RECRUITING Phase 1 / Phase 2 ClinicalTrials.gov

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

Sponsor: Changchun GeneScience Pharmaceutical Co., Ltd. • ClinicalTrials.gov ID: NCT07264595

Registry updated: 2026-09-16

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Description

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.

Interventions

GS3-007a dry suspension (Drug)
GS3-007a
Placebo for GS3-007a dry suspension (Drug)
Placebo for GS3-007a dry suspension
rhGH injection (Drug)
rhGH injection

Eligibility criteria

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

Conditions

Pediatric Growth Hormone Deficiency

Locations

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

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Official Source

Data sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

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