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
- Are there results?
- Not yet: the study is under way
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
Show all 15Show fewer
- 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
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT07264595
- Acronym / sponsor ID
- GenSci073-201
- Conditions
- Pediatric Growth Hormone Deficiency
- Keywords
- GS3-007a dry suspension, PGHD
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 88 participants (estimated)
- Lead sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 18 Jan 2026 (actual)
- Primary completion
- Nov 2030 (estimated)
- Study completion
- Nov 2030 (estimated)
- First posted
- 4 Dec 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Dose Level 1~ Dose Level 4 | Placebo comparator | Drug: GS3-007a dry suspension, Drug: Placebo for GS3-007a dry suspensiondose level 1、dose level 2、dose level 3、dose level 4 |
| Cohort 1~ Cohort 3 | Active comparator | Drug: GS3-007a dry suspension, Drug: rhGH injectionGS3-007a low dose , GS3-007a high dose , rhGH 0.033mg/kg |
| GS3-007a cohort | Experimental | Drug: GS3-007a dry suspensionGS3-007a optimal dose |
Who may qualify
- Ages
- 3 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (15 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GS3-007a dry suspensionAlso: GS3-007aGS3-007a | Drug | Cohort 1~ Cohort 3, Dose Level 1~ Dose Level 4, GS3-007a cohort |
| Placebo for GS3-007a dry suspensionAlso: placeboPlacebo for GS3-007a dry suspension | Drug | Dose Level 1~ Dose Level 4 |
| rhGH injectionAlso: rhGHrhGH injection | Drug | Cohort 1~ Cohort 3 |
Primary outcome
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Time frame: up to 21 days (Ib phase)
Annualized height velocity (AHV) at Week 26 of treatment
Time frame: 26 weeks (II phase)
Number of Participants With TEAEs
Time frame: up to 160 weeks (II phase extension)
6 secondary outcomes
Plasma Concentrations of GS3-007a and metabolite GS3-017. Due to Spare sampling time, there are no PK parameter
Time frame: 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)
Time frame: up to 14 days (Ib phase)
AHVs at each evaluation point
Time frame: 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
Time frame: 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks (II phase)
AHVs at each evaluation point
Time frame: 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
Time frame: up to 156 weeks (II phase extension)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Sep 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
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.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 16 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Lingli Sun, bachelor (+86 18006171657, sunlingli@genscigroup.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Treatment Emergent Adverse Events (TEAEs) (time frame: up to 21 days (Ib phase)). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Sep 2026.