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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04724213
Recruitment Status : Not yet recruiting
First Posted : January 26, 2021
Last Update Posted : January 26, 2021
Sponsor:
Collaborator:
Sanofi
Information provided by (Responsible Party):
University Children's Hospital, Zurich

Study Description
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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
Pompe Disease Other: Testing of reliability / validity of new questionnaire Not Applicable

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated 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
Estimated Study Start Date : July 1, 2021
Estimated Primary Completion Date : September 1, 2022
Estimated Study Completion Date : January 1, 2024

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
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
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Primary Outcome Measures :
  1. Reliability/ validity of questionnaire [ Time Frame: 15 months ]

Eligibility Criteria
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Ages Eligible for Study:   8 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04724213


Contacts
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Contact: Martina Huemer, Prof Dr +41 44 266 ext 7593 martina.huemer@kispi.uzh.ch
Contact: Helene Werner, PD Dr. +41 44 266 ext 7111 helene.werner@kispi.uzh.ch

Sponsors and Collaborators
University Children's Hospital, Zurich
Sanofi
Investigators
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Principal Investigator: Martina Huemer, Prof dr University Childrens Hospital Zurich
More Information
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Responsible Party: University Children's Hospital, Zurich
ClinicalTrials.gov Identifier: NCT04724213    
Other Study ID Numbers: Pompe_HrQol
First Posted: January 26, 2021    Key Record Dates
Last Update Posted: January 26, 2021
Last Verified: January 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Glycogen Storage Disease Type II
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
 Metabolism, Inborn Errors
Genetic Diseases, Inborn
Glycogen Storage Disease
Carbohydrate Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases