Validation of a Questionnaire That Identifies the Reasons for Non-adherence to Existing Growth Hormone Therapy

Study Overview
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Brief Summary
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The aim of the study is to examine the suitability, the so-called validation, of a questionnaire, with which one can grasp the reasons why injections of growth hormones are omitted by patients (non-adherence). Participants are treated with growth hormone and are therefore eligible to take part in the study. Study doctor will ask participants to answer questionnaires. two times within 14 days. The first time participants answer during the routine visit to the practice / clinic and the second time at home. There are no risks associated with participating in the study as it does not affect participant's medical treatment.

Official Title
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Validation of a Patient-reported Outcome (PRO) Measure That Assesses Reasons for Non-adherence to Growth Hormone Therapy (GHT)

Conditions
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Growth Hormone Deficiency in Children

Intervention / Treatment
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* Other: No treatment given


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: - Children/adolescents, male or female, aged 8 to 18 years who self-inject GH as well as their parents/legal guardians or Parents/Legal guardians who administer GH to a child/adolescent, male or female, from birth to 18 years - The child/adolescent is on prescribed daily GHT due to idiopathic growth hormone deficiency (GHD), multiple pituitary hormone deficiency (MPHD/organic GHD), small for gestational age (SGA) or Turner syndrome (TS) for at least 6 months before screening visit - Signed informed consent obtained by parent/legal guardian and child/adolescent before any study-related activities (study-related activities are any procedure related to recording of data according to the protocol) Exclusion Criteria: - Mental incapacity, unwillingness or language barrier of participant precluding adequate understanding or cooperation - Previous participation of participant in this study. Participation is defined as having given informed consent in this study - Treatment of child/adolescent with any investigational drug within 30 days prior to enrolment into the study
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Children and adolescents<br>Children and adolescents, who administer growth hormone (GH) themselves (self-injections) will be asked to complete the questionnaire. | Other: No treatment given<br>* No treatment is given to study participants. Only evaluation of a questionnaire assessing reasons for non-adherence to growth hormone therapy (GHT) and to determine its validity and reliability. The preliminary working title of the questionnaire GHCQ used in the outcome measures was renamed for simplicity reasons into BAR-GHT (abbreviation for barriers to growth hormone therapy) after study completion.<br>|
| Parents/legal guardians<br>Parents/legal guardians who administer the GH to their child will be asked to complete the questionnaire. | Other: No treatment given<br>* No treatment is given to study participants. Only evaluation of a questionnaire assessing reasons for non-adherence to growth hormone therapy (GHT) and to determine its validity and reliability. The preliminary working title of the questionnaire GHCQ used in the outcome measures was renamed for simplicity reasons into BAR-GHT (abbreviation for barriers to growth hormone therapy) after study completion.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Validity of the Growth Hormone Compliance Questionnaire child/adolescent version (GHCQ-CA) | The GHCQ-CA is a patient-reported outcome measure designed to assess reasons for non-adherence to growth hormone therapy in children/adolescents from the child's/adolescent's perspective. The GHCQ-CA currently has 34 items. All GHCQ-CA questions are answered on a 5-point Likert scale from 'Never' to 'Always'. | Day 1 | 
| Validity of the GHCQ-CA | The GHCQ-CA is a patient-reported outcome measure designed to assess reasons for non-adherence to growth hormone therapy in children/adolescents from the child's/adolescent's perspective. The GHCQ-CA currently has 34 items. All GHCQ-CA questions are answered on a 5-point Likert scale from 'Never' to 'Always'. | Day 14 | 
| Reliability of the GHCQ-CA | The GHCQ-CA is a patient-reported outcome measure designed to assess reasons for non-adherence to growth hormone therapy in children/adolescents from the child's/adolescent's perspective. The GHCQ-CA currently has 34 items. All GHCQ-CA questions are answered on a 5-point Likert scale from 'Never' to 'Always'. | Day 1 | 
| Reliability of the GHCQ-CA | The GHCQ-CA is a patient-reported outcome measure designed to assess reasons for non-adherence to growth hormone therapy in children/adolescents from the child's/adolescent's perspective. The GHCQ-CA currently has 34 items. All GHCQ-CA questions are answered on a 5-point Likert scale from 'Never' to 'Always'. | Day 14 | 
| Validity of the Growth Hormone Compliance Questionnaire parent version (GHCQ-P) | The GHCQ-P is a patient-reported outcome measure designed to assess reasons for non-adherence to growth hormone therapy in children/adolescents from the parents'/legal guardian's perspective. The GHCQ-P currently has 50 items. All GHCQ-P questions are answered on a 5-point Likert scale from 'Never' to 'Always'. | Day 1 | 
| Validity of the GHCQ-P | The GHCQ-P is a patient-reported outcome measure designed to assess reasons for non-adherence to growth hormone therapy in children/adolescents from the parents'/legal guardian's perspective. The GHCQ-P currently has 50 items. All GHCQ-P questions are answered on a 5-point Likert scale from 'Never' to 'Always'. | Day 14 | 
| Reliability of the GHCQ-P | The GHCQ-P is a patient-reported outcome measure designed to assess reasons for non-adherence to growth hormone therapy in children/adolescents from the parents'/legal guardian's perspective. The GHCQ-P currently has 50 items. All GHCQ-P questions are answered on a 5-point Likert scale from 'Never' to 'Always'. | Day 1 | 
| Reliability of the GHCQ-P | The GHCQ-P is a patient-reported outcome measure designed to assess reasons for non-adherence to growth hormone therapy in children/adolescents from the parents'/legal guardian's perspective. The GHCQ-P currently has 50 items. All GHCQ-P questions are answered on a 5-point Likert scale from 'Never' to 'Always'. | Day 14 |