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
1 other identifier
observational
150
0 countries
N/A
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for all trials
Started Oct 2021
Shorter than P25 for all trials
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
September 27, 2021
CompletedFirst Posted
Study publicly available on registry
October 7, 2021
CompletedStudy Start
First participant enrolled
October 11, 2021
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 30, 2022
CompletedStudy Completion
Last participant's last visit for all outcomes
August 30, 2022
CompletedOctober 7, 2021
September 1, 2021
11 months
September 27, 2021
September 27, 2021
Conditions
Outcome Measures
Primary Outcomes (1)
Genetic testing
To detect the disease-causing genes of SGA children with short stature
At baseline
Study Arms (1)
short stature children born small for gestational age
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.
Interventions
Genetic tests in this study are sequential testing, including MS-MLPA, whole exome sequencing, whole genome sequencing, as well as RNA-seq.
Eligibility Criteria
Children's hospical
You may qualify if:
- 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.
You may not qualify if:
- 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;
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Chunxiu Gonglead
- Shanghai Children's Hospitalcollaborator
- Second Affiliated Hospital of Guangzhou Medical Universitycollaborator
- Shenzhen Children's Hospitalcollaborator
- Tongji Hospitalcollaborator
- The Children's Hospital of Zhejiang University School of Medicinecollaborator
- Chengdu Women's and Children's Central Hospitalcollaborator
- West China Second University Hospitalcollaborator
- The First Affiliated Hospital with Nanjing Medical Universitycollaborator
- Changchun GeneScience Pharmaceutical Co., Ltd.collaborator
Biospecimen
whole blood
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Chunxiu Gong, doctor
Beijing Children's Hospital
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- RETROSPECTIVE
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR INVESTIGATOR
- PI Title
- Department of Endocrinology, Genetics and Metabolism
Study Record Dates
First Submitted
September 27, 2021
First Posted
October 7, 2021
Study Start
October 11, 2021
Primary Completion
August 30, 2022
Study Completion
August 30, 2022
Last Updated
October 7, 2021
Record last verified: 2021-09
Data Sharing
- IPD Sharing
- Will not share