AchondroplasiaNCT05603936
Adaption and Testing of the Quality of Life in Short Stature Youth (QoLISSY) Questionnaire for Parents With Children From 0-4
An observational study of Achondroplasia and Small for Gestational Age, run by Universitätsklinikum Hamburg-Eppendorf at 1 site in Germany.
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Achondroplasia, Small for Gestational Age, Growth Hormone Deficiency
- What is being tested?
- Questionnaire
- How many people
- 168 participants
- Where
- 1 siteGermany
- Study dates
- 1 Mar 2022to 31 Dec 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Parents of children aged 0-4 with the diagnosis Achondroplaisa, Short for Gestational Age or Growth Hormone Defiency
Who may be able to join
- Parents of children aged 0-4 with the diagnosis Achondroplaisa, Short for Gestational Age or Growth Hormone Defiency
Who cannot join
- Other diagnosis than Achondroplaisa, Short for Gestational Age or Growth Hormone Defiency
- Children older than 4 years of age
Trial locations and contacts
1 site in 1 country.
Germany1 site, 1 recruiting
- Recruiting
University Medical Center Hamburg-Eppendorf
Hamburg, 20246
Julia Quitmann, Dipl. Psych., +4940741052789, j.quitmann@uke.de
About this study
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.
Read the detailed 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.
- Being tested
- Questionnaire
- Keywords
- Health related quality of life
- Medical subject headings
- Achondroplasia, Dwarfism, Pituitary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
QuestionnaireOther
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.
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: June 2022 - March 2023
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Nov 2022
- Study start1 Mar 2022
- Primary completion30 Sep 2023 (estimated)
- Study completion31 Dec 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AchondroplasiaStudies of the same condition, open and under way.
- More trials run by Universitätsklinikum Hamburg-EppendorfThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Cross-sectional
- Enrolment
- 168 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Universitätsklinikum Hamburg-Eppendorf
- Sponsor type
- University, hospital or other
- Collaborators
- Bundesverband Kleinwüchsige Menschen und ihre Familien e.V., Pfizer
- Responsible party
- Sponsor
- Investigators
- Julia Quitmann, Principal investigator, Center for Psychosocial Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05603936
- Sponsor's protocol ID
- QoLISSY 0-4
- Status last verified
- Oct 2022
- First submitted
- 28 Oct 2022
- Last updated
- 3 Nov 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Adaption and Testing of the Quality of Life in Short Stature Youth (QoLISSY) Questionnaire for Parents With Children From 0-4
- ClinicalTrials.gov ID
- NCT05603936
- Acronym / sponsor ID
- QoLISSY 0-4
- Keywords
- Health related quality of life
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 168 participants (estimated)
- Lead sponsor
- Universitätsklinikum Hamburg-EppendorfUniversity, hospital or other
- Collaborators
- Bundesverband Kleinwüchsige Menschen und ihre Familien e.V., Pfizer
- Study start
- 1 Mar 2022 (actual)
- Primary completion
- 30 Sep 2023 (estimated)
- Study completion
- 31 Dec 2023 (estimated)
- First posted
- 3 Nov 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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.
Study design
- Observational model
- Other
- Time perspective
- Cross-sectional
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Parents of children aged 0-4 with the diagnosis Achondroplaisa, Short for Gestational Age or Growth Hormone Defiency
Key inclusion criteria (1 listed)
- Parents of children aged 0-4 with the diagnosis Achondroplaisa, Short for Gestational Age or Growth Hormone Defiency
Key exclusion criteria (2 listed)
- Other diagnosis than Achondroplaisa, Short for Gestational Age or Growth Hormone Defiency
- Children older than 4 years of age
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| QuestionnaireThe 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. | Other | – |
Primary outcome
Health related quality of life measured by the adapted version of the QoLISSY questionnaire
Time frame: June 2022 - March 2023
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.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Nov 2022.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Julia Quitmann principal investigator
Center for Psychosocial Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Achondroplasia and Small for Gestational Age, run by Universitätsklinikum Hamburg-Eppendorf at 1 site in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 3 Nov 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Julia Quitmann, Dipl. Psych. (+49 (040) 74 10 52 789, j.quitmann@uke.de), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Health related quality of life measured by the adapted version of the QoLISSY questionnaire (time frame: June 2022 - March 2023). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Nov 2022.