Skeletal Muscle Effects of GH in Boys

NCT04020913 | Completed FDA Drug

• 2 other identifiers: #19-09U111112218502
• Study Type: observational
• Target: 45
• Locations: 1 country
• Sites: 1

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.

Conditions

Growth Hormone Deficiency; Idiopathic Short Stature

Keywords

GH; muscle strength; prepubertal; boys; short stature; GH treatment; muscle agility

Outcome Measures

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

  12 months

Secondary Outcomes (6)

• Skeletal muscle power (Watts)

  12 months

• Muscle agility (seconds)

  12 months

• Muscle endurance

  12 months

• Lean body mass accrual

  12 months

• Bone mineral density

  12 months

Study Arms (2)

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

Somatropin injection 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

Also known as: Recombinant Growth Hormone, Norditropin

Short Stature Boys

Eligibility Criteria

• Age: 6 Years - 11 Years
• Sex: male
• Healthy Volunteers: Yes
• Age Groups: Child (0-17)
• 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.

You may qualify if:

• Short Stature Group
• boys between 6-11 years of age
• Prepubertal
• Short stature (height ≤ -2SDS) due to either GH deficiency or idiopathic
• Stable treatment of other pituitary hormone deficiencies
• Naïve to GH therapy, or GH discontinued at least 6 months prior to study
• Normal Stature Group
• boys between 6-11 years of age
• Prepubertal
• Normal height (10th to 90th%)

You may not qualify if:

• Actively growing brain tumors
• Chronic medical conditions that could affect study outcomes
• Long-term steroid use
• Intense regular physical training programs or organized team sports

Sponsors & Collaborators

• Nemours Children's Clinic: lead
• Novo Nordisk A/S: collaborator

Study Sites (1)

Nemours Children's Clinic

Jacksonville, Florida, 32207, United States

Location

Biospecimen

Retention: SAMPLES WITHOUT DNA

Serum/plasma will be obtained.

MeSH Terms

Conditions

Dwarfism, Pituitary; Dwarfism

Interventions

Human Growth Hormone; Growth Hormone

Condition Hierarchy (Ancestors)

Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Bone Diseases, Endocrine; Hypopituitarism; Pituitary Diseases; Hypothalamic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Endocrine System Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Study Officials

• Nelly Mauras, MD

  Nemours Children's Clinic

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD

Study Record Dates

• First Submitted: May 24, 2019
• First Posted: July 16, 2019
• Study Start: July 22, 2019
• Primary Completion: September 30, 2025
• Study Completion: October 30, 2025
• Last Updated: November 4, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)