An observational study in Achondroplasia, Small for Gestational Age and Growth Hormone Deficiency, sponsored by Universitätsklinikum Hamburg-Eppendorf. Status unknown at 1 site in Germany. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2022-11-03.
Sponsored by Universitätsklinikum Hamburg-Eppendorf · Observational
Among 10 studies in Achondroplasia with a site in Germany — 2 open to participants now, 3 under way, 2 of them phase 3. Achondroplasia · Germany.
Among the 10 studies in Achondroplasia with a site in Germany, this one is the only one that started in 2022, is one of 5 observational ones, and was the 8th registered.
47 studies on the registry are indexed under Achondroplasia; 13 are open to participants now.
Browse Achondroplasia studies →2 of the 10 studies in Achondroplasia with a site in Germany are open now. Recruiting first, the newest first:
Ascendis Pharma A/S · Sites in Germany, United States, Australia and 12 more countries
Ascendis Pharma Growth Disorders A/S · Sites in Germany, United States, Australia and 9 more countries
A study can be open while its sites in Germany have not opened yet or have stopped. Only those recruiting at a site in Germany →
Of the 9 Achondroplasia studies recruiting now, 2 (22%) set no upper age limit and 7 (78%) stop at 75 or younger; 9 take children under 18; 0 take healthy volunteers.
This study: 18 Years and older · all sexes · no healthy volunteers.
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10 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 2 3 · Phase 3 3
First in 2016; this study’s year is marked. 1 started in 2022.
This study's planned enrollment of 168 is below the median of 200 across 19 observational studies indexed under Achondroplasia.
Median enrollment 119 across the 5 interventional studies, 260 across the 5 observational. Of the 6 that completed or were terminated, 3 (50%) have results posted.
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9 of 10 have an industry lead sponsor. The 6 most frequent:
Universitätsklinikum Hamburg-Eppendorf is the lead sponsor of 416 studies on the registry; 86 are open to participants now.
Universitätsklinikum Hamburg-Eppendorf has 1 study filed under Achondroplasia — fewer than ten, so there is no comparison to draw.
None of the studies under way reaches primary completion within a year.
Among the 10 studies in Achondroplasia with a site in Germany, from their registry updates:
Nothing recorded among them since 2026-09-25.
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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.
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.
Parents of children aged 0-4 with the diagnosis Achondroplaisa, Short for Gestational Age or Growth Hormone Defiency
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
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.
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.
Time frame: June 2022 - March 2023
Plan to share: No
No publications or documents are linked to this record.
Started Mar 2022 (actual). Primary completion is estimated for Sep 2023, 18 months after the start.
How these figures are worked out, and which studies are left out of them.
This study is status unknown, as verified in Oct 2022. You cannot join it, but the record below documents what was studied.
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Universitätsklinikum Hamburg-Eppendorf →