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

Sponsor

GeneScience Pharmaceuticals Co., Ltd. (Industry)

Overall Status

Not yet recruiting

CT.gov ID

NCT05353192

Collaborator

Children's Hospital of Fudan University (Other), Tongji Hospital (Other), Children's Hospital of Nanjing Medical University (Other), Jiangxi Province Children's Hospital (Other), Chengdu Women's and Children's Central Hospital (Other), Shandong Provincial Hospital (Other), Shengjing Hospital (Other), Shanghai Children's Hospital (Other), Children's Hospital of The Capital Institute of Pediatrics (Other)

Enrollment

Location

Arm

30.1

Anticipated Duration (Months)

1.3

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
Achondroplasia	Drug: Recombinant human growth hormone	Phase 4

Study Design

Study Type:

Interventional

Anticipated Enrollment :

38 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

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

Anticipated Study Start Date :

Aug 1, 2022

Anticipated Primary Completion Date :

Feb 1, 2025

Anticipated Study Completion Date :

Feb 1, 2025

Arms and Interventions

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

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

Outcome Measures

Primary Outcome Measures

Change From Baseline in Annualized Height Velocity (AHV) at Week 52 [week 52]
The change in AHV after 52 weeks treatment

Secondary Outcome Measures

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

Other Outcome Measures

Change From Baseline in Metabolism-related Proteins [week 13, week 26, week 52]
Change From Baseline in Serum Proteomics at week 13, week 26, week 52

Eligibility Criteria

Criteria

Ages Eligible for Study:

2 Years to 10 Years

Sexes Eligible for Study:

All

Accepts 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Children's Hospital of Fudan University	Shanghai	Shanghai	China	201102

Investigators

Principal Investigator: Feihong Luo, Children's Hospital of Fudan University
Principal Investigator: Xiaoping Luo, Tongji Hospital
Principal Investigator: Wei Gu, Children's Hospital of Nanjing Medical University
Principal Investigator: Yu Yang, Jiangxi Province Children's Hospital
Principal Investigator: Xinran Cheng, Chengdu Women's and Children's Central Hospital
Principal Investigator: Guimei Li, Shandong Provincial Hospital
Principal Investigator: Ying Xin, Shengjing Hospital of China Medical Hospital
Principal Investigator: Pin Li, Shanghai Children's Hospital
Principal Investigator: Xiaobo Chen, Children's Hospital of The Capital Institute of Pediatrics

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

GeneScience Pharmaceuticals Co., Ltd.

ClinicalTrials.gov Identifier:

NCT05353192

Other Study ID Numbers:

GenSci001-01

First Posted:

Apr 29, 2022

Last Update Posted:

Apr 29, 2022

Last Verified:

Feb 1, 2022

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Keywords provided by GeneScience Pharmaceuticals Co., Ltd.

Achondroplasia

Additional relevant MeSH terms: