Ignite Creation Date:
2025-12-25 @ 2:30 AM
Ignite Modification Date:
2025-12-26 @ 6:52 PM
Study NCT ID:
NCT05070234
Status:
UNKNOWN
Last Update Posted:
2021-10-07
First Post:
2021-09-27
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Genetic Diagnosis and Human Growth Hormone Treatment in Small for Gestational Age Children With Short Stature
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D056730', 'term': 'Silver-Russell Syndrome'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}], 'ancestors': [{'id': 'D019465', 'term': 'Craniofacial Abnormalities'}, {'id': 'D009139', 'term': 'Musculoskeletal Abnormalities'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D000015', 'term': 'Abnormalities, Multiple'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D025063', 'term': 'Chromosome Disorders'}, {'id': 'D004392', 'term': 'Dwarfism'}, {'id': 'D000096803', 'term': 'Imprinting Disorders'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D005820', 'term': 'Genetic Testing'}], 'ancestors': [{'id': 'D019411', 'term': 'Clinical Laboratory Techniques'}, {'id': 'D019937', 'term': 'Diagnostic Techniques and Procedures'}, {'id': 'D003933', 'term': 'Diagnosis'}, {'id': 'D008919', 'term': 'Investigative Techniques'}, {'id': 'D005821', 'term': 'Genetic Techniques'}, {'id': 'D033142', 'term': 'Genetic Services'}, {'id': 'D006296', 'term': 'Health Services'}, {'id': 'D005159', 'term': 'Health Care Facilities Workforce and Services'}, {'id': 'D003954', 'term': 'Diagnostic Services'}, {'id': 'D011314', 'term': 'Preventive Health Services'}]}}, 'protocolSection': {'designModule': {'bioSpec': {'retention': 'SAMPLES_WITH_DNA', 'description': 'whole blood'}, 'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'RETROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 150}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'NOT_YET_RECRUITING', 'startDateStruct': {'date': '2021-10-11', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-09', 'completionDateStruct': {'date': '2022-08-30', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2021-09-27', 'studyFirstSubmitDate': '2021-09-27', 'studyFirstSubmitQcDate': '2021-09-27', 'lastUpdatePostDateStruct': {'date': '2021-10-07', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2021-10-07', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-08-30', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Genetic testing', 'timeFrame': 'At baseline', 'description': 'To detect the disease-causing genes of SGA children with short stature'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Small for Gestational Age Infant', 'Silver-Russell Syndrome', 'Genetic Diseases, Inborn']}, 'descriptionModule': {'briefSummary': '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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '20 Years', 'minimumAge': '2 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': "Children's hospical", 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Single birth, clinical diagnosis of SGA;\n2. rhGH treatment beginned before puberty (Tanner stage I), regardless of gender;\n3. Before starting rhGH treatment, height was lower than -2 SDS compared with normal children of the same age and sex;\n4. All the subjects and their guardians signed the informed consent and the informed consent for genetic testing.\n\nExclusion Criteria:\n\n1. No efficacy and safety data were recorded after treatment with rhGH;\n2. 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;\n3. Other conditions that the investigator considered unsuitable for inclusion in this study.'}, 'identificationModule': {'nctId': 'NCT05070234', 'briefTitle': 'Genetic Diagnosis and Human Growth Hormone Treatment in Small for Gestational Age Children With Short Stature', 'organization': {'class': 'OTHER', 'fullName': "Beijing Children's Hospital"}, 'officialTitle': 'Genetic Diagnosis and the Response to Recombinant Human Growth Hormone Treatment in Small for Gestational Age Children With Short Stature', 'orgStudyIdInfo': {'id': 'GenSci-GH-21016'}}, 'armsInterventionsModule': {'armGroups': [{'label': '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.', 'interventionNames': ['Genetic: Blood collection for genetic analysis']}], 'interventions': [{'name': 'Blood collection for genetic analysis', 'type': 'GENETIC', 'description': 'Genetic tests in this study are sequential testing, including MS-MLPA, whole exome sequencing, whole genome sequencing, as well as RNA-seq.', 'armGroupLabels': ['short stature children born small for gestational age']}]}, 'contactsLocationsModule': {'centralContacts': [{'name': 'Bingyan Cao, doctor', 'role': 'CONTACT', 'email': 'caoby1982@163.com', 'phone': '13811748954'}, {'name': 'Jiajia Chen, doctor', 'role': 'CONTACT', 'email': 'chenjiaj2009@126.com', 'phone': '13810773718'}], 'overallOfficials': [{'name': 'Chunxiu Gong, doctor', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': "Beijing Children's Hospital"}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Chunxiu Gong', 'class': 'OTHER'}, 'collaborators': [{'name': "Shanghai Children's Hospital", 'class': 'OTHER'}, {'name': 'Second Affiliated Hospital of Guangzhou Medical University', 'class': 'OTHER'}, {'name': "Shenzhen Children's Hospital", 'class': 'OTHER_GOV'}, {'name': 'Tongji Hospital', 'class': 'OTHER'}, {'name': "The Children's Hospital of Zhejiang University School of Medicine", 'class': 'OTHER'}, {'name': "Chengdu Women's and Children's Central Hospital", 'class': 'OTHER'}, {'name': 'West China Second University Hospital', 'class': 'OTHER'}, {'name': 'The First Affiliated Hospital with Nanjing Medical University', 'class': 'OTHER'}, {'name': 'Changchun GeneScience Pharmaceutical Co., Ltd.', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR_INVESTIGATOR', 'investigatorTitle': 'Department of Endocrinology, Genetics and Metabolism', 'investigatorFullName': 'Chunxiu Gong', 'investigatorAffiliation': "Beijing Children's Hospital"}}}}