliability / validity of new questionnaire
Intervention Description
Participants will answer new questionnaire and other, established, generic and chronic generic HrQol instruments
Primary Outcome Measure Information:
Title
Reliability/ validity of questionnaire
Time Frame
15 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
8 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Martina Huemer, Prof Dr
Phone
+41 44 266
Ext
7593
Email
martina.huemer@kispi.uzh.ch
First Name & Middle Initial & Last Name or Official Title & Degree
Helene Werner, PD Dr.
Phone
+41 44 266
Ext
7111
Email
helene.werner@kispi.uzh.ch
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Martina Huemer, Prof dr
Organizational Affiliation
University Childrens Hospital Zurich
Official's Role
Principal Investigator
Facility Information:
Facility Name
University Childrens Hospital Zürich
City
Zürich
ZIP/Postal Code
8032
Country
Switzerland
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Martina Huemer, Prof
Phone
+41442667593
Email
martina.huemer@kispi.uzh.ch

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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