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)

NCT ID: NCT02580032

Last Updated: 2020-09-25

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

252 participants

Study Classification

OBSERVATIONAL

Study Start Date

2015-10-05

Study Completion Date

2018-05-14

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).

Detailed Description

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Conditions

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Growth Disorder Growth Hormone Deficiency in Children

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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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.

No treatment given

Intervention Type 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.

No treatment given

Intervention Type 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.

No treatment given

Intervention Type OTHER

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.

No treatment given

Intervention Type OTHER

No treatment given.

Interventions

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No treatment given

No treatment given.

Intervention Type OTHER

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

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
Minimum Eligible Age

4 Years

Maximum Eligible Age

13 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novo Nordisk A/S

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Global Clinical Registry (GCR, 1452)

Role: STUDY_DIRECTOR

Novo Nordisk A/S

Locations

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Novo Nordisk Investigational Site

Phoenix, Arizona, United States

Site Status

Novo Nordisk Investigational Site

Centennial, Colorado, United States

Site Status

Novo Nordisk Investigational Site

Wilmington, Delaware, United States

Site Status

Novo Nordisk Investigational Site

Margate, Florida, United States

Site Status

Novo Nordisk Investigational Site

Miami, Florida, United States

Site Status

Novo Nordisk Investigational Site

Tallahassee, Florida, United States

Site Status

Novo Nordisk Investigational Site

Atlanta, Georgia, United States

Site Status

Novo Nordisk Investigational Site

Wheaton, Illinois, United States

Site Status

Novo Nordisk Investigational Site

Iowa City, Iowa, United States

Site Status

Novo Nordisk Investigational Site

Louisville, Kentucky, United States

Site Status

Novo Nordisk Investigational Site

Boston, Massachusetts, United States

Site Status

Novo Nordisk Investigational Site

Saint Paul, Minnesota, United States

Site Status

Novo Nordisk Investigational Site

St Louis, Missouri, United States

Site Status

Novo Nordisk Investigational Site

Lebanon, New Hampshire, United States

Site Status

Novo Nordisk Investigational Site

Albany, New York, United States

Site Status

Novo Nordisk Investigational Site

Buffalo, New York, United States

Site Status

Novo Nordisk Investigational Site

Mineola, New York, United States

Site Status

Novo Nordisk Investigational Site

New York, New York, United States

Site Status

Novo Nordisk Investigational Site

Spring Valley, New York, United States

Site Status

Novo Nordisk Investigational Site

Cleveland, Ohio, United States

Site Status

Novo Nordisk Investigational Site

Columbus, Ohio, United States

Site Status

Novo Nordisk Investigational Site

Pittsburgh, Pennsylvania, United States

Site Status

Novo Nordisk Investigational Site

Dallas, Texas, United States

Site Status

Novo Nordisk Investigational Site

Dallas, Texas, United States

Site Status

Novo Nordisk Investigational Site

Birmingham, , United Kingdom

Site Status

Novo Nordisk Investigational Site

Liverpool, , United Kingdom

Site Status

Novo Nordisk Investigational Site

London, , United Kingdom

Site Status

Novo Nordisk Investigational Site

Manchester, , United Kingdom

Site Status

Countries

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United States United Kingdom

References

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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.

Reference Type DERIVED
PMID: 36255609 (View on PubMed)

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.

Reference Type DERIVED
PMID: 33433896 (View on PubMed)

Related Links

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http://novonordisk-trials.com

Clinical Trials at Novo Nordisk

Other Identifiers

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U1111-1168-7911

Identifier Type: OTHER

Identifier Source: secondary_id

NN8640-4231

Identifier Type: -

Identifier Source: org_study_id

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