Skeletal Muscle Effects of GH in Boys

NCT ID: NCT04020913

Last Updated: 2025-11-04

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 45 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2019-07-22
• Study Completion Date: 2025-10-30

Brief Summary

The purpose of the study is to measure the functional effects of recombinant GH in skeletal muscle, in addition to growth promotion, in short prepubertal boys with either growth hormone deficiency or idiopathic short stature. Patients will be similarly short. The investigators will also compare these values in the short stature cohort to those obtained in testing performed in normally growing age-matched healthy control boys not on GH. The group on GH will be studied before and after 6 and 12 months of GH treatment.

Detailed Description

Prepubertal boys with significant short stature (height SDS ≤-2.0) diagnosed with either GH deficiency or idiopathic short stature (ISS) who are identified as candidates for GH treatment will recruited. Subjects will have a battery of studies to assess skeletal muscle strength, agility, power and endurance, as well as assessment of body composition and energy expenditure before and after GH administration. GH (Somatropin) treatment at standard doses will be given as daily injections at bedtime. Subjects will be followed at three-month intervals per the clinical routine, when anthropometric measurements will be obtained. Baseline studies will be repeated at 6 and 12 months from initiation of treatment, each patient will serve as his own control pre and post GH.

A group of normally statured healthy boys will have the same testing as the study patients but without GH treatment to assess the impact of natural growth on the muscle measures above.

Conditions

Growth Hormone Deficiency; Idiopathic Short Stature

Study Design

• Observational Model Type: CASE_CONTROL
• Study Time Perspective: PROSPECTIVE

Study Groups

Short Stature Boys

Prepubertal boys with short stature defined as a height ≤-2 SDS with either GH deficiency (defined as peak GH responses to pharmacologic stimuli \<10ng/ml) or idiopathic short stature (i.e., no identifiable pathology) will be studied pre and post 12 months of GH therapy.

Somatropin injection

Intervention Type: DRUG

Boys with short stature will be studied for measures of:

1. skeletal muscle strength, power, and endurance

2. muscle agility

3. lean body mass accrual

4. bone mineral density and resting energy expenditure before and after 6 months and 12 months of GH administration

Normally Statured Boys

A group of 15 healthy, normally statured (between 10th- 90th %), age-matched boys not on Growth Hormone replacement, preferably siblings (although not exclusively), will be recruited to serve as healthy controls.

No interventions assigned to this group

Interventions

Somatropin injection

Boys with short stature will be studied for measures of:

1. skeletal muscle strength, power, and endurance

2. muscle agility

3. lean body mass accrual

4. bone mineral density and resting energy expenditure before and after 6 months and 12 months of GH administration

Intervention Type: DRUG

Other Intervention Names

Recombinant Growth Hormone; Norditropin

Eligibility Criteria

Inclusion Criteria

Short Stature Group

1. 30 boys between 6-11 years of age

2. Prepubertal

3. Short stature (height ≤ -2SDS) due to either GH deficiency or idiopathic

4. Stable treatment of other pituitary hormone deficiencies

5. Naïve to GH therapy, or GH discontinued at least 6 months prior to study

Normal Stature Group

1. 15 boys between 6-11 years of age

2. Prepubertal

3. Normal height (10th to 90th%)

Exclusion Criteria

1. Actively growing brain tumors

2. Chronic medical conditions that could affect study outcomes

3. Long-term steroid use

4. Intense regular physical training programs or organized team sports

• Minimum Eligible Age: 6 Years
• Maximum Eligible Age: 11 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: Yes

Sponsors

Novo Nordisk A/S

INDUSTRY

Sponsor Role: collaborator

Nemours Children's Clinic

OTHER

Sponsor Role: lead

Responsible Party

Nelly Mauras

MD

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Nelly Mauras, MD

Role: PRINCIPAL_INVESTIGATOR

Nemours Children's Clinic

Locations

Nemours Children's Clinic

Jacksonville, Florida, United States

Countries

United States

Other Identifiers

U111112218502

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

#19-09

Identifier Type: -

Identifier Source: org_study_id