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NCT Number: NCT07264595

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

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.

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Key information

Conditions

Pediatric Growth Hormone Deficiency

Age range

3 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

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

Wuhan, Hubei, 430000, China

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

GS3-007a dry suspension

Drug

GS3-007a

Other names: GS3-007a

Placebo for GS3-007a dry suspension

Drug

Placebo for GS3-007a dry suspension

Other names: placebo

rhGH injection

Drug

rhGH injection

Other names: rhGH

Primary outcomes

  1. Number of Participants With Treatment Emergent Adverse Events (TEAEs)

    Time frame: up to 21 days (Ib phase)

  2. Annualized height velocity (AHV) at Week 26 of treatment

    Time frame: 26 weeks (II phase)

  3. Number of Participants With TEAEs

    Time frame: up to 160 weeks (II phase extension)

Secondary outcomes

  1. PK concentrations of GS3-007a and metabolite GS3-017

    Time frame: up to 7 days (Ib phase)

  2. Concentrations of serum growth hormone (GH), insulin-like growth factor-1 (IGF-1), and insulin-like growth factor-binding protein 3 (IGFBP-3)

    Time frame: up to 14 days (Ib phase)

  3. AHVs at each evaluation point

    Time frame: 13 weeks, 39 weeks, 52 weeks (II phase)

  4. 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

    Time frame: 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks (II phase)

  5. AHVs at each evaluation point

    Time frame: up to 156 weeks (II phase extension)

  6. Change from extension baseline in the height standard deviation score at each evaluation point (ΔHT SDS)

    Time frame: up to 156 weeks (II phase extension)

Study contacts

Contact information is provided by the study sponsor or research team.

Lingli Sun, bachelor

CONTACT

[email protected]

+86 18006171657

Sponsors and collaborators

Lead sponsor

Changchun GeneScience Pharmaceutical Co., Ltd.

Industry

Registry information

Official study title

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

Important dates

Study start
2025
Primary completion
2030
Study completion
2030
First posted
Dec 4, 2025
Registry last updated
Dec 4, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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