Last reviewed · How we verify
A Study to Evaluate the Safety and Tolerability of Multiple Ascending Doses of GS3-007a Dry Suspension in Patients With PGHD and to Evaluate the Efficacy and Safety of 52-week of Treatment With GS3-007a Dry Suspension: a Two-part, Multicenter, Randomized, Phase Ib/II Clinical Trial
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.
Details
| Lead sponsor | Changchun GeneScience Pharmaceutical Co., Ltd. |
|---|---|
| Phase | Phase 1/Phase 2 |
| Status | NOT_YET_RECRUITING |
| Enrolment | 88 |
| Start date | 2025-12 |
| Completion | 2030-11 |
Conditions
- Pediatric Growth Hormone Deficiency
Interventions
- GS3-007a dry suspension
- Placebo for GS3-007a dry suspension
- rhGH injection
Primary outcomes
- 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)
Countries
China