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Skeletal Muscle Effects of GH in Boys

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ClinicalTrials.gov Identifier: NCT04020913
Recruitment Status : Not yet recruiting
First Posted : July 16, 2019
Last Update Posted : July 16, 2019
Sponsor:
Collaborator:
Novo Nordisk A/S
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment
Growth Hormone Deficiency Idiopathic Short Stature Drug: Somatropin injection

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.


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Study Type : Observational
Estimated Enrollment : 45 participants
Observational Model: Case-Control
Time Perspective: Prospective
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
Estimated Study Start Date : July 22, 2019
Estimated Primary Completion Date : July 21, 2022
Estimated Study Completion Date : July 21, 2022


Group/Cohort Intervention/treatment
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

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
Other Names:
  • Recombinant Growth Hormone
  • Norditropin

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.



Primary Outcome Measures :
  1. Skeletal muscle strength (Newtons) [ Time Frame: 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 :
  1. Skeletal muscle power (Watts) [ Time Frame: 12 months ]
    Lower extremity power will be assessed by vertical jump performed using a Power time mat

  2. Muscle agility (seconds) [ Time Frame: 12 months ]
    Muscle agility will be assessed by a timed shuttle run

  3. Muscle endurance [ Time Frame: 12 months ]
    Upper body muscular endurance will be assessed using a modified push up test for repetitions

  4. Lean body mass accrual [ Time Frame: 12 months ]
    Assessed by DEXA scan of the whole body

  5. Bone mineral density [ Time Frame: 12 months ]
    Assessed by DEXA scan of the lumbar spine and whole body

  6. Resting energy expenditure [ Time Frame: 12 months ]
    Indirect calorimetry will be performed after overnight fast


Biospecimen Retention:   Samples Without DNA
Serum/plasma will be obtained.


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Ages Eligible for Study:   6 Years to 11 Years   (Child)
Sexes Eligible for Study:   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.

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04020913


Contacts
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Contact: Nelly Mauras, MD 904-697-3674 nmauras@nemours.org
Contact: Ashish Malpani, MD 904-755-3542 ashish.malpani@nemours.org

Locations
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United States, Florida
Nemours Children's Clinic
Jacksonville, Florida, United States, 32207
Sponsors and Collaborators
Nemours Children's Clinic
Novo Nordisk A/S
Investigators
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Principal Investigator: Nelly Mauras, MD Nemours Children's Clinic

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Responsible Party: Nemours Children's Clinic
ClinicalTrials.gov Identifier: NCT04020913     History of Changes
Other Study ID Numbers: #19-09
U111112218502 ( Other Grant/Funding Number: Novo Nordisk )
First Posted: July 16, 2019    Key Record Dates
Last Update Posted: July 16, 2019
Last Verified: May 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Keywords provided by Nemours Children's Clinic:
GH
muscle strength
prepubertal
boys
short stature
GH treatment
muscle agility
Additional relevant MeSH terms:
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Dwarfism
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Genetic Diseases, Inborn
Endocrine System Diseases
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs