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

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

Study Overview

• Status: Recruiting
• Conditions: Achondroplasia
• Intervention / Treatment: Drug: Recombinant human growth hormone

• Study Type: Interventional
• Enrollment (Anticipated): 38
• Phase: Phase 4

Contacts and Locations

Study Locations

• China
  • Shanghai
    • Shanghai, Shanghai, China, 201102
      • Recruiting
      • Children's Hospital of Fudan University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 10 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

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

What is the study measuring?

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

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
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

Collaborators and Investigators

• Sponsor: GeneScience Pharmaceuticals Co., Ltd.
• Collaborators: Tongji Hospital; Shengjing Hospital; Shandong Provincial Hospital; Children's Hospital of Fudan University; Shanghai Children's Hospital; Chengdu Women's and Children's Central Hospital; Jiangxi Province Children's Hospital; Children's Hospital of Nanjing Medical University; Children's Hospital of The Capital Institute of Pediatrics; West China Second University Hospital, Sichuan University
• Investigators: Principal Investigator: Jin Wu, West China Second University Hospital, Sichuan University

Study record dates

Study Major Dates

• Study Start (Actual): September 23, 2022
• Primary Completion (Anticipated): February 1, 2025
• Study Completion (Anticipated): February 1, 2025

Study Registration Dates

• First Submitted: April 25, 2022
• First Submitted That Met QC Criteria: April 25, 2022
• First Posted (Actual): April 29, 2022

Study Record Updates

• Last Update Posted (Estimate): February 28, 2023
• Last Update Submitted That Met QC Criteria: February 26, 2023
• Last Verified: February 1, 2023

More Information

Terms related to this study

• Keywords: Achondroplasia
• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Musculoskeletal Diseases; Bone Diseases; Dwarfism; Bone Diseases, Developmental; Osteochondrodysplasias; Achondroplasia; Physiological Effects of Drugs; Hormones, Hormone Substitutes, and Hormone Antagonists; Hormones
• Other Study ID Numbers: GenSci001-01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Drugs belong to the sponsor before they are put into clinical trials and the site is only a contract research organization; It is an industry practice that intellectual property rights belong to the sponsor.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No