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

August 4, 2026 updated by: Changchun GeneScience Pharmaceutical Co., Ltd.

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

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
39

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

2 years to 10 years (Child)

Accepts Healthy Volunteers

No

Description

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:

  • 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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
Recombinant human growth hormone (15IU/5mg/3ml/bottle)，0.05 mg/kg/d by subcutaneous injection for 52 weeks

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Change From Baseline in Annualized Height Velocity (AHV) at Week 52
Time Frame: week 52
The change in AHV after 52 weeks treatment
week 52

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Change From Baseline in AHV
Time Frame: week 4, week 13, week 26, week 39
Change in AHV at week, week 13, week 26, week 39
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 Ht SDS at week 4, week 13, week 26, week 39, week 52
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 BMI SDS at week 4, week 13, week 26, week 39, week 52
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 SH/LL SDS at week 4, week 13, week 26, week 39, week 52
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 SDS at week 4, week 13, week 26, week 39, week 52
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 IGF-1/IGFBP-3 ratio at week 4, week 13, week 26, week 39, week 52
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
Change From Baseline in BA/CA at week 52
week 52

Other Outcome Measures

Other Outcome Measures

For clinical trials, a planned measurement described in the protocol that is used to determine the effect of an intervention/treatment on participants. For observational studies, a measurement or observation that is used to describe patterns of diseases or traits, or associations with exposures, risk factors, or treatment. Types of outcome measures include primary outcome measure and secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Change From Baseline in Metabolism-related Proteins
Time Frame: week 13, week 26, week 52
Change From Baseline in Serum Proteomics at week 13, week 26, week 52
week 13, week 26, week 52

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Changchun GeneScience Pharmaceutical Co., Ltd.

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Tongji Hospital
Shengjing Hospital
Shandong Provincial Hospital
Children's Hospital of Fudan University
Shanghai Children's Hospital
Shanghai Children's Medical Center
Chengdu Women's and Children's Central Hospital
West China Second University Hospital
Jiangxi Province Children's Hospital
Children's Hospital of Nanjing Medical University
Children's Hospital of The Capital Institute of Pediatrics

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Xiaoping Luo, Tongji Hospital
  • Principal Investigator: Feihong Luo, Children's Hospital of Fudan University
  • Principal Investigator: Wei Gu, Children's Hospital of Nanjing Medical University
  • Principal Investigator: Yu Yang, Jiangxi Province Children's Hospital
  • Principal Investigator: Xinran Cheng, Chengdu Women's and Children's Central Hospital
  • Principal Investigator: Guimei Li, Shandong Provincial Hospital
  • Principal Investigator: Ying Xin, Shengjing Hospital of China Medical Hospital
  • Principal Investigator: Pin Li, Shanghai Children's Hospital
  • Principal Investigator: Xiaobo Chen, Children's Hospital of The Capital Institute of Pediatrics
  • Principal Investigator: Jin Wu, West China Second University Hospital
  • Principal Investigator: Xiumin Wang, Shanghai Children's Medical Center

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
August 20, 2022

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
August 25, 2025

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 12, 2025

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
April 25, 2022

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 25, 2022

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
April 29, 2022

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
August 6, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 4, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • GenSci001-01

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

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.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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