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

NCT ID: NCT07264595

Last Updated: 2025-12-04

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

NOT_YET_RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

88 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-12-31

Study Completion Date

2030-11-30

Brief Summary

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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.

Detailed Description

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Conditions

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Pediatric Growth Hormone Deficiency

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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Dose Level 1~ Dose Level 4

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

Group Type PLACEBO_COMPARATOR

GS3-007a dry suspension

Intervention Type DRUG

GS3-007a

Placebo for GS3-007a dry suspension

Intervention Type DRUG

Placebo for GS3-007a dry suspension

Cohort 1~ Cohort 3

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

Group Type ACTIVE_COMPARATOR

GS3-007a dry suspension

Intervention Type DRUG

GS3-007a

rhGH injection

Intervention Type DRUG

rhGH injection

GS3-007a cohort

GS3-007a optimal dose

Group Type EXPERIMENTAL

GS3-007a dry suspension

Intervention Type DRUG

GS3-007a

Interventions

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GS3-007a dry suspension

GS3-007a

Intervention Type DRUG

Placebo for GS3-007a dry suspension

Placebo for GS3-007a dry suspension

Intervention Type DRUG

rhGH injection

rhGH injection

Intervention Type DRUG

Other Intervention Names

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GS3-007a placebo rhGH

Eligibility Criteria

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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
Minimum Eligible Age

3 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Changchun GeneScience Pharmaceutical Co., Ltd.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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

Wuhan, Hubei, China

Site Status

Countries

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China

Central Contacts

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Lingli Sun, bachelor

Role: CONTACT

+86 18006171657

Other Identifiers

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GenSci073-201

Identifier Type: -

Identifier Source: org_study_id

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