Pediatric Growth Hormone DeficiencyPhase 1/2NCT07264595
A Study (Phase 1b/2) of GS3-007a Oral Treatment in Children With Growth Hormone Deficiency (PGHD)
A Phase 1/2 study testing GS3-007a dry suspension and rhGH injection in people with Pediatric Growth Hormone Deficiency, run by Changchun GeneScience Pharmaceutical Co., Ltd. at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 3 years and older
- Conditions the study is for
- Pediatric Growth Hormone Deficiency
- What is being tested?
- GS3-007a dry suspension and rhGH injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 88 participants
- Where
- 1 siteChina
- Study dates
- 18 Jan 2026to Nov 2030
- Phase
- Phase 1/2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology
Wuhan, Hubei, 430000
About this study
Official 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
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
- Being tested
- GS3-007a dry suspension, Placebo for GS3-007a dry suspension, rhGH injection
- Keywords
- GS3-007a dry suspension, PGHD
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GS3-007a dry suspensionDrug
Also called GS3-007a
GS3-007a
Placebo for GS3-007a dry suspensionDrug
Also called placebo
Placebo for GS3-007a dry suspension
rhGH injectionDrug
Also called rhGH
rhGH injection
| Study group | Role | What they receive |
|---|---|---|
| Dose Level 1~ Dose Level 4 | Placebo comparator | dose level 1、dose level 2、dose level 3、dose level 4 Receives: 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 Receives: Drug: GS3-007a dry suspension; Drug: rhGH injection |
| GS3-007a cohort | Experimental | GS3-007a optimal dose Receives: Drug: GS3-007a dry suspension |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Measured: up to 21 days (Ib phase)
Annualized height velocity (AHV) at Week 26 of treatment
Measured: 26 weeks (II phase)
Number of Participants With TEAEs
Measured: up to 160 weeks (II phase extension)
Secondary outcomes (6)
Plasma Concentrations of GS3-007a and metabolite GS3-017. Due to Spare sampling time, there are no PK parameter
Measured: 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)
Measured: up to 14 days (Ib phase)
AHVs at each evaluation point
Measured: 13 weeks, 39 weeks, 52 weeks (II phase)
The standard deviation score (SDS) for an individual IGF-I measurement is calculated using the age-specific L, M, and S parameters, no minimum and maximum values, with higher scores indicating treatment improvement in PGHD
Measured: 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks (II phase)
AHVs at each evaluation point
Measured: up to 156 weeks (II phase extension)
The standard deviation score (SDS) for an individual height measurement is calculated using the age-specific L, M, and S parameters, no minimum and maximum values, with higher scores indicating treatment improvement in PGHD
Measured: up to 156 weeks (II phase extension)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Dec 2025
- Study start18 Jan 2026
- Primary completionNov 2030 (estimated)
- Study completionNov 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pediatric Growth Hormone DeficiencyStudies of the same condition, open and under way.
- More trials run by Changchun GeneScience Pharmaceutical Co., Ltd.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 88 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07264595
- Sponsor's protocol ID
- GenSci073-201
- Status last verified
- Nov 2025
- First submitted
- 24 Nov 2025
- Last updated
- 16 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No