AchondroplasiaPhase 4NCT05353192
A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Achondroplasia
A Phase 4 study testing Recombinant human growth hormone in people with Achondroplasia, run by Changchun GeneScience Pharmaceutical Co., Ltd. at 1 site in China.
Trial summary
To Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children with Achondroplasia Read more about this study
- Who is it for?
- Ages 2 to 10 years
- Conditions the study is for
- Achondroplasia
- What is being tested?
- Recombinant human growth hormone
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 39 participants
- Where
- 1 siteChina
- Study dates
- 20 Aug 2022to 12 Dec 2025
- Phase
- Phase 4
- Are there results?
- None posted in the registry
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 2 to 10 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 2-10 years old;
- In Tanner I stage;
- ACH, documented and confirmed by genetic testing;
- Short stature;
- Ambulatory and able to stand or walk without assistance;
- Parent(s) or guardian(s) consent；
- Had never been treated with growth hormone
Who cannot join
- Short stature condition other than ACH;
- Evidence of growth plate closure (proximal tibia, distal femur);
- Had a fracture of the long bones within 6 months prior to screening;
- Planned or expected bone-related surgery;
- Chronic diseases condition that affect bone metabolism and weight;
- Severe intracranial hypertension
Trial locations and contacts
1 site in 1 country.
China1 site
Children's Hospital of Fudan University
Shanghai, Shanghai Municipality, 201102
About this study
Official title: A Multicenter, Open-label, Single Arm Phase IV Clinical Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Achondroplasia
To Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children with Achondroplasia
- Conditions
- Achondroplasia
- Being tested
- Recombinant human growth hormone
- Keywords
- Achondroplasia
- Medical subject headings
- Achondroplasia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Recombinant human growth hormoneDrug
Recombinant human growth hormone (15IU/5mg/3ml/bottle)，0.05 mg/kg/d by subcutaneous injection for 52 weeks
| Study group | Role | What they receive |
|---|---|---|
| Recombinant human growth hormone | Experimental | Recombinant human growth hormone Injection (15IU/5mg/3ml/bottle)；0.05 mg/kg/d by subcutaneous injection for 52 weeks Receives: Drug: Recombinant human growth hormone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Annualized Height Velocity (AHV) at Week 52
The change in AHV after 52 weeks treatment
Measured: week 52
Secondary outcomes (7)
Change From Baseline in AHV
Change in AHV at week, week 13, week 26, week 39
Measured: week 4, week 13, week 26, week 39
Change From Baseline in Height Standard Deviation Score (Ht SDS)
Change From Baseline in Ht SDS at week 4, week 13, week 26, week 39, week 52
Measured: week 4, week 13, week 26, week 39, week 52
Change From Baseline in BMI Standard Deviation Score (BMI SDS)
Change From Baseline in BMI SDS at week 4, week 13, week 26, week 39, week 52
Measured: week 4, week 13, week 26, week 39, week 52
Change From Baseline in Sitting Height/Leg Length ratio Standard Deviation Score (SH/LL SDS)
Change From Baseline in SH/LL SDS at week 4, week 13, week 26, week 39, week 52
Measured: week 4, week 13, week 26, week 39, week 52
Change From Baseline in IGF-1 Standard Deviation Score (IGF -1 SDS)
Change From Baseline in IGF-1 SDS at week 4, week 13, week 26, week 39, week 52
Measured: week 4, week 13, week 26, week 39, week 52
Change From Baseline in IGF-1/IGFBP-3 ratio
Change From Baseline in IGF-1/IGFBP-3 ratio at week 4, week 13, week 26, week 39, week 52
Measured: week 4, week 13, week 26, week 39, week 52
Change From Baseline in Bone Age/Chronological Age ratio (BA/CA)
Change From Baseline in BA/CA at week 52
Measured: week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Apr 2022
- Study start20 Aug 2022
- Primary completion25 Aug 2025
- Study completion12 Dec 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AchondroplasiaStudies 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 39 participants (actual)
Sponsor and investigators
- Lead sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Children's Hospital of Fudan University, Tongji Hospital, Children's Hospital of Nanjing Medical University, Jiangxi Province Children's Hospital, Chengdu Women's and Children's Central Hospital, Shandong Provincial Hospital, Shengjing Hospital, Shanghai Children's Hospital, Children's Hospital of The Capital Institute of Pediatrics, West China Second University Hospital, Shanghai Children's Medical Center
- Responsible party
- Sponsor
- Investigators
- Feihong Luo, Principal investigator, Children's Hospital of Fudan UniversityXiaoping Luo, Principal investigator, Tongji HospitalWei Gu, Principal investigator, Children's Hospital of Nanjing Medical UniversityYu Yang, Principal investigator, Jiangxi Province Children's HospitalXinran Cheng, Principal investigator, Chengdu Women's and Children's Central HospitalGuimei Li, Principal investigator, Shandong Provincial HospitalYing Xin, Principal investigator, Shengjing Hospital of China Medical HospitalPin Li, Principal investigator, Shanghai Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05353192
- Sponsor's protocol ID
- GenSci001-01
- Status last verified
- Aug 2026
- First submitted
- 25 Apr 2022
- Last updated
- 6 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Drugs belong to the sponsor before they are put into clinical trials and the site is only a contract research organization; It is an industry practice that intellectual property rights belong to the sponsor.
Study at a glance
From the registry record, last updated by the sponsor on 6 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Open-label, Single Arm Phase IV Clinical Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Achondroplasia
- ClinicalTrials.gov ID
- NCT05353192
- Acronym / sponsor ID
- GenSci001-01
- Conditions
- Achondroplasia
- Keywords
- Achondroplasia
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 39 participants (actual)
- Lead sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.Industry (pharma, biotech, devices)
- Collaborators
- Children's Hospital of Fudan University, Tongji Hospital, Children's Hospital of Nanjing Medical University, Jiangxi Province Children's Hospital, Chengdu Women's and Children's Central Hospital, Shandong Provincial Hospital, Shengjing Hospital, Shanghai Children's Hospital, Children's Hospital of The Capital Institute of Pediatrics, West China Second University Hospital, Shanghai Children's Medical Center
- Study start
- 20 Aug 2022 (actual)
- Primary completion
- 25 Aug 2025 (actual)
- Study completion
- 12 Dec 2025 (actual)
- First posted
- 29 Apr 2022 (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.
To Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children with Achondroplasia
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Recombinant human growth hormone | Experimental | Drug: Recombinant human growth hormoneRecombinant human growth hormone Injection (15IU/5mg/3ml/bottle)；0.05 mg/kg/d by subcutaneous injection for 52 weeks |
Who may qualify
- Ages
- 2 to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- 2-10 years old;
- In Tanner I stage;
- ACH, documented and confirmed by genetic testing;
- Short stature;
- Ambulatory and able to stand or walk without assistance;
Key exclusion criteria (6 listed)
- Short stature condition other than ACH;
- Evidence of growth plate closure (proximal tibia, distal femur);
- Had a fracture of the long bones within 6 months prior to screening;
- Planned or expected bone-related surgery;
- Chronic diseases condition that affect bone metabolism and weight;
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 |
|---|---|---|
| Recombinant human growth hormoneRecombinant human growth hormone (15IU/5mg/3ml/bottle)，0.05 mg/kg/d by subcutaneous injection for 52 weeks | Drug | Recombinant human growth hormone |
Primary outcome
Change From Baseline in Annualized Height Velocity (AHV) at Week 52
Time frame: week 52
The change in AHV after 52 weeks treatment
7 secondary outcomes
Change From Baseline in AHV
Time frame: week 4, week 13, week 26, week 39
Change From Baseline in Height Standard Deviation Score (Ht SDS)
Time frame: week 4, week 13, week 26, week 39, week 52
Change From Baseline in BMI Standard Deviation Score (BMI SDS)
Time frame: week 4, week 13, week 26, week 39, week 52
Change From Baseline in Sitting Height/Leg Length ratio Standard Deviation Score (SH/LL SDS)
Time frame: week 4, week 13, week 26, week 39, week 52
Change From Baseline in IGF-1 Standard Deviation Score (IGF -1 SDS)
Time frame: week 4, week 13, week 26, week 39, week 52
Change From Baseline in IGF-1/IGFBP-3 ratio
Time frame: week 4, week 13, week 26, week 39, week 52
Change From Baseline in Bone Age/Chronological Age ratio (BA/CA)
Time frame: week 52
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Aug 2026.
- Individual participant dataSharing planned: No. Drugs belong to the sponsor before they are put into clinical trials and the site is only a contract research organization; It is an industry practice that intellectual property rights belong to the sponsor.
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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. This study is not recruiting at the moment.
Feihong Luo principal investigator
Children's Hospital of Fudan University
Xiaoping Luo principal investigator
Tongji Hospital
Wei Gu principal investigator
Children's Hospital of Nanjing Medical University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Recombinant human growth hormone in people with Achondroplasia, 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 7 inclusion and 6 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: finished, as of the sponsor's last update on 6 Aug 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline in Annualized Height Velocity (AHV) at Week 52 (time frame: week 52). 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 6 Aug 2026.