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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Dose Level 1~ Dose Level 4 | Placebo Comparator | dose level 1、dose level 2、dose level 3、dose level 4 |
|
| Cohort 1~ Cohort 3 | Active Comparator | GS3-007a low dose , GS3-007a high dose , rhGH 0.033mg/kg |
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| GS3-007a cohort | Experimental | GS3-007a optimal dose |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| GS3-007a dry suspension | Drug | GS3-007a |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | up to 21 days (Ib phase) | |
| Annualized height velocity (AHV) at Week 26 of treatment | 26 weeks (II phase) | |
| Number of Participants With TEAEs | up to 160 weeks (II phase extension) |
| Measure | Description | Time Frame |
|---|---|---|
| PK concentrations of GS3-007a and metabolite GS3-017 | 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) | up to 14 days (Ib phase) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Lingli Sun, bachelor | Contact | +86 18006171657 | sunlingli@genscigroup.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology | Wuhan | Hubei | 430000 | China |
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| Placebo for GS3-007a dry suspension | Drug | Placebo for GS3-007a dry suspension |
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| rhGH injection | Drug | rhGH injection |
|
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| AHVs at each evaluation point | 13 weeks, 39 weeks, 52 weeks (II phase) |
| Changes from baseline (ΔIGF-1 SDS) in the standard deviation score of the PD indicator insulin-like growth factor-1 (IGF-1 SDS) at each evaluation point | 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks (II phase) |
| AHVs at each evaluation point | up to 156 weeks (II phase extension) |
| Change from extension baseline in the height standard deviation score at each evaluation point (ΔHT SDS) | up to 156 weeks (II phase extension) |