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).

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

252

participants targeted

Target at P75+ for all trials

Timeline

Completed

Started Oct 2015

Typical duration for all trials

Geographic Reach

2 countries

28 active sites

Status

completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

2.6 years study duration

Study Start

First participant enrolled

October 5, 2015

Completed

11 days until next milestone

First Submitted

Initial submission to the registry

October 16, 2015

Completed

4 days until next milestone

First Posted

Study publicly available on registry

October 20, 2015

Completed

2.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 14, 2018

Completed

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

May 14, 2018

Completed

Last Updated

September 25, 2020

Status Verified

September 1, 2020

Enrollment Period

2.6 years

First QC Date

October 16, 2015

Last Update Submit

September 24, 2020

Conditions

Growth Disorder Growth Hormone Deficiency in Children

Outcome Measures

Primary Outcomes (1)

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 Outcomes (1)

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

Study Arms (4)

Child Treatment Naïve group (Group A)

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

Child Maintenance group (Group B)

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

Parent Treatment Naïve group (Group C)

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

Parent Maintenance group (Group D)

Other: No treatment given

Interventions

No treatment givenOTHER

No treatment given.

Child Maintenance group (Group B)Child Treatment Naïve group (Group A)Parent Maintenance group (Group D)Parent Treatment Naïve group (Group C)

Eligibility Criteria

Age4 Years - 13 Years

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17)

Sampling MethodNon-Probability Sample

Study Population

The 2 populations (child and parent/guardian) will each be divided into a Treatment Naïve group and a Maintenance group.

You may qualify if:

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)
+7 more criteria

You may not qualify if:

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
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)
+5 more criteria

Contact the study team to confirm eligibility.