A Study (Phase 1b/2) of GS3-007a Oral Treatment in Children With Growth Hormone Deficiency (PGHD)

Summary

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.

Timeline

Start

2025-12

Primary completion

2030-11

Completion

2030-11

Drugs

Indications