This study comprises two phases: Phase Ib and Phase II. Phase Ib is a multicenter, randomized, open-label, active-controlled, single-dose, dose-escalation to assess safety, tolerability, PK/PD profile, and immunogenicity of GenSci134 in children with GHD. Phase II is a multicenter, randomized, open-label, active-controlled, multiple-dose, parallel-group study to assess the efficacy and safety of multiple subcutaneous doses of GenSci134 at different levels versus Norditropin® in children with GHD. It will also evaluate PK/PD profile and immunogenicity to support dose selection for Phase III.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
128
only one dose of GenSci134 to be given, subcutaneous , 6 dose levels will be assigned.
multiple doses of Norditropin® FlexPro® quaque die (QD) for 28 consecutive days by subcutaneous injections.
Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology
Wuhan, Hubei, China
Phase Ib: Treatment Emergent Adverse Events (TEAEs)
Time frame: From the first dose to Day 35
Phase II: Annualized height velocity (AHV) at Week 24 of treatment
Time frame: 24 weeks
Phase Ib: Areas under the drug concentration-time curve (AUC0-t, AUC0-∞) of GenSci134
Time frame: From Day 1 to Day 29
Phase Ib: Time to maximum concentration (Tmax) of GenSci134
Time frame: From Day 1 to Day 29
Phase Ib: Maximum concentration (Cmax) of GenSci134
Time frame: From Day 1 to Day 29
Phase Ib: Half-life (t1/2) of GenSci134
Time frame: From Day 1 to Day 29
Phase Ib: Serum level of IGF-1 and IGFBP-3 and their changes from baseline.
Time frame: From Day 1 to Day 29
Phase Ib: Incidence and timing of positive anti-drug antibody (ADA) and/or neutralizing antibody (NAb) (if applicable)
Time frame: From Day 1 to Day 29
Phase II: Change from baseline in HT SDS at each visit
Time frame: From baseline to Week 28 of the extension period
Phase II: Change from baseline in AHV at each visit
Time frame: From baseline to Week 28 of the extension period
Phase II: Change from baseline in BA/CA at each visit.
Time frame: From baseline to Week 28 of the extension period
Phase II: Treatment Emergent Adverse Events(TEAEs)
Time frame: From the first dose to Week 29 of the extension period
Phase II: Serum concentration of GenSci134.
Time frame: From baseline to Week 28 of the extension period
Phase II: Serum level of IGF-1and IGFBP-3 and their changes from baseline.
Time frame: From baseline to Week 28 of the extension period
Phase II: Incidence and timing of positive ADA and/or NAb (if applicable).
Time frame: From baseline to Week 28 of the extension period
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