Growth hormone deficiency MedDRA version: 19.0 Level: PT Classification code 10056438 Term: Growth hormone deficiency System Organ Class: 10014698 - Endocrine disorders
Conditions
Interventions
Product Name: Somavaratan (VRS-317)
Pharmaceutical Form: Injection
INN or Proposed INN: VRS-317
CAS Number: 1448335-08-7
Other descriptive name: VRS-317
Concentration unit: µg/kg microgram(s)/kilogram
Sponsors
Versartis, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Chronological Age = 3.0 years and = 10.0 (girls) and = 11.0 (boys). 2. Pre-pubertal status: Absent breast development in girls, testicular volume =65 years) no F.1.3.1 Number of subjects for this age range
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 |
|---|---|
| Main Objective: Compare the safety and efficacy of subcutaneous VRS-317 and daily rhGH during 12 months of treatment.;Secondary Objective: Evaluate and compare changes in pharmacodynamic responses (IGF-I, IGF binding protein-3 (IGFBP-3), growth hormone binding protein (GHBP) and acid labile subunit (ALS)), bone age, weight, body mass index, height standard deviation scores, pubertal development and anti-drug antibody responses.;Primary end point(s): Annual height velocity after 12 months continous treatment with either VRS-317 or daily rhGH.;Timepoint(s) of evaluation of this end point: End of study. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - IGF-1 and IGFBP-3 responses to study drug administration. - Change in height SDS. - Change in bone age. - Change in body weight. - Change in body mass index.;Timepoint(s) of evaluation of this end point: End of study. | — |
Countries
Belgium, Canada, Netherlands, Poland, Sweden, United States
Contacts
Public ContactGlobal Project Manager
ResearchPoint Global
Outcome results
None listed