Achondroplasia
Conditions
Keywords
Achondroplasia
Brief summary
To Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children with Achondroplasia
Interventions
DRUGRecombinant human growth hormone
Recombinant human growth hormone (15IU/5mg/3ml/bottle)，0.05 mg/kg/d by subcutaneous injection for 52 weeks
Sponsors
Changchun GeneScience Pharmaceutical Co., Ltd.
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
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Years to 10 Years
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
* 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Annualized Height Velocity (AHV) at Week 52 | week 52 | The change in AHV after 52 weeks treatment |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in AHV | week 4, week 13, week 26, week 39 | Change in AHV at week, 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 Ht SDS at 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 BMI SDS at 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 SH/LL SDS at 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 | Change From Baseline in IGF-1 SDS at week 4, week 13, week 26, week 39, week 52 |
| Change From Baseline in IGF-1/IGFBP-3 ratio | 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 |
| Change From Baseline in Bone Age/Chronological Age ratio (BA/CA) | week 52 | Change From Baseline in BA/CA at week 52 |
Countries
China
Contacts
PRINCIPAL_INVESTIGATORFeihong Luo
Children's Hospital of Fudan University
PRINCIPAL_INVESTIGATORXiaoping Luo
Tongji Hospital
PRINCIPAL_INVESTIGATORWei Gu
Children's Hospital of Nanjing Medical University
PRINCIPAL_INVESTIGATORYu Yang
Jiangxi Province Children's Hospital
PRINCIPAL_INVESTIGATORXinran Cheng
Chengdu Women's and Children's Central Hospital
PRINCIPAL_INVESTIGATORGuimei Li
Shandong Provincial Hospital
PRINCIPAL_INVESTIGATORYing Xin
Shengjing Hospital of China Medical Hospital
PRINCIPAL_INVESTIGATORPin Li
Shanghai Children's Hospital
PRINCIPAL_INVESTIGATORXiaobo Chen
Children's Hospital of The Capital Institute of Pediatrics
PRINCIPAL_INVESTIGATORJin Wu
West China Second University Hospital
PRINCIPAL_INVESTIGATORXiumin Wang
Shanghai Children's Medical Center
Outcome results
None listed