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)

Growth Hormone Deficiency in Children Clinical Trial

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02580032
• Other study ID #: NN8640-4231
• Secondary ID: U1111-1168-7911
• Status: Completed
• Start date: October 5, 2015
• Est. completion date: May 14, 2018

Study information

• Verified date: September 2020
• Source: Novo Nordisk A/S
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 252
• Est. completion date: May 14, 2018
• Est. primary completion date: May 14, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Years to 13 Years

Eligibility

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

Related Conditions & MeSH terms

• Dwarfism, Pituitary
• Endocrine System Diseases
• Growth Disorder
• Growth Disorders
• Growth Hormone Deficiency in Children

Intervention

Other:
• No treatment given
No treatment given.

Locations

Country	Name	City	State
United Kingdom	Novo Nordisk Investigational Site	Birmingham
United Kingdom	Novo Nordisk Investigational Site	Liverpool
United Kingdom	Novo Nordisk Investigational Site	London
United Kingdom	Novo Nordisk Investigational Site	Manchester
United States	Novo Nordisk Investigational Site	Albany	New York
United States	Novo Nordisk Investigational Site	Atlanta	Georgia
United States	Novo Nordisk Investigational Site	Boston	Massachusetts
United States	Novo Nordisk Investigational Site	Buffalo	New York
United States	Novo Nordisk Investigational Site	Centennial	Colorado
United States	Novo Nordisk Investigational Site	Cleveland	Ohio
United States	Novo Nordisk Investigational Site	Columbus	Ohio
United States	Novo Nordisk Investigational Site	Dallas	Texas
United States	Novo Nordisk Investigational Site	Dallas	Texas
United States	Novo Nordisk Investigational Site	Iowa City	Iowa
United States	Novo Nordisk Investigational Site	Lebanon	New Hampshire
United States	Novo Nordisk Investigational Site	Louisville	Kentucky
United States	Novo Nordisk Investigational Site	Margate	Florida
United States	Novo Nordisk Investigational Site	Miami	Florida
United States	Novo Nordisk Investigational Site	Mineola	New York
United States	Novo Nordisk Investigational Site	New York	New York
United States	Novo Nordisk Investigational Site	Phoenix	Arizona
United States	Novo Nordisk Investigational Site	Pittsburgh	Pennsylvania
United States	Novo Nordisk Investigational Site	Saint Louis	Missouri
United States	Novo Nordisk Investigational Site	Saint Paul	Minnesota
United States	Novo Nordisk Investigational Site	Spring Valley	New York
United States	Novo Nordisk Investigational Site	Tallahassee	Florida
United States	Novo Nordisk Investigational Site	Wheaton	Illinois
United States	Novo Nordisk Investigational Site	Wilmington	Delaware

Sponsors (1)

Lead Sponsor	Collaborator
Novo Nordisk A/S

Countries where clinical trial is conducted

United States,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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

External Links

•   Clinical Trials at Novo Nordisk