NCT07264595

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

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

• Status: Not Yet Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 88 (estimated)
• Sponsor: Changchun GeneScience Pharmaceutical Co., Ltd. · Industry
• Sex: All
• Age: 3 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Pediatric Growth Hormone Deficiency

Interventions

Type	Name	Description
DRUG	GS3-007a dry suspension	GS3-007a
DRUG	Placebo for GS3-007a dry suspension	Placebo for GS3-007a dry suspension
DRUG	rhGH injection	rhGH injection

Timeline

• Start date: 2025-12-01
• Primary completion: 2030-11-01
• Completion: 2030-11-01
• First posted: 2025-12-04
• Last updated: 2025-12-04

Locations

1 site across 1 country: China