Small for Gestational Age InfantNCT05070234
Genetic Diagnosis and Human Growth Hormone Treatment in Small for Gestational Age Children With Short Stature
An observational study of Small for Gestational Age Infant and Silver-Russell Syndrome, run by Chunxiu Gong.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 to 20 years
- Conditions the study is for
- Small for Gestational Age Infant, Silver-Russell Syndrome, Genetic Diseases, Inborn
- What is being tested?
- Blood collection for genetic analysis
- How many people
- 150 participants
- Where
- No sites listed yet
- Study dates
- 11 Oct 2021to 30 Aug 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 20 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Children's hospical
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
No sites are listed yet.
About this study
Official title: 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.
- Being tested
- Blood collection for genetic analysis
- Medical subject headings
- Silver-Russell Syndrome, Genetic Diseases, Inborn
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Blood collection for genetic analysisGenetic
Genetic tests in this study are sequential testing, including MS-MLPA, whole exome sequencing, whole genome sequencing, as well as RNA-seq.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Genetic: Blood collection for genetic analysis |
What is being measured
The primary outcome is the main question the study is designed to answer.
Genetic testing
To detect the disease-causing genes of SGA children with short stature
Measured: At baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Oct 2021
- Study start11 Oct 2021 (estimated)
- Primary completion30 Aug 2022 (estimated)
- Study completion30 Aug 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Small for Gestational Age InfantStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 150 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chunxiu Gong
- Sponsor type
- University, hospital or other
- Collaborators
- Shanghai Children's Hospital, Second Affiliated Hospital of Guangzhou Medical University, Shenzhen Children's Hospital, Tongji Hospital, The Children's Hospital of Zhejiang University School of Medicine, Chengdu Women's and Children's Central Hospital, West China Second University Hospital, The First Affiliated Hospital with Nanjing Medical University, Changchun GeneScience Pharmaceutical Co., Ltd.
- Responsible party
- Sponsor-investigator, Chunxiu Gong, Beijing Children's Hospital
- Investigators
- Chunxiu Gong, doctor, Principal investigator, Beijing Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05070234
- Sponsor's protocol ID
- GenSci-GH-21016
- Status last verified
- Sep 2021
- First submitted
- 27 Sep 2021
- Last updated
- 7 Oct 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Oct 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Genetic Diagnosis and the Response to Recombinant Human Growth Hormone Treatment in Small for Gestational Age Children With Short Stature
- ClinicalTrials.gov ID
- NCT05070234
- Acronym / sponsor ID
- GenSci-GH-21016
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 150 participants (estimated)
- Lead sponsor
- Chunxiu GongUniversity, hospital or other
- Collaborators
- Shanghai Children's Hospital, Second Affiliated Hospital of Guangzhou Medical University, Shenzhen Children's Hospital, Tongji Hospital, The Children's Hospital of Zhejiang University School of Medicine, Chengdu Women's and Children's Central Hospital, West China Second University Hospital, The First Affiliated Hospital with Nanjing Medical University, Changchun GeneScience Pharmaceutical Co., Ltd.
- Study start
- 11 Oct 2021 (estimated)
- Primary completion
- 30 Aug 2022 (estimated)
- Study completion
- 30 Aug 2022 (estimated)
- First posted
- 7 Oct 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| short stature children born small for gestational age | – | Genetic: Blood collection for genetic analysisThis 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. |
Who may qualify
- Ages
- 2 to 20 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Children's hospical
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Blood collection for genetic analysisGenetic tests in this study are sequential testing, including MS-MLPA, whole exome sequencing, whole genome sequencing, as well as RNA-seq. | Genetic | short stature children born small for gestational age |
Primary outcome
Genetic testing
Time frame: At baseline
To detect the disease-causing genes of SGA children with short stature
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Oct 2021.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Chunxiu Gong, doctor principal investigator
Beijing Children's Hospital
Questions about this trial
What is this clinical trial studying?
An observational study of Small for Gestational Age Infant and Silver-Russell Syndrome, run by Chunxiu Gong.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 7 Oct 2021. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Bingyan Cao, doctor (13811748954, caoby1982@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Genetic testing (time frame: At baseline). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Oct 2021.