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

NCT ID: NCT04724213

Last Updated: 2023-12-05

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

NA

Total Enrollment

101 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-07-01

Study Completion Date

2023-12-01

Brief Summary

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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.

Detailed Description

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Conditions

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Pompe Disease

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

OTHER

Blinding Strategy

NONE

Study Groups

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Testing of reliability / validity of new questionnaire

Group Type OTHER

Testing of reliability / validity of new questionnaire

Intervention Type OTHER

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

Interventions

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Testing of reliability / validity of new questionnaire

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

Intervention Type OTHER

Eligibility Criteria

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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
Minimum Eligible Age

8 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Sanofi

INDUSTRY

Sponsor Role collaborator

University Children's Hospital, Zurich

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Martina Huemer, Prof dr

Role: PRINCIPAL_INVESTIGATOR

University Childrens Hospital Zurich

Locations

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University Childrens Hospital Zürich

Zurich, , Switzerland

Site Status

Countries

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Switzerland

Other Identifiers

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Pompe_HrQol

Identifier Type: -

Identifier Source: org_study_id