Clinical trials

Necrotizing enterocolitis (NEC) is one of the most severe gastrointestinal emergencies in preterm infants, characterized by insidious onset, rapid progression, and high mortality. It can lead to serious adverse outcomes such as intestinal perforation, short bowel syndrome, and neurodevelopmental disorders, making it a critical condition that significantly impacts the survival quality and long-term prognosis of preterm infants.With the advancement of perinatal medicine in China, the survival rates of extremely low and very low birth weight infants have been continuously improving. NEC has become a critical bottleneck constraining the quality of care and long-term prognosis for preterm infants. Previous studies have demonstrated that various perinatal and early postnatal factors, including gestational age, birth weight, infection, feeding methods, blood transfusion, mechanical ventilation, and antibiotic exposure, are associated with the occurrence of NEC. However, these clinical factors still fail to adequately explain the interindividual variations in NEC incidence risk and disease severity under similar clinical exposure conditions.Existing NEC prediction models primarily rely on static baseline variables for one-time risk assessment, lacking dynamic risk updates during hospitalization, and most are derived from single-center retrospective studies. With the application of clinical exome sequencing (CES), the role of genetic factors in susceptibility to NEC has gradually attracted attention.The research team has previously conducted NEC risk gene screening based on CES, genetic burden analysis, and exploration of genetic-clinical factor interactions, suggesting that genetic information can provide important supplementation for NEC risk assessment.Meanwhile, dynamic changes in immune-inflammatory markers such as peripheral blood eosinophils, NLR, absolute neutrophil count, and platelet count may already exhibit abnormalities prior to the onset of NEC, providing repeatable, low-cost, and clinically available signals for early identification.Based on this, this study aims to establish a multidimensional dynamic early warning system for NEC integrating single-center preliminary genetic research foundations with multi-center retrospective/prospective validation resources. This initiative seeks to enhance the identification capability of high-risk individuals and provide evidence for subsequent precision prevention and control as well as stratified management.

This is a single-arm, open-label, single-dose, dose-escalation trial that plans to enrol 3-18 patients with transfusion-dependent β-thalassaemia (TDT) or sickle-cell disease (SCD). Its primary aims are to evaluate the safety and tolerability of a single administration of YOLT-204 and to obtain preliminary data on its effect on plasma fetal-haemoglobin levels. The main-study screening period may last up to 60 days; the treatment day is Day 0 (D0). Safety follow-up continues through Week 52 post-dose. After completion of the main study, participants will enter long-term follow-up extending to 15 years post-dose.

The goal of this observational study is to learn how brain activity in children with autism spectrum disorder (ASD) differs from typical developing children using magnetoencephalography (MEG). The main questions this study aims to answer are: Do children with ASD show different patterns of neural oscillation compared to typically developing children? Can MEG identify specific brain features that help ASD diagnosis? Participants will undergo MEG scan in both resting and tasking state, and their brain activity will be analyzed for characteristic neural oscillations and connectivity patterns. This study may help develop better tools for early ASD diagnosis.

The goal of this observational study is to evaluate the effectiveness of Magnetoencephalography (MEG) in the preoperative assessment of children with refractory epilepsy, particularly in identifying the seizure focus, and to compare it with the gold standard methods of Stereo-electroencephalography (SEEG) and Electrocorticography (ECoG). The main questions this study aims to answer are: 1. How consistent is MEG with SEEG/ECoG in locating the epileptic focus in children with refractory epilepsy? 2. What is the clinical efficacy of MEG in identifying epileptic foci? Participants will: 1. Be divided into 2 groups: the case group and the control group. Participants in the case group will undergo both MEG and SEEG/ECoG for preoperative assessment, while patients in the control group will undergo only SEEG/ECoG before surgery. 2. Be followed up at 1, 3, 6, and 12 months after surgery to assess seizure control, neurological function, and quality of life.

This observational study aims to evaluate the effectiveness of magnetoencephalography (MEG) in the preoperative assessment of pediatric brain tumors, particularly in determining the extent of resection, and compare it with computed tomography (CT) and magnetic resonance imaging (MRI). The main questions this study aims to answer are: What is the consistency between MEG and CT/MRI in localizing pediatric brain tumors? What is the clinical efficacy of MEG in determining the extent of resection of pediatric brain tumors preoperatively? Participants will: Undergo both MEG and CT/MRI examinations for preoperative assessment, and the extent of tumor resection will be determined by the comprehensive results of the above examinations. Be followed up with MEG and CT/MRI at 1, 3, and 6 months after surgery, along with neurological and quality-of-life assessments.

This is a prospective, single-arm, open, single-center clinical trial to evaluate the safety and efficacy of autologous NK cells combined with chemotherapy and GD2 monoclonal antibody in the treatment of newly diagnosed high-risk or relapsed/refractory neuroblastoma in children. Fifteen eligible subjects are planned to be included. The objective is to evaluate the safety and efficacy of autologous NK cells combined with GD2 monoclonal antibody in the treatment of children with newly diagnosed high-risk or relapsed/refractory neuroblastoma, which is expected to be safe and effective in improving PFS and DCR in children with refractory/recurrent neuroblastoma.

The long-term goal of the Human Genes and Microbiota in Early Life (HuGME) is to explore the short- and long-term effects of maternal microbiota during pregnancy and the microbiota colonization of their offspring early in life and their interaction with the host on maternal-offspring health consequences in later life in the born in Guangzhou cohort study in China. Identification of the effect of microbiota in early life, as well as environmental factors and microbe-host interaction, will lead to a better understanding of disease pathogenesis and provide a foundation for targeted mechanistic investigation into the consequences of microbial-host crosstalk for long-term health. It also can result in new strategies to predict and prevent diseases in later life.

The main objective of this study is to test the efficacy of an intervention package (dietary, physical and lifestyle modification) during pregnancy in improving the clinical outcomes of mother-infant dyads.

The goal of this prospective, open, single-arm clinical trial was to evaluate the safety and potential efficacy of CAR T cell therapy in children with refractory/recurrent lupus nephritis. The persistence and cell phenotype of CAR-T cells in vivo and CAR-T treatment-related inflammatory factors were evaluated after treatment. To explore new therapeutic methods, in order to reduce the side effects of traditional therapeutic drugs, increase curative effect, and finally make patients obtain long-term survival and improve survival quality.

The goal of this observational study is to conduct a multicenter, prospective neonatal acute kidney injury (AKI) follow-up cohort to describe the clinical characteristics of chronic kidney disease (CKD) in AKI cases within 1 year of follow-up and screen for effective biomarkers that can early warn of CKD progression. Ultimately, a neonatal AKI adverse prognosis warning model based on multidimensional data will be established to provide new data and new models for CKD management.

The goal of this clinical trial is to evaluate the safety and efficacy of human umbilical cord mesenchymal stem cells (hUC-MSC) in the treatment of Alport syndrome (AS) in a randomized, single-blind, placebo-controlled trial, to provide a clinical basis for the development of stem cell products for the treatment of AS, and to further clarify the therapeutic effect of hUC-MSC in the treatment of AS.

In this study, CD19/CD22 dual-target CAR-T therapy will be carried out among children patients who are still positive after induction remission, and subsequent chemotherapy will continue after CAR-T cells exert their functions. This study intends to use retroviral vector-based tandem CAR-T cells targeting CD19/CD22 to treat MRD-positive ALL. The CAR-T cells were provided by Shenzhen Cell Valley. The results of the research team from Stanford University School of Medicine in the United States have already demonstrated the feasibility and safety of producing bispecific CD19/CD22.BB.z-CAR T cells in a closed system as well as the high clinical activity shown in the treatment of CAR19-resistant B-ALL (B-lineage acute lymphoblastic leukemia) and LBCL (Large B-cell lymphoma). The investigators look forward to expanding the application of CAR-T cells in MRD positive B-ALL through this clinical study on safety and efficacy and greatly improving the prognosis of children patients with this type of B-ALL.

This is a single-arm, open label, phase 1 study in subjects with beta-thalassemia. This study will evaluate the safety and efficacy of preconditioning-free super-transplantation on beta-thalassemia pediatric patients.

This is a prospective, open-label, multicenter, single-arm clinical study designed to compare the efficacy and safety of neoadjuvant treatment with T-DXd in combination with an immune checkpoint inhibitor in patients with primary intermediate- to high-risk HR-positive, HER2-overexpressing early-stage breast cancer.

The MGBIG is a study exploring cross-generational effects in 5000 women and their children and grandchildren located in Guangzhou, China. Its initial aim is to facilitate research on understanding the interplay between genes and environmental factors on disease etiology and on explaining the cause of the disease from the perspective of "grandmother-mother-child" heredity. Data are collected regarding environmental exposures, lifestyle, and social support on grandmother from birth to old age. Biological samples including blood and tissue samples are collected.

To explore the efficacy of adding anti-GD2 immunotherapy (Naxitamab) at the stage of induction chemotherapy for newly diagnosed high risk neuroblastoma patients. To investigate wether anding Naxitamab in the induction phase will improve the response rate at the end of induction therapy and further improve the overall survival rate.

The Offspring Born to Mothers with Polycystic Ovary Syndrome in Guangzhou Cohort study (PCOS-BIG) was established to investigate the short- and long-term effects of intrauterine exposure to maternal PCOS on the health of offspring in Guangzhou, China. Data are collected regarding maternal PCOS subtypes, nursing, diet and education as well as health outcomes in their later life. Biological samples including blood and tissue samples are also collected from participants.

The Preterm Birth Cohort Study in Guangzhou (PBCSG) aims to explore the impact of genetic and environmental factors including life styles on preterm birth, to examine the interaction effect between these factors, and to follow up the short-term and long-term outcomes of preterm childrens.

The IVF Offspring Born in Guangzhou Cohort Study (IVF-BIG) was established to investigate the short- and long-term effects of exposure in early life on the health of mothers and offspring in Guangzhou, China. Data are collected regarding assisted reproductive technology (ART), environmental, occupational and lifestyle exposures as well as health outcomes in their later life. Biological samples including blood and tissue samples are also collected from participants.

The 2-BIG is a twin birth cohort study located in Guangzhou, China.Its initial aim is to facilitate research on understanding the interplay between genes and environmental factors on disease etiology. Data are collected regarding environmental, family and lifestyle exposures on twins from birth to 18 years old. Biological samples including blood and tissue samples are also collected from the twins and their parents.

The Born in Guangzhou Cohort Study (BIGCS) was established to investigate the short- and long-term effects of exposure in early life on health consequences in Guangzhou, China. Data are collected regarding environmental, occupational and lifestyle exposures as well as health outcomes in later life. Biological samples including blood and tissue samples are also collected from the participants.

Gestational diabetes mellitus (GDM) is one of the most common disorders which occured during pregnancy. GDM is not only associated with short-term maternal and fetal adverse outcomes, but also related to a wide range of long-term consequences for both mother and child. The GDM and Its Consequences for mothers and offsprings (GDMCMO) aims to establish a cohort to follow both maternal and offsprings'short-term and long-term outcomes, including fetal malformations including congenital heart diseases, birth weight, preterm birth, caesarean section delivery, body growth and neurodevelopment after birth, obesity, type 2 diabetes and impaired insulin sensitivity and secretion, lung health and allergic diseases later in life for offspring, as well as future type 2 diabetes and cardiovascular risk factors for mother after delivery. Biological samples including blood and tissue samples of mothers and children are also collected during pregnancy and after delivery.

The purpose of this study is to determine the incidence,development trajectory and risk factors of the main peripartum mental health problems as well as explore its adverse outcomes.

In this prospective study, we tried to select the operation time according to the cyst size and evaluate the treatment effect.

The purpose of this prospective cohort study is to build a large platform that includes clinical information (prenatal diagnosis and postnatal follow-up data) and biological specimen banks of fetuses/infants with IUGR or congenital anomalies, which provide vital support and research foundation for accurate diagnosis, precision treatment and meticulous management.