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

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 Overview

• Status: Not yet recruiting
• Conditions: Pediatric Growth Hormone Deficiency
• Intervention / Treatment: Drug: GS3-007a dry suspension; Drug: Placebo for GS3-007a dry suspension; Drug: rhGH injection

• Study Type: Interventional
• Enrollment (Estimated): 88
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Lingli Sun, bachelor; Phone Number: +86 18006171657; Email: sunlingli@genscigroup.com

Study Locations

• China
  • Hubei
    • Wuhan, Hubei, China, 430000
      • Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• chronological age (CA) ≥3 years at Screening
• Prepubertal girls or boys
• Height at Screening lower than the reference height for normal children of the same chronological age and sex minus 2 standard deviations (-2 SD)
• A confirmed diagnosis of growth hormone deficiency (GHD)
• Having not been treated with any growth-promoting drugs
• BA delayed by ≥6 months compared with the CA at Screening Subjects who meet all of the following criteria are eligible to enroll in the extension study (applicable for Part II extension period)
• Subjects who have completed the 52-week treatment period of phase II
• Subjects who do not permanently discontinue the investigational Medicinal Product (IMP) during the 52-week treatment period of phase II

Exclusion Criteria:

• A highly allergic constitution
• Suspected or confirmed total pituitary deficiency, including patients previously confirmed with deficiency of ≥2 pituitary hormones other than GH
• Being confirmed with other chromosomal abnormalities or growth abnormalities affecting growth
• Congenital skeletal dysplasia or serious spinal anomalies
• Cognitive hypofunction, neurodevelopmental disorders, or psychiatric/psychological disorders that, in the investigator's opinion, may interfere with evaluation of study endpoints
• Any clinically significant abnormality that may affect growth or evaluation of the IMP
• Screening magnetic resonance imaging (MRI) scan of the sellar region confirming prior or current intracranial tumor growth
• Concurrent use of any medications that may affect growth or response to growth hormone therapy
• Epiphyseal closure
• Electrocardiogram (ECG) QTcF interval abnormal, with a history of QT/QTc interval prolonged
• Hepatic function indicators abnormal at Screening Patients meeting any of the following criteria may not be enrolled in this extension study (applicable for Part II extension period)
• Subjects with closed epiphyses
• Any clinically significant abnormality that may affect growth or evaluation of the IMP
• Known or suspected allergy to the IMP
• Women with positive blood human chorionic gonadotropin (hCG) at the pre-treatment visit

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: Triple

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Dose Level 1~ Dose Level 4; dose level 1、dose level 2、dose level 3、dose level 4	Drug: GS3-007a dry suspension; GS3-007a; Other Names: GS3-007a; Drug: Placebo for GS3-007a dry suspension; Placebo for GS3-007a dry suspension; Other Names: placebo
Active Comparator: Cohort 1~ Cohort 3; GS3-007a low dose , GS3-007a high dose , rhGH 0.033mg/kg	Drug: GS3-007a dry suspension; GS3-007a; Other Names: GS3-007a; Drug: rhGH injection; rhGH injection; Other Names: rhGH
Experimental: GS3-007a cohort; GS3-007a optimal dose	Drug: GS3-007a dry suspension; GS3-007a; Other Names: GS3-007a

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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)

Secondary Outcome Measures

Outcome Measure	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)
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)

Collaborators and Investigators

• Sponsor: Changchun GeneScience Pharmaceutical Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Estimated): December 1, 2025
• Primary Completion (Estimated): November 1, 2030
• Study Completion (Estimated): November 1, 2030

Study Registration Dates

• First Submitted: November 24, 2025
• First Submitted That Met QC Criteria: November 24, 2025
• First Posted (Estimated): December 4, 2025

Study Record Updates

• Last Update Posted (Estimated): December 4, 2025
• Last Update Submitted That Met QC Criteria: November 24, 2025
• Last Verified: November 1, 2025

More Information

Terms related to this study

• Keywords: PGHD; GS3-007a dry suspension
• Other Study ID Numbers: GenSci073-201

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No