Growth, Growth Disorders, Growth Failure, Growth Hormone Treatment
Conditions
Keywords
Growth Hormone
Brief summary
A prospective, randomized, open-label single-blinded study of 50 subjects with growth hormone deficiency, ages 5 to 15 years in which 25 subjects will initiate rhGH therapy at 0.3mg/kg/week and the remaining 25 subjects will initiate their rhGH treatment at 0.2 mg/kg/week for the first 12 months of treatment. Safety parameters, height velocity, and adult height prediction by bone age determination will be assessed at 4-month intervals for 1 year following the initiation of rhGH therapy.
Detailed description
Investigators propose a prospective randomized, open-label single-blinded study of 50 subjects with growth hormone deficiency, ages 5 to 15 years. 25 subjects will be randomized to initiate a dose of 0.3 mg/kg/week (0.28-0.32 mg/kg/week) and the remaining 25 subjects will initiate their rhGH treatment at 0.2 mg/kg/week (0.18-0.22 mg/kg/week) for the first 12 months of treatment. Safety parameters, height velocity, and adult height prediction by bone age determination will be assessed at 4-month intervals for 1 year following the initiation of rhGH therapy.
Interventions
DRUGSomatropin
The treatment of children with subcutaneous recombinant human growth hormone (rhGH) is the current gold standard of care for children with diagnosed GHD. This study will serve only to investigate the optimal dose of treatment in the first year of treatment with rhGH.
Sponsors
Northwell Health
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
SINGLE (Subject)
Eligibility
Sex/Gender
ALL
Age
5 Years to 15 Years
Healthy volunteers
Yes
Inclusion criteria
1. Provision of signed and dated informed consent form 2. Stated willingness to comply with all study procedures and availability for the duration of the study 3. Male or female, aged 5-15 years 4. In good general health as evidenced by medical history or diagnosed with growth hormone deficiency 5. Ability to take subcutaneous GH injections nightly
Exclusion criteria
Subjects will be excluded if they have GH resistance, or syndromic short stature such as Prader Willi syndrome and Turner syndrome. Patients will also be excluded if they have active malignancies, or systemic illnesses such as heart failure, kidney failure, or liver failure.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Annualized Growth Velocity (GV) in the first year after treatment | I year | Height (cm) at 0- month visit; height (cm) at 12-month visit |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| IGF1- 0,4,8,12 months | 12 Months | IGF-1 will be collected at timepoints - 0,4,8,12 months |
| IGFBP3 - 0,4,8,12 months | 12 Months | IGFBP3 will be collected at timepoints - 0,4,8,12 months |
| HbA1c - 4,8,12 months | 12 Months | HbA1c will be collected at timepoints 4,8,12 months |
| Total T4 or Free T4 - 0,12 months | 12 Months | Total T4 or Free T4 will be collected at timepoints 0,12 months |
| TSH - 0, 12 months | 12 Months | TSH will be collected at timepoints 0, 12 months |
Countries
United States
Contacts
Primary ContactJennifer Apsan, MD
Backup ContactRashida Talib, MPH
Outcome results
None listed