Growth Disorders, Pediatric Growth Hormone Deficiency
Conditions
Keywords
GHD
Brief summary
The trial will consist of three stages: 1) a 30 day Phase 2 PK and PD evaluation of somavaratan, 2) an optional Phase 2 Extension and 3) a 12 month Phase 3 safety and efficacy stage. Upon completion of the PK/PD stage, the PK/PD profiles for the GHD children in this study will be compared to the PK/PD profiles for the GHD children treated in the Western study Phase 1b/2a study (Protocol 12VR2) and identify the somavaratan dose to be used in the Phase 3 stage in Japan. The Phase 3 stage will continue dosing for 12 months to obtain safety and efficacy data on 48 subjects.
Detailed description
The trial will consist of three stages: 1) a 30 day Phase 2 PK and PD evaluation of somavaratan, 2) an optional Phase 2 Extension and 3) a 12 month Phase 3 safety and efficacy stage. The study is a randomized, multi-center, open label study. The primary endpoint is height velocity at 12 months.
Interventions
DRUGSomavaratan (VRS-317)
Long acting recombinant human growth hormone
Sponsors
Versartis Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
3 Years to 10 Years
Healthy volunteers
No
Inclusion criteria
* Chronological Age ≥ 3.0 years and ≤ 9.0 years (girls) or ≤ 10.0 years (boys) * Pre-pubertal status * Diagnosis of GHD as documented by two or more GH stimulation test results * Height SD score ≤ -2.0 at screening * Weight for Stature ≥ 10th percentile * IGF-I SD score ≤ -1.0 at screening * Delayed bone age
Exclusion criteria
* Prior treatment with any growth promoting agent * History of, or current, significant disease * Chromosomal aneuploidy, significant gene mutations (other than those that cause GHD) or confirmed diagnosis of a named syndrome * Birth weight and/or birth length less than 5th percentile for gestational age * A diagnosis of Attention Deficit Hyperactivity Disorder * Daily use of anti-inflammatory doses of glucocorticoid * Prior history of leukemia, lymphoma, sarcoma or cancer * Ocular findings suggestive of increased intracranial pressure and/or retinopathy at screening * Significant spinal abnormalities including scoliosis, kyphosis and spina bifida variants * Significant abnormality in screening laboratory studies
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Efficacy (Annual Height velocity) | 12 months | Annual Height velocity. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Safety (Number of subjects with adverse events ) | 12 months | Number of subjects with adverse events (including repeat dose immunogenicity). |
| Safety (Concomitant medications) | 12 months | Concomitant medications |
| Safety (Safety labs) | 12 months | Safety labs |
| Safety (Vital signs) | 12 months | Vital signs |
| Safety (Physical Exams) | 12 months | Physical Exams |
| Pharmacodynamics (IGF-I responses to study drug administration) | 12 months | IGF-I responses to study drug administration. |
| Pharmacodynamics (IGFBP-3 responses to study drug administration) | 12 months | IGFBP-3 responses to study drug administration. |
Other
| Measure | Time frame | Description |
|---|---|---|
| Secondary Efficacy (Change in height SDS) | 12 months | Change in height SDS. |
| Secondary Efficacy (Change in pubertal staging.) | 12 months | Change in pubertal staging. |
| Secondary Efficacy | 12 months | Change in body weight |
| Secondary Efficacy (Change in bone age) | 12 months | Change in bone age. |
Countries
Japan
Outcome results
None listed