Adaption and Testing of the Quality of Life in Short Stature Youth (QoLISSY) Questionnaire for Parents With Children From 0-4

NCT ID: NCT05603936

Last Updated: 2022-11-03

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

UNKNOWN

Total Enrollment

168 participants

Study Classification

OBSERVATIONAL

Study Start Date

2022-03-01

Study Completion Date

2023-12-31

Brief Summary

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The aim of the study is the adaption, implementation and validation of the instrument for the investigation of the short stature specific quality of life (QoLISSY) for children (age 0-4 years) with achondroplasia (ACH), Small for Gestational Age (SGA) and Growth Hormone Deficiency (GHD) from a parental perspective.

Detailed Description

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The questionnaire is intended to be particularly applicable in clinical trials, with a focus on the impact of short stature and the physical functionality of those affected. The instrument will be further developed according to the "Guidelines for PRO instrument development". It should be practical in its application and interpretation and applicable in the international context.

In addition, it will be investigated whether, from the parents' point of view, other instruments are suitable for measuring the quality of life of their short statured children. Their preference regarding the instruments will be assessed so that a recommendation can be made as to which instruments are particularly suitable.

A systematic literature review will be conducted to identify suitable instruments to measure the quality of life of short statured children aged 0 to 4.

Approximately 10-20 parents of 0-4 year old children with ACH, GHD and SGA will be recruited for the qualitative data collection (n = 30 to 60). The patients will be recruited through the BKMF (German Association for People of Short Stature and their Families).

In order to test the psychometric properties of the instruments, approximately 30 parents (15 mothers / 15 fathers) of 0-4 year old children with ACH, GHD and SGA will be recruited for the quantitative part of the study (n = 90). All participants will be asked to complete the QoLISSY or other instruments found suitable in the pilot test. For the purpose of cross-validation, the PEDSQL will be used in parallel.

Two weeks after receiving the field test, the re-test will be sent to ensure the reliability of the study. The re-test will be administered to a minimum of 20% of the participants.

Conditions

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Achondroplasia Small for Gestational Age Growth Hormone Deficiency

Study Design

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Observational Model Type

OTHER

Study Time Perspective

CROSS_SECTIONAL

Interventions

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Questionnaire

The original QOLISSY questionnaire was first developed in 2011, it is available as a patient reported and a parent reported instrument. It includes:

* QoLISSY questionnaire for children and adolescents (8-12, 13-18 yrs)
* QoLISSY questionnaire for parents of children with short stature (4-7, 8-12 and 13-18 yrs) The QoLISSY instrument can be used to assess the health-related quality of life in short stature youth regardless of its cause; however it was specifically developed for patients with GHD and ISS and their parents.The aim of the current study is the adaption, implementation and validation of the instrument for the investigation of the short stature specific quality of life (QoLISSY) for children (age 0-4 years) with achondroplasia (ACH), Small for Gestational Age (SGA) and Growth Hormone Deficiency (GHD) from a parental perspective.

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

* Parents of children aged 0-4 with the diagnosis Achondroplaisa, Short for Gestational Age or Growth Hormone Defiency

Exclusion Criteria

* Other diagnosis than Achondroplaisa, Short for Gestational Age or Growth Hormone Defiency
* Children older than 4 years of age
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Bundesverband Kleinwüchsige Menschen und ihre Familien e.V.

UNKNOWN

Sponsor Role collaborator

Pfizer

INDUSTRY

Sponsor Role collaborator

Universitätsklinikum Hamburg-Eppendorf

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Julia Quitmann

Role: PRINCIPAL_INVESTIGATOR

Center for Psychosocial Medicine

Locations

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University Medical Center Hamburg-Eppendorf

Hamburg, , Germany

Site Status RECRUITING

Countries

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Germany

Central Contacts

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Julia Quitmann, Dipl. Psych.

Role: CONTACT

+49 (040) 74 10 52 789

Facility Contacts

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Julia Quitmann, Dipl. Psych.

Role: primary

+4940741052789

Other Identifiers

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QoLISSY 0-4

Identifier Type: -

Identifier Source: org_study_id

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