Skeletal Muscle Effects of GH in Boys

Active, not recruiting

• Conditions: Growth Hormone Deficiency; Idiopathic Short Stature
• Interventions: Drug: Somatropin injection

• Registration Number: NCT04020913
• Lead Sponsor: Nemours Children's Clinic

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.

Study Timeline

• Study First Submitted: 2019-05-24
• Study First Submitted QC: 2019-07-12
• Study First Posted: 2019-07-16
• Study Start: 2019-07-22
• Status Verified: 2024-11
• Last Update Submitted: 2024-11-12
• Last Update Posted: 2024-11-14
• Primary Completion: 2025-03-31
• Study Completion: 2025-07-21

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: Male
• Target Recruitment: 45

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%)

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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

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Short Stature Boys	Somatropin injection	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.

Primary Outcome Measures

Name	Time	Method
Skeletal muscle strength (Newtons)	12 months	Upper and lower body muscle strength will be assessed using a handheld dynamometer for manual muscle testing of peak force

Secondary Outcome Measures

Name	Time	Method
Lean body mass accrual	12 months	Assessed by dual energy x-ray absorptiometry (DEXA) scan of the whole body
Muscle endurance	12 months	Upper body muscular endurance will be assessed using a modified push up test for repetitions
Skeletal muscle power (Watts)	12 months	Lower extremity power will be assessed by vertical jump performed using a Power time mat
Muscle agility (seconds)	12 months	Muscle agility will be assessed by a timed shuttle run
Resting energy expenditure	12 months	Indirect calorimetry will be performed after overnight fast
Bone mineral density	12 months	Assessed by DEXA scan of the lumbar spine and whole body

Trial Locations

Locations (1)

Nemours Children's Clinic; 🇺🇸 Jacksonville, Florida, United States