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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT05603936
Other study ID # QoLISSY 0-4
Secondary ID
Status Recruiting
Phase
First received
Last updated
Start date March 1, 2022
Est. completion date December 31, 2023

Study information

Verified date October 2022
Source Universitätsklinikum Hamburg-Eppendorf
Contact Julia Quitmann, Dipl. Psych.
Phone +49 (040) 74 10 52 789
Email j.quitmann@uke.de
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

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.


Description:

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.


Recruitment information / eligibility

Status Recruiting
Enrollment 168
Est. completion date December 31, 2023
Est. primary completion date September 30, 2023
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility 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

Study Design


Intervention

Other:
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.

Locations

Country Name City State
Germany University Medical Center Hamburg-Eppendorf Hamburg

Sponsors (3)

Lead Sponsor Collaborator
Universitätsklinikum Hamburg-Eppendorf Bundesverband Kleinwüchsige Menschen und ihre Familien e.V., Pfizer

Country where clinical trial is conducted

Germany, 

Outcome

Type Measure Description Time frame Safety issue
Primary Health related quality of life measured by the adapted version of the QoLISSY questionnaire The studies aim is to identify factors influencing the health related quality of life of children with Achondroplaise, Growth Hormone Deficiency and Small for Gestational Age to adapt and test the QOLISSY-Questionnaire for children from the age of 0-4. June 2022 - March 2023
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