This is a two-part clinical study for children with growth hormone deficiency. In the first part, participants will be randomly assigned to receive different doses of an oral treatment (GS3-007a dry suspension) or a placebo for 14 days. This part is double-blinded, meaning neither the participants nor the doctors will know who is receiving the treatment or placebo. The goal is to find a safe and well-tolerated dose. In the second part, participants will be randomly assigned to receive either the selected dose of GS3-007a or another approved treatment for 52 weeks. This part is open-label, so everyone will know which treatment is being given. After that, all participants may continue taking GS3-007a for another 156 weeks in an extension phase to study long-term effects.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
88
GS3-007a
Placebo for GS3-007a dry suspension
rhGH injection
Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology
Wuhan, Hubei, China
RECRUITINGNumber of Participants With Treatment Emergent Adverse Events (TEAEs)
Time frame: up to 21 days (Ib phase)
Annualized height velocity (AHV) at Week 26 of treatment
Time frame: 26 weeks (II phase)
Number of Participants With TEAEs
Time frame: up to 160 weeks (II phase extension)
Plasma Concentrations of GS3-007a and metabolite GS3-017. Due to Spare sampling time, there are no PK parameter
Time frame: up to 7 days (Ib phase)
Concentrations of serum growth hormone (GH), insulin-like growth factor-1 (IGF-1), and insulin-like growth factor-binding protein 3 (IGFBP-3)
Time frame: up to 14 days (Ib phase)
AHVs at each evaluation point
Time frame: 13 weeks, 39 weeks, 52 weeks (II phase)
The standard deviation score (SDS) for an individual IGF-I measurement is calculated using the age-specific L, M, and S parameters, no minimum and maximum values, with higher scores indicating treatment improvement in PGHD
Time frame: 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks (II phase)
AHVs at each evaluation point
Time frame: up to 156 weeks (II phase extension)
The standard deviation score (SDS) for an individual height measurement is calculated using the age-specific L, M, and S parameters, no minimum and maximum values, with higher scores indicating treatment improvement in PGHD
Time frame: up to 156 weeks (II phase extension)
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