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CompletedNCT04020913Updated Nov 4, 2025

Skeletal Muscle Effects of GH in Boys

An observational study in Growth Hormone Deficiency and Idiopathic Short Stature, sponsored by Nemours Children's Clinic. Completed at 1 site in United States. Open to male participants aged 6 Years to 11 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2025-11-04.

Sponsored by Nemours Children's Clinic · Observational

Study type
Observational
Model
Case-control
Time perspective
Prospective
Enrollment
45
Ages
6 Years to 11 Years
Sex
Male
01

Study 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.

Read the 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.

02

Conditions studied

  • Growth Hormone Deficiency
  • Idiopathic Short Stature

Keywords

  • GH
  • muscle strength
  • prepubertal
  • boys
  • short stature
  • GH treatment
  • muscle agility
03

Who can participate

Ages eligible
6 Years to 11 Years
Sexes eligible
Male
Accepts healthy volunteers
Yes
Sampling method
Non-probability sample

Study population

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

Normally Statured Boys: A group of 15 healthy, normally statured (between 10th- 90th %), age-matched boys not on somatropin, preferably siblings (although not exclusively) will be studied similarly.

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

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
04

Study design

Observational model
Case-control
Time perspective
Prospective
Enrollment
45 participants (actual)
Patient registry
No
Biospecimen retention
Samples without dna

Groups and cohorts

  • 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.

    Drug: Somatropin injection

  • 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.

Interventions

  • DrugSomatropin 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

    Also known as: Recombinant Growth Hormone, Norditropin

05

What researchers measure

Primary outcomes

  1. Skeletal muscle strength (Newtons)

    Upper and lower body muscle strength will be assessed using a handheld dynamometer for manual muscle testing of peak force

    Time frame: 12 months

Secondary outcomes

  1. Skeletal muscle power (Watts)

    Lower extremity power will be assessed by vertical jump performed using a Power time mat

    Time frame: 12 months

  2. Muscle agility (seconds)

    Muscle agility will be assessed by a timed shuttle run

    Time frame: 12 months

  3. Muscle endurance

    Upper body muscular endurance will be assessed using a modified push up test for repetitions

    Time frame: 12 months

  4. Lean body mass accrual

    Assessed by dual energy x-ray absorptiometry (DEXA) scan of the whole body

    Time frame: 12 months

  5. Bone mineral density

    Assessed by DEXA scan of the lumbar spine and whole body

    Time frame: 12 months

  6. Resting energy expenditure

    Indirect calorimetry will be performed after overnight fast

    Time frame: 12 months

06

Study locations

1 site
  • Nemours Children's Clinic
    Jacksonville, Florida 32207, United States
07

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

08

Registry details

Key details

Study ID
NCT04020913
Lead sponsor
Nemours Children's Clinic
Collaborators
Novo Nordisk A/S
Responsible party
Nelly Mauras (MD, Nemours Children's Clinic) — Principal investigator
First posted
Jul 16, 2019
Start date
Jul 22, 2019
Primary completion
Sep 30, 2025
Completion
Oct 30, 2025
Last update
Nov 4, 2025

Study contacts

Nelly Mauras, MD
principal investigator · Nemours Children's Clinic

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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