Validation of Two Measures for Growth Hormone Deficiency in Children, the Treatment Related Impact Measure of Childhood Growth Hormone Deficiency (TRIM-CGHD) and the Treatment Burden Measure of Childhood Growth Hormone Deficiency (TB-CGHD)

Study Overview
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Brief Summary
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This study is conducted in Europe and the United States of America (USA). The aim of the study is to validate two measures for growth hormone deficiency in children, the Treatment Related Impact Measure of Childhood Growth Hormone Deficiency (TRIM-CGHD) and the Treatment Burden Measure of Childhood Growth Hormone Deficiency (TB-CGHD).

Official Title
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Validation of Two Measures for Growth Hormone Deficiency in Children, the Treatment Related Impact Measure of Childhood Growth Hormone Deficiency (TRIM-CGHD) and the Treatment Burden Measure of Childhood Growth Hormone Deficiency (TB-CGHD)

Conditions
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Growth Disorder, 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: Informed consent obtained Child population - treatment näive: Confirmed diagnosis of Growth Hormone Deficiency (GHD) prior to enrolment as determined by a Growth Hormone (GH) stimulation test, defined as a peak GH level of 7.0 ng/ml or less. The GH stimulation test will be according to local clinical standards Pre pubertal children age 9 to less than 13 years at enrolment No prior exposure to GH therapy (GH-treatment naïve) Annualized height velocity (HV) below the 25th percentile for Chronological Age (CA) (HV less than -0.7 SD scores) and sex according to the standards of Prader et al (1989) Body Mass Index (BMI) percentile greater than 5th and below 95th percentile according to Centers for Disease Control and Prevention (CDC) BMI-for-age growth charts Child population - maintenance patients: Confirmed diagnosis of GHD prior to enrolment as determined by a GH stimulation test, defined as a peak GH level of 10.0 ng/ml or less. The GH stimulation test will be according to local clinical standards Pre pubertal children age 9 to less than 13 years at enrolment Body Mass Index (BMI) percentile greater than 5th and below 95th percentile according to Centers for Disease Control and Prevention (CDC) BMI-for-age growth charts Parent/Guardian population - treatment näive: Parent/Guardian of child with a confirmed diagnosis of GHD prior to enrolment as determined by a GH stimulation test, defined as a peak GH level of 7.0 ng/ml or less. The GH stimulation test will be according to local clinical standards Parent/Guardian of pre pubertal child age 4 to less than 9 years at enrolment Parent/Guardian of child with no prior exposure to GH therapy (GH-treatment naïve) Parent/Guardian of child with annualized height velocity (HV) below the 25th percentile for CA (HV less than -0.7 SD scores) and sex according to the standards of Prader et al (1989) Parent/Guardian of child with Body Mass Index (BMI) percentile greater than 5th and below 95th percentile according to Centers for Disease Control and Prevention (CDC) BMI-for-age growth charts Parent/Guardian living in the same residence as the child at least 50% of the time Parent/Guardian population - maintenance patients: Parent/Guardian of child with confirmed diagnosis of GHD prior to enrolment as determined by a GH stimulation test, defined as a peak GH level of 10.0 ng/ml or less. The GH stimulation test will be according to local clinical standards Parent/Guardian of pre pubertal child age 4 to less than 9 years at enrolment Parent/Guardian of child with Body Mass Index (BMI) percentile greater than 5th and below 95th percentile according to Centers for Disease Control and Prevention (CDC) BMI-for-age growth charts Parent/Guardian living in the same residence as the child at least 50% of the time Exclusion Criteria: Child population - treatment näive and maintenance patients: Any clinically significant abnormality likely to affect growth or the ability to evaluate growth: a) Chromosomal abnormalities and medical syndromes, e.g. but not limited to Turner's syndrome, Laron syndrome, Noonan syndrome, or absence of GH receptors b) Congenital abnormalities (causing skeletal abnormalities), e.g. but not limited to Russell-Silver Syndrome, skeletal dysplasia's c) Significant spinal abnormalities including scoliosis, kyphosis and spina bifida variants - Children born small for gestational age (SGA - birth weight and/or birth length less than -2 SD for gestational age) Children diagnosed with diabetes mellitus or fasting blood glucose greater than or equal to 126 mg/dl (7.0 mmol/L), or HbA1c greater than or equal to 6.5% at enrolment Current inflammatory diseases (e.g. but not limited to arthritis, inflammatory bowel diseases) requiring systemic corticosteroid treatment or glucocorticoids treatment for longer than 2 weeks within the last 3 months prior to enrolment Children requiring glucocorticoid therapy (e.g. asthma) who are taking a dose of greater than 400 µg/day of inhaled budesonide or equivalents for longer than 1 month the year prior to enrolment Concomitant administration of other treatments that may have an effect on growth, e.g. but not limited to anabolic steroids and methylphenidate for attention deficit hyperactivity disorder (ADHD). Hormone replacement therapies (thyroxin, hydrocortisone, desmopressin) are allowed for inclusion Any disorder which, in the opinion of the investigator, might jeopardise subject's safety or compliance with the protocol The subject and/or the parent/Legally Acceptable Representative (LAR) are likely to be non-compliant in respect to trial conduct, as judged by the investigator Parent/Guardian population - treatment näive and maintenance patients: Parent/Guardian of child with any clinically significant abnormality likely to affect growth or the ability to evaluate growth: a) Chromosomal abnormalities and medical syndromes, e.g. but not limited to Turner's syndrome, Laron syndrome, Noonan syndrome, or absence of GH receptors b) Congenital abnormalities (causing skeletal abnormalities), e.g. but not limited to Russell-Silver Syndrome, skeletal dysplasia's c) Significant spinal abnormalities including scoliosis, kyphosis and spina bifida variants - Parent/Guardian of child born small for gestational age (SGA - birth weight and/or birth length less than -2 SD for gestational age) Parent/Guardian of child diagnosed with diabetes mellitus or fasting blood glucose greater than or equal to 126 mg/dl (7.0 mmol/L), or HbA1c greater than or equal to 6.5% at enrolment Parent/Guardian of child with current inflammatory diseases (e.g. but not limited to arthritis, inflammatory bowel diseases) requiring systemic corticosteroid treatment or glucocorticoids treatment for longer than 2 weeks within the last 3 months prior to enrolment Parent/Guardian of children requiring glucocorticoid therapy (e.g. asthma) who are taking a dose of greater than 400 µg/day of inhaled budesonide or equivalents for longer than 1 month the year prior to enrolment Parent/Guardian of child with concomitant administration of other treatments that may have an effect on growth, e.g. but not limited to anabolic steroids and methylphenidate for attention deficit hyperactivity disorder (ADHD). Hormone replacement therapies (thyroxin, hydrocortisone, desmopressin) are allowed for inclusion Parent/Guardian of child with any disorder which, in the opinion of the investigator, might jeopardise subject's safety or compliance with the protocol The subject and/or the parent/LAR are likely to be non-compliant in respect to trial conduct, as judged by the investigator

Ages Eligible for Study
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Minimum Age: 4 Years
Maximum Age: 13 Years

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 |
| --- | --- |
| Child Treatment Naïve group (Group A)<br>Pre-pubertal boy or girl, ages of 9 to 13 years with a confirmed diagnosis of GHD prior to enrolment as determined by one GH stimulation test, defined as a peak GH level of equal or below 7.0 ng/ml. | Other: No treatment given<br>* No treatment given.<br>|
| Child Maintenance group (Group B)<br>Pre-pubertal boy or girl, ages of 9 to 13 years with a confirmed diagnosis of GHD prior to enrolment as determined by one GH stimulation test, defined as a peak GH level of equal or below 10.0 ng/ml who have been taking prescription treatment for GHD for 6 months or more. | Other: No treatment given<br>* No treatment given.<br>|
| Parent Treatment Naïve group (Group C)<br>Parents/guardians, who live with a pre-pubertal boy or girl, age 4 to 9 years with a confirmed diagnosis of GHD prior to enrolment as determined by one GH stimulation test, defined as a peak GH level of below or equal to 7.0 ng/ml. | Other: No treatment given<br>* No treatment given.<br>|
| Parent Maintenance group (Group D)<br>Parents/guardians, who live with a pre-pubertal boy or girl, age 4 to 9 years with a confirmed diagnosis of GHD prior to enrolment as determined by one GH stimulation test, defined as a peak GH level of below or equal to 10.0 ng/ml who have been taking prescription treatment for GHD for 6 months or more. | Other: No treatment given<br>* No treatment given.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Changes in CGI (Clinician Global Impression Scale) |  | After the physician scheduled MCID assessment visit (variable between week 3 and week 11) and week 12 follow-up visit after initiation of treatment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Changes in PGI (Patient Global Impression Scale) |  | After the physician scheduled MCID assessment visit (variable between week 3 and week 11) and week 12 follow-up visit after initiation of treatment |