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

NCT07264595 | Not Yet Recruiting Phase 1

• 1 other identifier: GenSci073-201
• Study Type: interventional
• Target: 88
• Locations: 1 country
• Sites: 1

Brief 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

Keywords

GS3-007a dry suspension; PGHD

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (6)

• 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)

Study Arms (3)

Dose Level 1~ Dose Level 4

PLACEBO COMPARATOR

dose level 1、dose level 2、dose level 3、dose level 4

Drug: GS3-007a dry suspension; Drug: Placebo for GS3-007a dry suspension

Cohort 1~ Cohort 3

ACTIVE COMPARATOR

GS3-007a low dose , GS3-007a high dose , rhGH 0.033mg/kg

Drug: GS3-007a dry suspension; Drug: rhGH injection

GS3-007a cohort

EXPERIMENTAL

GS3-007a optimal dose

Drug: GS3-007a dry suspension

Interventions

GS3-007a dry suspension DRUG

GS3-007a

Also known as: GS3-007a

Cohort 1~ Cohort 3; Dose Level 1~ Dose Level 4; GS3-007a cohort

Placebo for GS3-007a dry suspension DRUG

Placebo for GS3-007a dry suspension

Also known as: placebo

Dose Level 1~ Dose Level 4

rhGH injection DRUG

rhGH injection

Also known as: rhGH

Cohort 1~ Cohort 3

Eligibility Criteria

• Age: 3 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Changchun GeneScience Pharmaceutical Co., Ltd.: lead

Study Sites (1)

Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology

Wuhan, Hubei, 430000, China

Location

Central Study Contacts

Lingli Sun, bachelor

CONTACT

+86 18006171657; sunlingli@genscigroup.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 24, 2025
• First Posted: December 4, 2025
• Study Start: December 1, 2025
• Primary Completion (Estimated): November 1, 2030
• Study Completion (Estimated): November 1, 2030
• Last Updated: December 4, 2025

Record last verified: 2025-11

Locations

CN (1)