Pediatric Growth Hormone Deficiency
Conditions
Keywords
Growth Hormone Deficiency, Pediatric Growth Hormone Deficiency, GHD, PGHD, VRS-317, Versartis, Growth disorder, Long acting growth hormone, Growth hormone, Weekly growth hormone dosing, Semi-monthly growth hormone dosing, Monthly growth hormone dosing, IGF-I, Short stature, Height velocity, Annual height velocity, Growth rate, Somavaratan
Brief summary
Protocol 13VR3 is is a multi-center, open-label study assessing long-term somavaratan administration. Patients will be monitored for safety throughout their participation in the study. Safety will be monitored by physical examination, inspection of injection sites, vital signs, clinical laboratory determinations (including fasting glucose, insulin, and lipids), 12-lead ECGs (for new treatment naïve subjects and subjects not previously exposed to somavaratan), PK/PD assessments, and immunogenicity assessments. Adverse events (AEs) and concomitant medications will be captured. AEs will be coded using CTCAE v 4.0. AEs will be coded using the MedDRA dictionary and CMs using the WHO Drug dictionary.
Detailed description
Protocol 13VR3 is a multi-center, open-label study assessing long-term somavaratan administration. It is open to subjects completing a somavaratan study in children with growth hormone deficiency (GHD), as well as up to 100 new treatment naïve subjects with GHD. All subjects will receive somavaratan 3.5 mg/kg twice monthly. The study will be conducted at approximately 70 Pediatric Endocrinology centers in the United States, Canada, and Europe.
Interventions
DRUGsomavaratan
Subcutaneous injection
Sponsors
Versartis Inc.
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
3 Years to No maximum
Healthy volunteers
No
Inclusion criteria
for Subjects Completing a Previous Somavaratan Study: 1. Completion of a somavaratan clinical study. 2. Willing and able to comply with all study procedures.
Exclusion criteria
for Subjects Completing a Previous somavaratan Study: 1. Withdrawal from a somavaratan clinical study. 2. Use of certain medications with potential to alter responses to the test product. 3. Presence of a significant medical condition. Inclusion Criteria for New Treatment Naïve Subjects: 1. Chronological Age ≥ 3.0 years. 2. Pre-pubertal status. 3. Diagnosis of GHD as documented by two or more GH stimulation test results ≤ 10.0 ng/mL. 4. Normal thyroid function at Screening Visit in subjects not being treated for hypothyroidism. 5. Normal adrenal function at Screening Visit or within 6 months of the Screening Visit, in subjects not being treated for adrenal insufficiency. Subjects with adrenal insufficiency must receive glucocorticoid treatment for a minimum of 4 weeks before study drug administration. 6. Pathology relating to cause of GHD must be stable for at least 6 months prior to screening. 7. Legally authorized representatives must be willing and able to give informed consent.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety | Up to 4 years | Safety assessments include recording of adverse events and concomitant medications, monitoring of injection sites, vital signs and clinical laboratory determinations. |
Other
| Measure | Time frame | Description |
|---|---|---|
| Evaluate the changes in pharmacodynamics (PD) responses, bone age, weight, height velocity, height standard deviation scores, metabolic parameters, pubertal development and anti-drug antibody responses during long-term somavaratan treatment | Up to 4 years | PK/PD peak and trough measurements will be assessed throughout the study with assessment of plasma VRS-317 concentrations and IGF-I and its binding proteins measured at pre-specified time points. |
Outcome results
None listed