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

Study Overview
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Brief Summary
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To Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children with Achondroplasia

Official Title
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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

Conditions
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Achondroplasia

Intervention / Treatment
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* Drug: Recombinant human growth hormone


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 10 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Recombinant human growth hormone<br>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<br>* Recombinant human growth hormone (15IU/5mg/3ml/bottle)，0.05 mg/kg/d by subcutaneous injection for 52 weeks<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change From Baseline in Annualized Height Velocity (AHV) at Week 52 | The change in AHV after 52 weeks treatment | week 52 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change From Baseline in AHV | 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) | 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) | 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) | 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) | 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 | 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) | Change From Baseline in BA/CA at week 52 | week 52 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Achondroplasia