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)

September 24, 2020 updated by: Novo Nordisk A/S
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).

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Actual)

252

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United Kingdom
      • Birmingham, United Kingdom, B4 6NH
        • Novo Nordisk Investigational Site
      • Liverpool, United Kingdom, L12 2AP
        • Novo Nordisk Investigational Site
      • London, United Kingdom, WC1N 3JH
        • Novo Nordisk Investigational Site
      • Manchester, United Kingdom, M13 9WL
        • Novo Nordisk Investigational Site
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85054
        • Novo Nordisk Investigational Site
    • Colorado
      • Centennial, Colorado, United States, 80112
        • Novo Nordisk Investigational Site
    • Delaware
      • Wilmington, Delaware, United States, 19803
        • Novo Nordisk Investigational Site
    • Florida
      • Margate, Florida, United States, 33063
        • Novo Nordisk Investigational Site
      • Miami, Florida, United States, 33155
        • Novo Nordisk Investigational Site
      • Tallahassee, Florida, United States, 32308
        • Novo Nordisk Investigational Site
    • Georgia
      • Atlanta, Georgia, United States, 30342-1551
        • Novo Nordisk Investigational Site
    • Illinois
      • Wheaton, Illinois, United States, 60187
        • Novo Nordisk Investigational Site
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • Novo Nordisk Investigational Site
    • Kentucky
      • Louisville, Kentucky, United States, 40202
        • Novo Nordisk Investigational Site
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Novo Nordisk Investigational Site
    • Minnesota
      • Saint Paul, Minnesota, United States, 55102
        • Novo Nordisk Investigational Site
    • Missouri
      • Saint Louis, Missouri, United States, 63017
        • Novo Nordisk Investigational Site
    • New Hampshire
      • Lebanon, New Hampshire, United States, 03756-1000
        • Novo Nordisk Investigational Site
    • New York
      • Albany, New York, United States, 12208-3412
        • Novo Nordisk Investigational Site
      • Buffalo, New York, United States, 14203
        • Novo Nordisk Investigational Site
      • Mineola, New York, United States, 11501
        • Novo Nordisk Investigational Site
      • New York, New York, United States, 10029
        • Novo Nordisk Investigational Site
      • Spring Valley, New York, United States, 10977
        • Novo Nordisk Investigational Site
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Novo Nordisk Investigational Site
      • Columbus, Ohio, United States, 43235
        • Novo Nordisk Investigational Site
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15218
        • Novo Nordisk Investigational Site
    • Texas
      • Dallas, Texas, United States, 75230
        • Novo Nordisk Investigational Site
      • Dallas, Texas, United States, 75235
        • Novo Nordisk Investigational Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

4 years to 13 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

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.

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
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
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
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
No treatment given.
Parent Maintenance group (Group D)
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
No treatment given.

What is the study measuring?

Primary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Novo Nordisk A/S

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 5, 2015

Primary Completion (Actual)

May 14, 2018

Study Completion (Actual)

May 14, 2018

Study Registration Dates

First Submitted

October 16, 2015

First Submitted That Met QC Criteria

October 16, 2015

First Posted (Estimate)

October 20, 2015

Study Record Updates

Last Update Posted (Actual)

September 25, 2020

Last Update Submitted That Met QC Criteria

September 24, 2020

Last Verified

September 1, 2020

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NN8640-4231
  • U1111-1168-7911 (Other Identifier: WHO)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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