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

Phase 4

Recruiting

• Conditions: Achondroplasia
• Interventions: Drug: Recombinant human growth hormone

• Registration Number: NCT05353192
• Lead Sponsor: Changchun GeneScience Pharmaceutical Co., Ltd.

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2022-04-25
• Study First Submitted QC: 2022-04-25
• Study First Posted: 2022-04-29
• Study Start: 2022-09-23
• Status Verified: 2023-02
• Last Update Submitted: 2023-02-26
• Last Update Posted: 2023-02-28
• Primary Completion: 2025-02
• Study Completion: 2025-02

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 38

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

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Recombinant human growth hormone	Recombinant human growth hormone	Recombinant human growth hormone Injection (15IU/5mg/3ml/bottle)；0.05 mg/kg/d by subcutaneous injection for 52 weeks

Primary Outcome Measures

Name	Time	Method
Change From Baseline in Annualized Height Velocity (AHV) at Week 52	week 52	The change in AHV after 52 weeks treatment

Secondary Outcome Measures

Name	Time	Method
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
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 AHV	week 4, week 13, week 26, week 39	Change in AHV at week, week 13, week 26, week 39

Trial Locations

Locations (1)

Children's Hospital of Fudan University; 🇨🇳 Shanghai, Shanghai, China