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

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05353192
• Other study ID #: GenSci001-01
• Status: Recruiting
• Phase: Phase 4
• Start date: September 23, 2022
• Est. completion date: February 2025

Study information

• Verified date: February 2023
• Source: GeneScience Pharmaceuticals Co., Ltd.
• Contact: Yanru Wu
• Phone: +86 15843110489
• Email: wuyanru[@]gensci-china.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

To Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children with Achondroplasia

Recruitment information / eligibility

• 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

Related Conditions & MeSH terms

• Achondroplasia

Intervention

Drug:
• Recombinant human growth hormone
Recombinant human growth hormone (15IU/5mg/3ml/bottle),0.05 mg/kg/d by subcutaneous injection for 52 weeks

Locations

Country	Name	City	State
China	Children's Hospital of Fudan University	Shanghai	Shanghai

Sponsors (11)

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

Country where clinical trial is conducted

China, 

Outcome

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