Achondroplasia Clinical Trial
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
Status | Recruiting |
Enrollment | 38 |
Est. completion date | February 2025 |
Est. primary completion date | February 2025 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 2 Years to 10 Years |
Eligibility | 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 |
Country | Name | City | State |
---|---|---|---|
China | Children's Hospital of Fudan University | Shanghai | Shanghai |
Lead Sponsor | Collaborator |
---|---|
GeneScience Pharmaceuticals Co., Ltd. | Chengdu Women's and Children's Central Hospital, Children's Hospital of Fudan University, Children's Hospital of Nanjing Medical University, Children's Hospital of The Capital Institute of Pediatrics, Jiangxi Province Children's Hospital, Shandong Provincial Hospital, Shanghai Children's Hospital, Shengjing Hospital, Tongji Hospital, West China Second University Hospital, Sichuan University |
China,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Other | Change From Baseline in Metabolism-related Proteins | Change From Baseline in Serum Proteomics at week 13, week 26, week 52 | week 13, week 26, week 52 | |
Primary | Change From Baseline in Annualized Height Velocity (AHV) at Week 52 | The change in AHV after 52 weeks treatment | week 52 | |
Secondary | Change From Baseline in AHV | Change in AHV at week, week 13, week 26, week 39 | week 4, week 13, week 26, week 39 | |
Secondary | 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 | |
Secondary | 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 | |
Secondary | 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 | |
Secondary | 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 | |
Secondary | 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 | |
Secondary | Change From Baseline in Bone Age/Chronological Age ratio (BA/CA) | Change From Baseline in BA/CA at week 52 | week 52 |
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