Growth Hormone TreatmentHF QoLNCT05195437
Evolution of the Quality of Life of Short Statured Children Treated With Growth Hormone : Evaluation at Adult Size
An observational study of Growth Hormone Treatment, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
Short stature can lead to emotional and social stress in children and adolescents, as well as their parents. Children and their parents want to be able to identify the cause of stunted growth and address it with treatment. Mitigating the impact of short stature on quality of life is one of the main goals of treatment. Read more about this study
- Who is it for?
- Ages 15 years and older
- Conditions the study is for
- Growth Hormone Treatment
- What is being tested?
- QoLiSSY and PedsQL 4.0 questionnaires
- How many people
- 36 participants
- Where
- 1 siteFrance
- Study dates
- 8 Mar 2022to 12 Oct 2024
- 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 15 years and older
- All sexes
- Child, Adult, Older adult
Study population: Adolescents and young adults followed or having been followed by the endocrinology, gynecology and pediatric diabetology department of the Necker Enfants Malades hospital, who reached their final height, who have been treated with growth hormone due to short stature and who had participed in the initial study : PedsQL 4.0 and QoLiSSY questionnaires completed at starting growth hormone therapy and after one year of growth hormone therapy.
Who may be able to join
- Adolescents and young adults followed or having been followed by the endocrinology, gynecology and pediatric diabetology department of the Necker Enfants Malades hospital and treated with growth hormone because of their short stature.
- Patient who participated in the initial study.
- Patient having reached the final height during the last consultation (growth rate less than 1 cm / year and / or bone age ≥ 15 years for girls, ≥ 17 years for boys).
- Holders of parental authority, minor patients and adult patients informed and not opposed to their participation in the study.
Who cannot join
- Refusal to participate in the study.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Evolution of the Quality of Life of Short Stature Children Treated With Growth Hormone : Evaluation at Adult Size
Short stature can lead to emotional and social stress in children and adolescents, as well as their parents. Children and their parents want to be able to identify the cause of stunted growth and address it with treatment. Mitigating the impact of short stature on quality of life is one of the main goals of treatment. The quality of life in children can be measured using adapted self-questionnaires.
The investigative team published in 2019 the results of a preliminary study which shows that after one year of treatment with growth hormone, the quality of life improves in children, in particular on the scales emotional and social. These evaluations were carried out in particular thanks to the general questionnaire of quality of life: Pediatric Quality of Life Inventory (PedsQL) 4.0, but also via a specific questionnaire of the size: Quality of Life of Short Stature Youth questionnaire (QoLiSSY).
50 of the 74 patients who participated in this study have now reached their final height. The objective of the present study is to reassess this cohort using the QoLiSSY and PedsQL 4.0 questionnaires. The patient will be his own witness.
Read the detailed description
Short stature can lead to emotional and social stress in children and adolescents, as well as their parents. Children and their parents want to be able to identify the cause of stunted growth and address it with treatment. Mitigating the impact of short stature on quality of life is one of the main goals of treatment. The quality of life in children can be measured using adapted self-questionnaires.
The investigative team published in 2019 the results of a preliminary study which shows that after one year of treatment with growth hormone, the quality of life improves in children, in particular on the scales emotional and social. These evaluations were carried out in particular thanks to the general questionnaire of quality of life: Pediatric Quality of Life Inventory (PedsQL) 4.0, but also via a specific questionnaire of the size: Quality of Life of Short Stature Youth questionnaire (QoLiSSY).
50 of the 74 patients who participated in this study have now reached their final height. The objective of the present study is to reassess this cohort using the QoLiSSY and PedsQL 4.0 questionnaires. The patient will be his own witness.
- Conditions
- Growth Hormone Treatment
- Being tested
- QoLiSSY and PedsQL 4.0 questionnaires
- Keywords
- Growth hormone treatment, Quality of Life of Short Stature Youth (QoLiSSY) questionnaire, Pediatric Quality of Life Inventory (PedsQL) 4.0, Quality of life improvement
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
QoLiSSY and PedsQL 4.0 questionnairesOther
Patients have been reaching adult size complete the two questionnaires of the study :
Quality of Life of Short Stature Youth questionnaire (QoLiSSY), assessing health-related quality of life in short stature youth.
Pediatric Quality of Life Inventory (PedsQL) 4.0 : general health-related quality of life.
The patients have previously completed the PedsQL 4.0 and QoLiSSY questionnaires when starting their growth hormone therapy and after one year of growth hormone therapy.
| Study group | Role | What they receive |
|---|---|---|
| Growth hormone treatment | Adolescents and young adults followed or having been followed by the endocrinology, gynecology and pediatric diabetology department of the Necker Enfants Malades hospital, who reached their final height and who have been treated with growth hormone due to short stature. Receives: Other: QoLiSSY and PedsQL 4.0 questionnaires |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evolution of the quality of life
Evolution of the quality of life in short patients who have been treated with growth hormone. Comparison of the scores of the initial Quality of Life in Short Stature Youth (QoLiSSY) questionnaire (start of growth hormone treatment) compared to the scores obtained when the patients reached their final height. QoLISSY questionnaires include two different forms: parent questionnaire (children aged 4 to 18 years of age) and child questionnaire (only for children ≥8 years of age). Both questionnaires have 50 items that cover three core domains (physical, emotional, and social), and additional complementary domains. The parent questionnaire includes two additional scales (16 items) assessing parental stress and anxiety. Scores are converted in SD scores and compared to age- and gender-specific data from a reference population of children and adolescents with short stature. A total score is computed as the mean score on the three core scales.
Measured: Day 0
Secondary outcomes (2)
Adult-size quality of life
Change in scores for the two questionnaires QoLISSY and Pediatric Quality of Life Inventory (PedsQL) 4.0 at adult size.
Children younger than 8 years of age completed the questionnaire PedsQL 4.0 with their parents. The PedsQL has 23 items that investigate physical, emotional, and social QoL, as well as school functioning. This tool has been validated in the 2- to 18-year age range. The parents complete the questionnaire if the child is younger than 5 years. After that age, the child completes the age-appropriate version of the questionnaire (5-7, 8-12, or 13-18 years). The score is expressed as a percentage, with scores from 0 to 100%, 100% being the best score. Questionnaire subscales are considered evaluable if answers are provided for at least 80% of the items. High results being associated with a better quality of life related to health.
Measured: Day 0
Correlation between quality of life changes and clinical response to growth hormone therapy
Correlation between the changes in scores on the 2 questionnaires QoLISSY and PedsQL 4.0 at adult size and number of centimeters taken since the start of growth hormone treatment.
Measured: Day 0
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jan 2022
- Study start8 Mar 2022
- Primary completion12 Oct 2024
- Study completion12 Oct 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Growth Hormone TreatmentStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe 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
- Case only
- Time perspective
- Prospective
- Enrolment
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Collaborators
- URC-CIC Paris Descartes Necker Cochin
- Responsible party
- Sponsor
- Investigators
- Michel Polak, MD, PhD, Principal investigator, Assistance Publique - Hôpitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05195437
- Sponsor's protocol ID
- APHP211523
- Other IDs
- 2024-A01272-45
- Acronym
- HF QoL
- Status last verified
- Sep 2025
- First submitted
- 4 Jan 2022
- Last updated
- 12 Sep 2025
- 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 12 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evolution of the Quality of Life of Short Stature Children Treated With Growth Hormone : Evaluation at Adult Size
- ClinicalTrials.gov ID
- NCT05195437
- Acronym / sponsor ID
- HF QoL / APHP211523
- Conditions
- Growth Hormone Treatment
- Keywords
- Growth hormone treatment, Quality of Life of Short Stature Youth (QoLiSSY) questionnaire, Pediatric Quality of Life Inventory (PedsQL) 4.0, Quality of life improvement
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Collaborators
- URC-CIC Paris Descartes Necker Cochin
- Study start
- 8 Mar 2022 (actual)
- Primary completion
- 12 Oct 2024 (actual)
- Study completion
- 12 Oct 2024 (actual)
- First posted
- 19 Jan 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.
Short stature can lead to emotional and social stress in children and adolescents, as well as their parents. Children and their parents want to be able to identify the cause of stunted growth and address it with treatment. Mitigating the impact of short stature on quality of life is one of the main goals of treatment. The quality of life in children can be measured using adapted self-questionnaires.
The investigative team published in 2019 the results of a preliminary study which shows that after one year of treatment with growth hormone, the quality of life improves in children, in particular on the scales emotional and social. These evaluations were carried out in particular thanks to the general questionnaire of quality of life: Pediatric Quality of Life Inventory (PedsQL) 4.0, but also via a specific questionnaire of the size: Quality of Life of Short Stature Youth questionnaire (QoLiSSY).
50 of the 74 patients who participated in this study have now reached their final height. The objective of the present study is to reassess this cohort using the QoLiSSY and PedsQL 4.0 questionnaires. The patient will be his own witness.
Detailed description
Short stature can lead to emotional and social stress in children and adolescents, as well as their parents. Children and their parents want to be able to identify the cause of stunted growth and address it with treatment. Mitigating the impact of short stature on quality of life is one of the main goals of treatment. The quality of life in children can be measured using adapted self-questionnaires.
The investigative team published in 2019 the results of a preliminary study which shows that after one year of treatment with growth hormone, the quality of life improves in children, in particular on the scales emotional and social. These evaluations were carried out in particular thanks to the general questionnaire of quality of life: Pediatric Quality of Life Inventory (PedsQL) 4.0, but also via a specific questionnaire of the size: Quality of Life of Short Stature Youth questionnaire (QoLiSSY).
50 of the 74 patients who participated in this study have now reached their final height. The objective of the present study is to reassess this cohort using the QoLiSSY and PedsQL 4.0 questionnaires. The patient will be his own witness.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Growth hormone treatment | – | Other: QoLiSSY and PedsQL 4.0 questionnairesAdolescents and young adults followed or having been followed by the endocrinology, gynecology and pediatric diabetology department of the Necker Enfants Malades hospital, who reached their final height and who have been treated with growth hormone due to short stature. |
Who may qualify
- Ages
- 15 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
- Study population
- Adolescents and young adults followed or having been followed by the endocrinology, gynecology and pediatric diabetology department of the Necker Enfants Malades hospital, who reached their final height, who have been treated with growth hormone due to short stature and who had participed in the initial study : PedsQL 4.0 and QoLiSSY questionnaires completed at starting growth hormone therapy and after one year of growth hormone therapy.
Key inclusion criteria (4 listed)
- Adolescents and young adults followed or having been followed by the endocrinology, gynecology and pediatric diabetology department of the Necker Enfants Malades hospital and treated with growth hormone because of their short stature.
- Patient who participated in the initial study.
- Patient having reached the final height during the last consultation (growth rate less than 1 cm / year and / or bone age ≥ 15 years for girls, ≥ 17 years for boys).
- Holders of parental authority, minor patients and adult patients informed and not opposed to their participation in the study.
Key exclusion criteria (1 listed)
- Refusal to participate in the study.
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 |
|---|---|---|
| QoLiSSY and PedsQL 4.0 questionnairesPatients have been reaching adult size complete the two questionnaires of the study : Quality of Life of Short Stature Youth questionnaire (QoLiSSY), assessing health-related quality of life in short stature youth. Pediatric Quality of Life Inventory (PedsQL) 4.0 : general health-related quality of life. The patients have previously completed the PedsQL 4.0 and QoLiSSY questionnaires when starting their growth hormone therapy and after one year of growth hormone therapy. | Other | Growth hormone treatment |
Primary outcome
Evolution of the quality of life
Time frame: Day 0
Evolution of the quality of life in short patients who have been treated with growth hormone. Comparison of the scores of the initial Quality of Life in Short Stature Youth (QoLiSSY) questionnaire (start of growth hormone treatment) compared to the scores obtained when the patients reached their final height. QoLISSY questionnaires include two different forms: parent questionnaire (children aged 4 to 18 years of age) and child questionnaire (only for children ≥8 years of age). Both questionnaires have 50 items that cover three core domains (physical, emotional, and social), and additional complementary domains. The parent questionnaire includes two additional scales (16 items) assessing parental stress and anxiety. Scores are converted in SD scores and compared to age- and gender-specific data from a reference population of children and adolescents with short stature. A total score is computed as the mean score on the three core scales.
2 secondary outcomes
Adult-size quality of life
Time frame: Day 0
Correlation between quality of life changes and clinical response to growth hormone therapy
Time frame: Day 0
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Sep 2025.
- 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.
Michel Polak, MD, PhD principal investigator
Assistance Publique - Hôpitaux de Paris
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 Growth Hormone Treatment, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
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 4 inclusion and 1 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: finished, as of the sponsor's last update on 12 Sep 2025. 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 France. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evolution of the quality of life (time frame: Day 0). 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 12 Sep 2025.