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

Chunxiu Gong

Status: Unknown

Conditions: Silver-Russell Syndrome; Small for Gestational Age Infant; Genetic Diseases, Inborn

Treatments: Genetic: Blood collection for genetic analysis

Study type: Observational

Funder types: Other; Industry

Identifiers

NCT05070234

GenSci-GH-21016

Details and patient eligibility

About

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.

Enrollment: 150 estimated patients

Sex: All

Ages: 2 to 20 years old

Volunteers: No Healthy Volunteers

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.

Trial design

150 participants in 1 patient group

short stature children born small for gestational age

Description:

This group was defined as a group of children whose birth weight and/or birth length equal or less than -2 SD for sex and gestational age, and who had failed to catch up in growth, remaining short after 2 years old.

Treatment:

Genetic: Blood collection for genetic analysis