Genetic Diagnosis and Human Growth Hormone Treatment in Small for Gestational Age Children With Short Stature

Genetic Diseases, Inborn Clinical Trial

Official title:

Genetic Diagnosis and the Response to Recombinant Human Growth Hormone Treatment in Small for Gestational Age Children With Short Stature

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT05070234
• Other study ID #: GenSci-GH-21016
• Status: Not yet recruiting
• Start date: October 11, 2021
• Est. completion date: August 30, 2022

Study information

• Verified date: September 2021
• Source: Beijing Children's Hospital
• Contact: Bingyan Cao, doctor
• Phone: 13811748954
• Email: caoby1982[@]163.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

This study is a multi-center, retrospective and non-interventional research. In this study, a total of 150 short children who were small for gestational age and had been treated with recombinant human growth hormone (rhGH) are selected for genetic testing. The aims of this study are to analyze the genetic etiology of SGA children with short stature, and to compare the efficacy and safety of rhGH treatment in subjects with different etiologies.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 150
• Est. completion date: August 30, 2022
• Est. primary completion date: August 30, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 20 Years

Eligibility

Inclusion Criteria:

1. Single birth, clinical diagnosis of SGA;

2. rhGH treatment beginned before puberty (Tanner stage I), regardless of gender;

3. Before starting rhGH treatment, height was lower than -2 SDS compared with normal children of the same age and sex;

4. All the subjects and their guardians signed the informed consent and the informed consent for genetic testing.

Exclusion Criteria:

1. No efficacy and safety data were recorded after treatment with rhGH;

2. A history of blood transfusion within 3 months before the collection of the genetic blood samples, or a history of bone marrow transplantation between rhGH treatment and the enrollment in this study;

3. Other conditions that the investigator considered unsuitable for inclusion in this study.

Related Conditions & MeSH terms

• Dwarfism
• Genetic Diseases, Inborn
• Small for Gestational Age Infant

Intervention

Genetic:
• Blood collection for genetic analysis
Genetic tests in this study are sequential testing, including MS-MLPA, whole exome sequencing, whole genome sequencing, as well as RNA-seq.

Locations

Country	Name	City	State
n/a

Sponsors (10)

Lead Sponsor

Collaborator

Chunxiu Gong

Chengdu Women's and Children's Central Hospital, GeneScience Pharmaceuticals Co., Ltd., Second Affiliated Hospital of Guangzhou Medical University, Shanghai Children's Hospital, Shenzhen Children's Hospital, The Children's Hospital of Zhejiang University School of Medicine, The First Affiliated Hospital with Nanjing Medical University, Tongji Hospital, West China Second University Hospital, Sichuan University

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Genetic testing	To detect the disease-causing genes of SGA children with short stature	At baseline