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CompletedNCT05353192Updated Aug 6, 2026

A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Achondroplasia

A Phase 4 interventional study of Recombinant human growth hormone in Achondroplasia, sponsored by Changchun GeneScience Pharmaceutical Co., Ltd.. Completed at 1 site in China. Open to participants aged 2 Years to 10 Years. Per ClinicalTrials.gov, last updated 2026-08-06.

Sponsored by Changchun GeneScience Pharmaceutical Co., Ltd. · Phase 4, Interventional, and Treatment

Phase
Phase 4
Study type
Interventional
Enrollment
39
Allocation
Not applicable
Ages
2 Years to 10 Years
Sex
All
01

Study summary

To Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children with Achondroplasia

02

Conditions studied

  • Achondroplasia

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Keywords

  • Achondroplasia
03

In context

Achondroplasia

47 studies on the registry are indexed under Achondroplasia; 14 are open to participants now.

This study's enrollment of 39 is below the median of 73 across 28 interventional studies indexed under Achondroplasia.

Browse Achondroplasia studies →

Lead sponsor

Changchun GeneScience Pharmaceutical Co., Ltd. is the lead sponsor of 108 studies on the registry; 47 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
2 Years to 10 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • 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

Exclusion criteria

Exclusion criteria:

  • 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
05

Study design

Phase
Phase 4
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
39 participants (actual)

Study arms

  • Experimental
    Recombinant human growth hormone

    Recombinant human growth hormone Injection (15IU/5mg/3ml/bottle);0.05 mg/kg/d by subcutaneous injection for 52 weeks

    Drug: Recombinant human growth hormone

Interventions

  • DrugRecombinant human growth hormone

    Recombinant human growth hormone (15IU/5mg/3ml/bottle),0.05 mg/kg/d by subcutaneous injection for 52 weeks

06

What researchers measure

Primary outcomes

  1. Change From Baseline in Annualized Height Velocity (AHV) at Week 52

    The change in AHV after 52 weeks treatment

    Time frame: week 52

Secondary outcomes

  1. Change From Baseline in AHV

    Change in AHV at week, week 13, week 26, week 39

    Time frame: week 4, week 13, week 26, week 39

  2. 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

    Time frame: week 4, week 13, week 26, week 39, week 52

  3. 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

    Time frame: week 4, week 13, week 26, week 39, week 52

  4. 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

    Time frame: week 4, week 13, week 26, week 39, week 52

  5. 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

    Time frame: week 4, week 13, week 26, week 39, week 52

  6. 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

    Time frame: week 4, week 13, week 26, week 39, week 52

  7. Change From Baseline in Bone Age/Chronological Age ratio (BA/CA)

    Change From Baseline in BA/CA at week 52

    Time frame: week 52

Other outcomes

  1. Change From Baseline in Metabolism-related Proteins

    Change From Baseline in Serum Proteomics at week 13, week 26, week 52

    Time frame: week 13, week 26, week 52

07

Study locations

1 site
  • Children's Hospital of Fudan University
    Shanghai, Shanghai Municipality 201102, China
08

References and documents

Individual participant data

Plan to share: 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.

No publications or documents are linked to this record.

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Aug 6, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT05353192
Lead sponsor
Changchun GeneScience Pharmaceutical Co., Ltd.
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
First posted
Apr 29, 2022
Start date
Aug 20, 2022
Primary completion
Aug 25, 2025
Completion
Dec 12, 2025
Last update
Aug 6, 2026

Study contacts

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
Yu Yang
principal investigator · Jiangxi Province Children's Hospital
Xinran Cheng
principal investigator · Chengdu Women's and Children's Central Hospital
Guimei Li
principal investigator · Shandong Provincial Hospital
Ying Xin
principal investigator · Shengjing Hospital of China Medical Hospital
Pin Li
principal investigator · Shanghai Children's Hospital
Xiaobo Chen
principal investigator · Children's Hospital of The Capital Institute of Pediatrics
Jin Wu
principal investigator · West China Second University Hospital
Xiumin Wang
principal investigator · Shanghai Children's Medical Center

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Aug 2026. You cannot join it, but the record below documents what was studied.

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