Clinical trial NCT02580032: 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)
Official title: 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 type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Growth Disorder
Growth Hormone Deficiency in Children
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2015-10-20
Enrollment: 252
Sex: ALL
Minimum age (years): 4
Maximum age (years): 13
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The 2 populations (child and parent/guardian) will each be divided into a Treatment Naïve group and a Maintenance group.

Brief summary: 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).


Eligibility criteria: 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

Primary outcomes:
measure: Changes in CGI (Clinician Global Impression Scale)
timeFrame: 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 outcomes:
measure: Changes in PGI (Patient Global Impression Scale)
timeFrame: After the physician scheduled MCID assessment visit (variable between week 3 and week 11) and week 12 follow-up visit after initiation of treatment
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Novo Nordisk Investigational Site
state: Arizona
city: Centennial
country: United States
facility: Novo Nordisk Investigational Site
state: Colorado
city: Wilmington
country: United States
facility: Novo Nordisk Investigational Site
state: Delaware
city: Margate
country: United States
facility: Novo Nordisk Investigational Site
state: Florida
city: Miami
country: United States
facility: Novo Nordisk Investigational Site
state: Florida
city: Tallahassee
country: United States
facility: Novo Nordisk Investigational Site
state: Florida
city: Atlanta
country: United States
facility: Novo Nordisk Investigational Site
state: Georgia
city: Wheaton
country: United States
facility: Novo Nordisk Investigational Site
state: Illinois
city: Iowa City
country: United States
facility: Novo Nordisk Investigational Site
state: Iowa
city: Louisville
country: United States
facility: Novo Nordisk Investigational Site
state: Kentucky
city: Boston
country: United States
facility: Novo Nordisk Investigational Site
state: Massachusetts
city: Saint Paul
country: United States
facility: Novo Nordisk Investigational Site
state: Minnesota
city: Saint Louis
country: United States
facility: Novo Nordisk Investigational Site
state: Missouri
city: Lebanon
country: United States
facility: Novo Nordisk Investigational Site
state: New Hampshire
city: Albany
country: United States
facility: Novo Nordisk Investigational Site
state: New York
city: Buffalo
country: United States
facility: Novo Nordisk Investigational Site
state: New York
city: Mineola
country: United States
facility: Novo Nordisk Investigational Site
state: New York
city: New York
country: United States
facility: Novo Nordisk Investigational Site
state: New York
city: Spring Valley
country: United States
facility: Novo Nordisk Investigational Site
state: New York
city: Cleveland
country: United States
facility: Novo Nordisk Investigational Site
state: Ohio
city: Columbus
country: United States
facility: Novo Nordisk Investigational Site
state: Ohio
city: Pittsburgh
country: United States
facility: Novo Nordisk Investigational Site
state: Pennsylvania
city: Dallas
country: United States
facility: Novo Nordisk Investigational Site
state: Texas
city: Dallas
country: United States
facility: Novo Nordisk Investigational Site
state: Texas
city: Birmingham
country: United Kingdom
facility: Novo Nordisk Investigational Site
city: Liverpool
country: United Kingdom
facility: Novo Nordisk Investigational Site
city: London
country: United Kingdom
facility: Novo Nordisk Investigational Site
city: Manchester
country: United Kingdom
facility: Novo Nordisk Investigational Site
Study officials:
affiliation: Novo Nordisk A/S
name: Global Clinical Registry (GCR, 1452)
role: STUDY_DIRECTOR
References:
citation: Brod M, Rasmussen MH, Alolga S, Beck JF, Bushnell DM, Lee KW, Maniatis A. Psychometric Validation of the Growth Hormone Deficiency-Child Treatment Burden Measure (GHD-CTB) and the Growth Hormone Deficiency-Parent Treatment Burden Measure (GHD-PTB). Pharmacoecon Open. 2023 Jan;7(1):121-138. doi: 10.1007/s41669-022-00373-z. Epub 2022 Oct 18.
citation: Brod M, Hojby Rasmussen M, Vad K, Alolga S, Bushnell DM, Bedoin J, Maniatis A. Psychometric Validation of the Growth Hormone Deficiency-Child Impact Measure (GHD-CIM). Pharmacoecon Open. 2021 Sep;5(3):505-518. doi: 10.1007/s41669-020-00252-5. Epub 2021 Jan 12.