Clinical trial NCT04020913: Skeletal Muscle Effects of GH in Boys
Official title: Recombinant Human Growth Hormone (GH): Effects on Metabolic Profile, Body Composition and Skeletal Muscle Strength and Function in Pre-pubertal Short Boys With and Without GH Deficiency
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Growth Hormone Deficiency
Idiopathic Short Stature
Keywords: GH
muscle strength
prepubertal
boys
short stature
GH treatment
muscle agility
Lead sponsor: Nemours Children's Clinic
Sponsor class: OTHER
Collaborators: name: Novo Nordisk A/S
First posted: 2019-07-16
Enrollment: 45
Sex: MALE
Minimum age (years): 6
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: yes
Observational model: CASE_CONTROL
Time perspective: PROSPECTIVE
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.

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.

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

Primary outcomes:
measure: Skeletal muscle strength (Newtons)
timeFrame: 12 months
Secondary outcomes:
measure: Skeletal muscle power (Watts)
timeFrame: 12 months
measure: Muscle agility (seconds)
timeFrame: 12 months
measure: Muscle endurance
timeFrame: 12 months
measure: Lean body mass accrual
timeFrame: 12 months
measure: Bone mineral density
timeFrame: 12 months
measure: Resting energy expenditure
timeFrame: 12 months
Other outcomes:
Locations:
city: Jacksonville
country: United States
facility: Nemours Children's Clinic
state: Florida
Study officials:
affiliation: Nemours Children's Clinic
name: Nelly Mauras, MD
role: PRINCIPAL_INVESTIGATOR
References: