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

September 27, 2021 updated by: Chunxiu Gong

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

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.

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Anticipated)

150

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 20 years (ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Children's hospical

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
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.
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Genetic testing
Time Frame: At baseline
To detect the disease-causing genes of SGA children with short stature
At baseline

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Chunxiu Gong

Collaborators

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

Investigators

  • Principal Investigator: Chunxiu Gong, doctor, Beijing Children's Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ANTICIPATED)

October 11, 2021

Primary Completion (ANTICIPATED)

August 30, 2022

Study Completion (ANTICIPATED)

August 30, 2022

Study Registration Dates

First Submitted

September 27, 2021

First Submitted That Met QC Criteria

September 27, 2021

First Posted (ACTUAL)

October 7, 2021

Study Record Updates

Last Update Posted (ACTUAL)

October 7, 2021

Last Update Submitted That Met QC Criteria

September 27, 2021

Last Verified

September 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • GenSci-GH-21016

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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