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.
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
41
Long acting recombinant human growth hormone
Hokkaido University Hospital
Sapporo, Japan
Efficacy (Annual Height velocity)
Annual Height velocity.
Time frame: 12 months
Pharmacodynamics (IGF-I responses to study drug administration)
IGF-I responses to study drug administration.
Time frame: 12 months
Pharmacodynamics (IGFBP-3 responses to study drug administration)
IGFBP-3 responses to study drug administration.
Time frame: 12 months
Safety (Number of subjects with adverse events )
Number of subjects with adverse events (including repeat dose immunogenicity).
Time frame: 12 months
Safety (Concomitant medications)
Concomitant medications
Time frame: 12 months
Safety (Safety labs)
Safety labs
Time frame: 12 months
Safety (Vital signs)
Vital signs
Time frame: 12 months
Safety (Physical Exams)
Physical Exams
Time frame: 12 months
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