Back to results

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

Primary Purpose

Achondroplasia

Status

Recruiting

Phase

Phase 4

Locations

China

Study Type

Interventional

Intervention

Recombinant human growth hormone

About this trial

This is an interventional treatment trial for Achondroplasia focused on measuring Achondroplasia

Eligibility Criteria

2 Years - 10 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Children's Hospital of Fudan UniversityRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Recombinant human growth hormone

Arm Description

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

Outcomes

Primary Outcome Measures

Change From Baseline in Annualized Height Velocity (AHV) at Week 52

The change in AHV after 52 weeks treatment

Secondary Outcome Measures

Change From Baseline in AHV

Change in AHV at week, 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

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

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

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

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

Change From Baseline in Bone Age/Chronological Age ratio (BA/CA)

Change From Baseline in BA/CA at week 52

Full Information

NCT ID

NCT05353192

First Posted

April 25, 2022

Last Updated

February 26, 2023

Sponsor

Changchun GeneScience Pharmaceutical Co., Ltd.

Collaborators

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

1. Study Identification

Unique Protocol Identification Number

NCT05353192

Brief Title

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

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

Study Type

Interventional

2. Study Status

Record Verification Date

February 2023

Overall Recruitment Status

Recruiting

Study Start Date

September 23, 2022 (Actual)

Primary Completion Date

February 2025 (Anticipated)

Study Completion Date

February 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Changchun GeneScience Pharmaceutical Co., Ltd.

Collaborators

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

Brief Summary

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Achondroplasia

Keywords

Achondroplasia

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 4

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

38 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Recombinant human growth hormone

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

Recombinant human growth hormone

Intervention Description

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

Primary Outcome Measure Information:

Title

Change From Baseline in Annualized Height Velocity (AHV) at Week 52

Description

The change in AHV after 52 weeks treatment

Time Frame

week 52

Secondary Outcome Measure Information:

Title

Change From Baseline in AHV

Description

Change in AHV at week, week 13, week 26, week 39

Time Frame

week 4, week 13, week 26, week 39

Title

Change From Baseline in Height Standard Deviation Score (Ht SDS)

Description

Change From Baseline in Ht SDS at week 4, week 13, week 26, week 39, week 52

Time Frame

week 4, week 13, week 26, week 39, week 52

Title

Change From Baseline in BMI Standard Deviation Score (BMI SDS)

Description

Change From Baseline in BMI SDS at week 4, week 13, week 26, week 39, week 52

Time Frame

week 4, week 13, week 26, week 39, week 52

Title

Change From Baseline in Sitting Height/Leg Length ratio Standard Deviation Score (SH/LL SDS)

Description

Change From Baseline in SH/LL SDS at week 4, week 13, week 26, week 39, week 52

Time Frame

week 4, week 13, week 26, week 39, week 52

Title

Change From Baseline in IGF-1 Standard Deviation Score (IGF -1 SDS)

Description

Change From Baseline in IGF-1 SDS at week 4, week 13, week 26, week 39, week 52

Time Frame

week 4, week 13, week 26, week 39, week 52

Title

Change From Baseline in IGF-1/IGFBP-3 ratio

Description

Change From Baseline in IGF-1/IGFBP-3 ratio at week 4, week 13, week 26, week 39, week 52

Time Frame

week 4, week 13, week 26, week 39, week 52

Title

Change From Baseline in Bone Age/Chronological Age ratio (BA/CA)

Description

Change From Baseline in BA/CA at week 52

Time Frame

week 52

Other Pre-specified Outcome Measures:

Title

Change From Baseline in Metabolism-related Proteins

Description

Change From Baseline in Serum Proteomics at week 13, week 26, week 52

Time Frame

week 13, week 26, week 52

10. Eligibility

Sex

All

Minimum Age & Unit of Time

2 Years

Maximum Age & Unit of Time

10 Years

Accepts Healthy Volunteers

Eligibility Criteria

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

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Yanru Wu

Phone

+86 15843110489

wuyanru@gensci-china.com

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Feihong Luo

Organizational Affiliation

Children's Hospital of Fudan University

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Xiaoping Luo

Organizational Affiliation

Tongji Hospital

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Wei Gu

Organizational Affiliation

Children's Hospital of Nanjing Medical University

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Yu Yang

Organizational Affiliation

Jiangxi Province Children's Hospital

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Xinran Cheng

Organizational Affiliation

Chengdu Women's and Children's Central Hospital

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Guimei Li

Organizational Affiliation

Shandong Provincial Hospital

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Ying Xin

Organizational Affiliation

Shengjing Hospital of China Medical Hospital

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Pin Li

Organizational Affiliation

Shanghai Children's Hospital

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Xiaobo Chen

Organizational Affiliation

Children's Hospital of The Capital Institute of Pediatrics

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Jin Wu

Organizational Affiliation

West China Second University Hospital

Official's Role

Principal Investigator

Facility Information:

Facility Name

Children's Hospital of Fudan University

City

Shanghai

State/Province

Shanghai

ZIP/Postal Code

201102

Country

China

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Feihong Luo

Phone

18017590900

luo_fh@163.com

12. IPD Sharing Statement

Plan to Share IPD

IPD Sharing 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.

Learn more about this trial

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

We'll reach out to this number within 24 hrs