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

Chunxiu Gong0 sites150 target enrollmentOctober 11, 2021

ConditionsSmall for Gestational Age Infant Silver-Russell Syndrome Genetic Diseases, Inborn

Overview

Phase: Not Applicable
Intervention: Not specified
Conditions: Small for Gestational Age Infant
Sponsor: Chunxiu Gong
Enrollment: 150
Primary Endpoint: Genetic testing
Last Updated: 4 years ago

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief 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.

Registry

clinicaltrials.gov

Start Date

October 11, 2021

End Date

August 30, 2022

Last Updated

4 years ago

Study Type

Observational

Sex

All

Investigators

Sponsor

Chunxiu Gong

Responsible Party

Sponsor Investigator

Principal Investigator

Chunxiu Gong

Department of Endocrinology, Genetics and Metabolism

Beijing Children's Hospital

Eligibility Criteria

Inclusion Criteria

•Single birth, clinical diagnosis of SGA;
•rhGH treatment beginned before puberty (Tanner stage I), regardless of gender;
•Before starting rhGH treatment, height was lower than -2 SDS compared with normal children of the same age and sex;
•All the subjects and their guardians signed the informed consent and the informed consent for genetic testing.

Exclusion Criteria

•No efficacy and safety data were recorded after treatment with rhGH;
•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;
•Other conditions that the investigator considered unsuitable for inclusion in this study.