A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Achondroplasia
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
1 other identifier
interventional
38
1 country
1
Brief Summary
To Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children with Achondroplasia
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_4
Started Sep 2022
Typical duration for phase_4
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
April 25, 2022
CompletedFirst Posted
Study publicly available on registry
April 29, 2022
CompletedStudy Start
First participant enrolled
September 23, 2022
CompletedPrimary Completion
Last participant's last visit for primary outcome
February 1, 2025
CompletedStudy Completion
Last participant's last visit for all outcomes
February 1, 2025
CompletedFebruary 28, 2023
February 1, 2023
2.4 years
April 25, 2022
February 26, 2023
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Change From Baseline in Annualized Height Velocity (AHV) at Week 52
The change in AHV after 52 weeks treatment
week 52
Secondary Outcomes (7)
Change From Baseline in AHV
week 4, week 13, week 26, week 39
Change From Baseline in Height Standard Deviation Score (Ht SDS)
week 4, week 13, week 26, week 39, week 52
Change From Baseline in BMI Standard Deviation Score (BMI SDS)
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)
week 4, week 13, week 26, week 39, week 52
Change From Baseline in IGF-1 Standard Deviation Score (IGF -1 SDS)
week 4, week 13, week 26, week 39, week 52
- +2 more secondary outcomes
Other Outcomes (1)
Change From Baseline in Metabolism-related Proteins
week 13, week 26, week 52
Study Arms (1)
Recombinant human growth hormone
EXPERIMENTALRecombinant human growth hormone Injection (15IU/5mg/3ml/bottle)；0.05 mg/kg/d by subcutaneous injection for 52 weeks
Interventions
Recombinant human growth hormone (15IU/5mg/3ml/bottle)，0.05 mg/kg/d by subcutaneous injection for 52 weeks
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Changchun GeneScience Pharmaceutical Co., Ltd.lead
- Children's Hospital of Fudan Universitycollaborator
- Tongji Hospitalcollaborator
- Children's Hospital of Nanjing Medical Universitycollaborator
- Jiangxi Province Children's Hospitalcollaborator
- Chengdu Women's and Children's Central Hospitalcollaborator
- Shandong Provincial Hospitalcollaborator
- Shengjing Hospitalcollaborator
- Shanghai Children's Hospitalcollaborator
- Children's Hospital of The Capital Institute of Pediatricscollaborator
- West China Second University Hospitalcollaborator
Study Sites (1)
Children's Hospital of Fudan University
Shanghai, Shanghai Municipality, 201102, China
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Feihong Luo
Children's Hospital of Fudan University
- PRINCIPAL INVESTIGATOR
Xiaoping Luo
Tongji Hospital
- 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
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 4
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 25, 2022
First Posted
April 29, 2022
Study Start
September 23, 2022
Primary Completion
February 1, 2025
Study Completion
February 1, 2025
Last Updated
February 28, 2023
Record last verified: 2023-02
Data Sharing
- IPD Sharing
- Will not share
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.