Growth hormone deficiency small stature
Conditions
Interventions
Subjects will receive commercial available recombinant human growth hormone
daily ór somavaratan every 15 days during 1 year.
child
deficiency
growth hormone
Sponsors
Versartis Inc.
Eligibility
Age
2 Years to 11 Years
Inclusion criteria
Inclusion criteria: 1. Chronological Age >= 3.0 years and = 10th percentile.;6. IGF-I SD score = 6 months as determined by the central reader). Left hand X-Ray must be obtained within 90 days of screening visit or during screening.;8. Normal thyroid function test results at screening visit (or a minimum of four weeks of thyroxine replacement therapy prior to study drug administration).;9. Available adrenal function test results at screening visit (or in the preceding 6 months) in all subjects without a minimum of four weeks glucocorticoid replacement therapy prior to study drug administration. ;10. Pathology relating to cause of GH deficiency must be stable for at least 6 months prior to screening.;11. Legally authorized representatives must be willing and able to give informed;consent.
Exclusion criteria
Exclusion criteria: 1. Prior treatment with any growth promoting agent (e.g., GH, IGF-I, GH releasing hormone (GHRH), gonadotrophins, sex steroids). Up to 10 day exposures to a growth promoting agent for diagnostic purposes are permitted if administered 30 or more days prior to screening. ;2. Documented history of, or current, significant disease.;3. Chromosomal aneuploidy, significant gene mutations (other than those that cause GHD) or confirmed diagnosis of a named syndrome.;4. Birth weight and/or birth length less than 5th percentile for gestational age using gestational age growth charts.;5. A diagnosis of Attention Deficit Hyperactivity Disorder (ADHD), use of ADHD;medications or a likelihood of starting ADHD medications during study participation.;6. Daily use of anti-inflammatory doses of glucocorticoid.;7. Prior history of leukemia, lymphoma, sarcoma or cancer.;8. Treatment with an investigational drug in the 30 days prior to screening.;9. Known allergy to constituents of the study drug formulation.;10. Ocular findings suggestive of increased intracranial pressure and/or retinopathy at screening.;11. Significant spinal abnormalities including scoliosis, kyphosis and spina bifida;variants.;12. Significant abnormality in screening laboratory studies.;13. Current social conditions which would prevent completion of study activities;(e.g., planned family move to a distant location).;14. History of pancreatitis or undiagnosed chronic abdominal pain.;15. History of spinal or total body irradiation.;16. Subjects with other pituitary hormone deficiency who are not treated properly.;17. Unwillingness to provide consent for participation in all trial activities.;18. Unwillingness to accept dose assignments.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary efficacy endpoint - Annual height velocity (cm/yr) after 12 months of treatment with either somavaratan, or daily rhGH | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary efficacy endpoints -IGF-I and IGFBP-3 responses to study drug administration -changes in height standard deviation score, body weight, body mass index, bone age | — |
Countries
The Netherlands
Outcome results
None listed