Clinical trials

The goal of this observational study is to learn about the safety and effects of atorvastatin in treatment of Chinese Kawasaki disease (KD) children complicated with coronary artery abnormalities (CAA) in acute phase. The main questions it aims to answer are: Is atorvastatin safe in Chinese children of acute KD? Does atorvastatin contribute to control the acute inflammation in KD and improve the CAA?

Precocious puberty is characterized by the premature appearance of secondary sexual characteristics. Globally, the timing of puberty onset in children has shown a certain tendency to advance. In China, the incidence of precocious puberty has been increasing year by year. Precocious puberty exerts long-term and systemic impacts on children's health: advanced bone age leads to short stature; earlier sexual development than peers may induce emotional problems such as anxiety and inferiority; it may increase the risk of obesity and type 2 diabetes, posing long-term hazards to cardiovascular health; it may also result in irregular menstruation or dysmenorrhea, exerting indirect effects on reproductive health. Modern traditional Chinese medicine (TCM) holds that: various factors lead to liver-kidney yin deficiency, hyperactivity of ministerial fire, and early arrival of tian gui (the substance responsible for promoting growth, development and reproduction), thereby triggering premature sexual development. The main syndrome types identified in clinical practice include yin deficiency with fire hyperactivity syndrome, liver stagnation transforming into fire syndrome, and phlegm-dampness internal accumulation syndrome. Since the late 1970s, the investigators' department has taken the lead in treating precocious puberty with TCM diagnostic methods, proposing that the pathogenesis of precocious puberty lies in "kidney yin deficiency and hyperactivity of ministerial fire", and adopting the therapy of nourishing yin and purging fire for its treatment. A number of studies have confirmed that TCM medicines with the effects of nourishing yin and purging fire can effectively alleviate the yin deficiency with fire hyperactivity syndrome in children, delay the development of secondary sexual characteristics and bone age. At present, central precocious puberty is mostly treated with gonadotropin-releasing hormone analogs (GnRHa). However, this treatment has the drawback of inhibiting the growth axis, yielding limited benefits for children with advanced bone age, overweight or obesity, and may even affect glucose and lipid metabolism. Moreover, some children with precocious puberty complicated with obesity may be intolerant to this therapy. In contrast, TCM therapy and integrated TCM-Western medicine therapy can effectively delay the development of secondary sexual characteristics and advanced bone age, and improve final adult height, thus being widely applied in China. Although a large number of relevant studies have been reported in recent years and TCM diagnosis and treatment guidelines for precocious puberty have been formulated, there is still a lack of high-quality evidence-based medical research to support the advantageous aspects of integrated TCM-Western medicine diagnosis and treatment. Additionally, the underlying mechanisms of diagnosis and treatment for different syndrome types of precocious puberty remain insufficiently studied. In this study, the investigators will conduct a multicenter, randomized, double-blind, placebo-controlled clinical trial to evaluate the effects of Sanghe Jianghuo Granules on the regulation of the hypothalamic-pituitary-gonadal (HPG) axis, metabolic homeostasis and inflammatory microenvironment, so as to verify its efficacy and safety. Furthermore, combined with transcriptomics, proteomics and network pharmacology, the investigators will identify the key targets and action pathways of Sanghe Jianghuo Granules, and verify its regulatory effect on the HPG axis through in vivo and in vitro experiments.

Child-parent screening for familial hypercholesterolemia has been proposed to identify children and their parent who are carrier of mutations and with high risk for inherited premature coronary artery disease. The investigators assessed the efficacy and feasibility of such screening in primary care practice. key scientific questions: 1. The 95th and 99th percentile of finger blood TC in children of 2 years old. 2. Mutations that contribute to high TC status ( serum TC \>99th percentiles) compared with international FH48 panel for FH genetic screening.

The goal of this pilot randomized controlled trial is to evaluate the feasibility and preliminary efficacy of a child life-based breathing exercise program for children recovering from severe pneumonia. The main questions it aims to answer are: * Is the child life-based breathing exercise program feasible for hospitalized children recovering from severe pneumonia? * Can the program improve caregiver satisfaction and promote recovery in children with severe pneumonia? Researchers will compare a child life-based breathing exercise program combined with routine health education with routine health education alone to determine whether the intervention improves rehabilitation outcomes. Participants will: * Receive either routine health education alone or routine health education combined with the child life-based breathing exercise program * Participate in breathing exercise training during hospitalization * Complete assessments of caregiver satisfaction and clinical recovery outcomes, including improvement in chest imaging findings and time to resolution of symptoms such as cough, fever, and dyspnea.

The purpose of this study is to compare the effect of high and ordinary energy density enteral nutrition for improving physical growth and brain cognitive development in infants with congenital heart disease after operation, as well as evaluate the safety of interventions.

The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-206 injection in treating sickle cell disease.

In contemporary clinical practice concerning the administration of inhaled nitric oxide (iNO) within neonatal intensive care units (NICUs), a stepwise reduction approach is frequently employed in accordance with established neonatal guidelines. Nonetheless, the comparative advantages of a linear reduction strategy-characterized by a gradual decrease in inhalation concentration-over the traditional stepwise method in terms of efficacy and safety have yet to be conclusively established. Furthermore, existing iNO delivery devices necessitate that healthcare professionals manually adjust parameters at intervals dictated by the patient's condition. This reliance on subjective clinical judgment often results in variability and a lack of standardization in the duration of the weaning process. Additionally, the protracted and intricate nature of the weaning procedure considerably heightens the workload for healthcare staff. Importantly, the development of a scientifically grounded, standardized, and real-time feedback mechanism for weaning may enhance clinical outcomes for patients and mitigate the risks associated with inappropriate weaning practices or inconsistent manual interventions. Consequently, this study seeks to leverage the newly introduced "intelligent closed-loop weaning" feature of the latest generation of iNO devices to facilitate automated linear concentration reduction during the weaning process. This innovation aims to alleviate the burden on healthcare personnel while establishing a more standardized and scientifically robust weaning protocol. However, it is noteworthy that there is currently a lack of clinical evidence, both domestically and internationally, regarding the safety and efficacy of this device's weaning protocol, underscoring the urgent need to validate its safety and effectiveness in real-world clinical settings.

This study is non-inferiority trial design. This study aimed to investigate the effect of prophylactic oral antibiotics on preventing cholangitis in biliary atresia (BA) patients after Kasai portoenterostomy (KP) by comparing the cholangitis rate in BA patients who received prophylactic oral antibiotics and those who did not. The patients were followed up for 2 years after KP.

This is a single-arm, open-label, single-center, exploratory clinical trial evaluating the safety and efficacy of decitabine in male patients aged 1 month to 18 years with X-linked magnesium transporter 1 (MAGT1) deficiency. Eligible patients have a confirmed MAGT1 gene mutation leading to XMEN disease ( X-linked MAGT1 deficiency with increased susceptibility to Epstein-Barr virus (EBV) infection and N-linked glycosylation defect). The study will assess changes in liver function, immune function, and NKG2D expression, as well as adverse events, over four treatment cycles and the follow-up period.

A clinical quality improvement bundle on early nutrition supplementation is developed to improving the clinical outcomes (including growth, organ function, and neurodevelopment outcomes) of extremely and/or very preterm infants. This bundle consists of three aspects: individualized and precise human milk feeding, early enteral zinc supplementation, and routine parenteral carnitine supplementation.

This study is a prospective, single center and observational open clinical study.

This study is a prospective, single center, practical, and observational open clinical study.

This study is a single-center observational clinical study, which evaluates the diagnostic value of magnetoencephalography (MEG) in the diagnosis of neurodevelopmental diseases in children, such as the localization of epileptic foci and brain functional areas, intracranial tumors, cerebrovascular diseases, autism, mental retardation, and neuropsychiatric disorders.

The aim of this study is to early detect kidney disease in the natural population cohort of children by urine and ultrasound screening, to assist in the precise prevention and treatment of children's kidney disease, and to establish a risk prediction system for children's kidney disease. About 10,000 children called KunQi Cohort are born in Jiangsu Province(8,000 in Kunshan and 2,000 in Qidong) and about 3,000 born in Shanghai. Through the project, child who is found with abnormal urine or ultrasound result will be referred to Children's Hospital of Fudan University to get further examination and treatment.

To investigate the role of preoperative serum FGF19 level in the prognosis of biliary atresia.

Prader-Willi syndrome (PWS) is a rare genetic disease, with hyperappetite and severe obesity. At present, there is no effective drugs and interventions to help control the appetite of PWS patients. More and more evidence has shown that gut microbiota is closely related to obesity. Probiotics and prebiotics can improve the structure of gut microbiota, thus improve blood lipid levels and other biochemical indicators of obese people. Therefore, this study intends to explore the effectiveness and safety of probiotics and prebiotics in controlling appetite and weight gain of PWS children.

This study will seek to determine if whole genome sequencing (WGS) improves diagnostic rates, and outcomes for congenital diarrhea and enteropathy (CODE) patients. The investigator will enroll 180 patients in a randomized controlled study to either WGS or whole exome sequencing (WES). This study is designed to evaluate whether CODE patients would benefit from WGS guided precision medicine.

Precocious puberty is characterized by the premature appearance of secondary sexual characteristics. Globally, the timing of puberty onset in children has shown a certain tendency to advance. In China, the incidence of precocious puberty has been increasing year by year. Precocious puberty exerts long-term and systemic impacts on children's health: advanced bone age leads to short stature; earlier sexual development than peers may induce emotional problems such as anxiety and inferiority; it may increase the risk of obesity and type 2 diabetes, posing long-term hazards to cardiovascular health; it may also result in irregular menstruation or dysmenorrhea, exerting indirect effects on reproductive health. Modern traditional Chinese medicine (TCM) holds that: various factors lead to liver-kidney yin deficiency, hyperactivity of ministerial fire, and early arrival of tian gui (the substance responsible for promoting growth, development and reproduction), thereby triggering premature sexual development. The main syndrome types identified in clinical practice include yin deficiency with fire hyperactivity syndrome, liver stagnation transforming into fire syndrome, and phlegm-dampness internal accumulation syndrome. Since the late 1970s, the investigators' department has taken the lead in treating precocious puberty with TCM diagnostic methods, proposing that the pathogenesis of precocious puberty lies in "kidney yin deficiency and hyperactivity of ministerial fire", and adopting the therapy of nourishing yin and purging fire for its treatment. A number of studies have confirmed that TCM medicines with the effects of nourishing yin and purging fire can effectively alleviate the yin deficiency with fire hyperactivity syndrome in children, delay the development of secondary sexual characteristics and bone age. At present, central precocious puberty is mostly treated with gonadotropin-releasing hormone analogs (GnRHa). However, this treatment has the drawback of inhibiting the growth axis, yielding limited benefits for children with advanced bone age, overweight or obesity, and may even affect glucose and lipid metabolism. Moreover, some children with precocious puberty complicated with obesity may be intolerant to this therapy. In contrast, TCM therapy and integrated TCM-Western medicine therapy can effectively delay the development of secondary sexual characteristics and advanced bone age, and improve final adult height, thus being widely applied in China. Although a large number of relevant studies have been reported in recent years and TCM diagnosis and treatment guidelines for precocious puberty have been formulated, there is still a lack of high-quality evidence-based medical research to support the advantageous aspects of integrated TCM-Western medicine diagnosis and treatment. Additionally, the underlying mechanisms of diagnosis and treatment for different syndrome types of precocious puberty remain insufficiently studied. In this study, the investigators will conduct a multicenter, randomized, double-blind, placebo-controlled clinical trial to evaluate the effects of Sanghe Jianghuo Granules on the regulation of the hypothalamic-pituitary-gonadal (HPG) axis, metabolic homeostasis and inflammatory microenvironment, so as to verify its efficacy and safety. Furthermore, combined with transcriptomics, proteomics and network pharmacology, the investigators will identify the key targets and action pathways of Sanghe Jianghuo Granules, and verify its regulatory effect on the HPG axis through in vivo and in vitro experiments.

This is a three-month randomized controlled trial to investigate the effects of auricular acupressure versus Intermittent carbohydrate restriction on cardiometabolic risk in obese children with gastric heat and dampness obstruction.

This study aims to develop and implement a pediatric palliative care (PPC) program. It is an open-label, randomized trial (2:1 randomization) in pediatric oncology department of Children's Hospital of Fudan University. The intervention group will receive Nurse-initiated Conversations for Early Integration of Palliative Care in Pediatric Oncology (NiCE). The control group will receive routine PPC (will be scheduled to meet with the PPC team only when participants themselves, their families, or the attending oncologist requested an appointment). The intervention will take 6 months.

This is a randomized controlled trial to compare the efficacy and safety of infliximab and immunosuppressives therapy alone or in combination for pediatric Crohn's disease.

The goal of this clinical study is to evaluate the effect of nutritional intervention program based on dietary products in the clinical treatment of newly diagnosed children with type 1 diabetes. The main question aims to answer is: whether high fiber diet can protect beta-cell function in children with newly onset type 1 diabetes. Participants will take 12 weeks of high fiber diet intervention and beta-cell function and gut microbiota structure will be analyzed.

This study evaluates the efficacy of sequential transplantation of umbilical cord blood stem cells and islet cells in children with monogenic immunodeficiency type 1 diabetes mellitus. Umbilical cord blood stem cell transplantation will be performed first. Children with stable immune reconstruction will than receive islet cell transplantation.

The purpose of this study is to establish a standardized nutrition intervention procedure for children with solid tumors, and to explore the effectiveness and clinical applicability of standardized nutrition management and short peptide-based enteral nutrition intervention for improving the nutritional status of children with malignant solid tumors. After admission, patients in the intervention group will receive standardized nutrition management provided by a nutrition support team composed of dietitians, nutritionists, clinicians, and nursing teams. Basic information, including diet, enteral and parenteral nutrition, nutritional status and clinical data, will be collected during the study.

The goal of this clinical trial is to learn if developmental care works when implemented in very low birth weight infants. The main questions it aims to answer are: * Does developmental care shorten the length of hospital stay in the very low birth weight infants? * Does developmental care increase the opportunity of family centered care in the very low birth weight infants? The clinical trial will use a 36-month stepped-wedge cluster-randomised trial conducted across 40 Neonatal intensive care units . A developmental care bundle will be tailored to meet the identified needs of participating NICUs.