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

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

Status
UNKNOWN
Phases
Unknown
Study type
Observational
Source
ClinicalTrials.gov
Registry ID
NCT05070234
Enrollment
150
Registered
2021-10-07
Start date
2021-10-11
Completion date
2022-08-30
Last updated
2021-10-07

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

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

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.

Interventions

GENETICBlood 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.

Sponsors

Shanghai Children's Hospital
CollaboratorOTHER
Second Affiliated Hospital of Guangzhou Medical University
CollaboratorOTHER
Shenzhen Children's Hospital
CollaboratorOTHER_GOV
Tongji Hospital
CollaboratorOTHER
The Children's Hospital of Zhejiang University School of Medicine
CollaboratorOTHER
Chengdu Women's and Children's Central Hospital
CollaboratorOTHER
West China Second University Hospital
CollaboratorOTHER
The First Affiliated Hospital with Nanjing Medical University
CollaboratorOTHER
Changchun GeneScience Pharmaceutical Co., Ltd.
CollaboratorINDUSTRY
Chunxiu Gong
Lead SponsorOTHER

Study design

Observational model
COHORT
Time perspective
RETROSPECTIVE

Eligibility

Sex/Gender
ALL
Age
2 Years to 20 Years
Healthy volunteers
No

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.

Design outcomes

Primary

MeasureTime frameDescription
Genetic testingAt baselineTo detect the disease-causing genes of SGA children with short stature

Contacts

Primary ContactBingyan Cao, doctor
caoby1982@163.com13811748954
Backup ContactJiajia Chen, doctor
chenjiaj2009@126.com13810773718

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026