Clinical trials

Brief Summary This study is a multicenter, randomized, double-blind, placebo-controlled trial designed to evaluate whether an injectable form of Coenzyme I (also known as nicotinamide adenine dinucleotide, or NAD⁺) is safe and effective for treating children with cardiac dysfunction (weak heart pumping). Who can join? We plan to enroll 180 children under 18 years of age who are hospitalized with cardiac dysfunction, meaning their heart's pumping ability (left ventricular ejection fraction, or LVEF) is below 55%. Children will be recruited from five hospitals in China. Children with certain serious conditions, such as end-stage heart failure, severe liver or kidney problems, known allergy to the study drug, or active participation in another drug trial, will not be eligible. What will participants receive? All children will receive standard heart failure care as recommended by current guidelines. In addition, they will be randomly assigned (like drawing lots) to one of three groups: Placebo group - receives standard care plus a saltwater (normal saline) infusion that looks like the study drug but contains no active medicine (60 children). Low-dose group - receives standard care plus Coenzyme I injection at 2 mg per kg of body weight per day (maximum 100 mg) (60 children). High-dose group - receives standard care plus Coenzyme I injection at 4 mg per kg of body weight per day (maximum 200 mg) (60 children). The study drug or placebo is given as a slow intravenous (IV) infusion once daily for 7 days. Each infusion takes at least 2 hours. What is the main goal? The main question is whether adding Coenzyme I improves heart function more than standard care alone. The primary endpoint is the change in LVEF from baseline to 4 weeks after treatment, measured by echocardiography. We will also assess NT-proBNP, a blood test that reflects the strain on the heart, at several time points. What else will we look at? We will also evaluate quality of life (using a child-friendly questionnaire), heart function grade (NYHA/ROSS), exercise capacity (6-minute walk test), growth and development, cardiac structure and remodeling (by echocardiography), global longitudinal strain (GLS), and clinical events such as heart failure-related rehospitalization, emergency interventions, and death. We will carefully monitor for any side effects or safety concerns throughout the study. Why is this important? Children with cardiac dysfunction often do not respond well enough to current treatments, and there are few specific therapies designed for them. Coenzyme I (NAD⁺) is a natural substance in the body that helps heart cells produce energy. Studies in adults and in laboratory models suggest it may protect the heart and improve its function. This trial is the first large-scale pediatric study to test whether it can help children with heart pumping problems. How long will participation last? Each child will be in the study for about 48 weeks - 7 days of treatment in the hospital, followed by follow-up visits at 4 weeks, 12 weeks, and 24 weeks after treatment, and a final telephone or clinic visit at 48 weeks to collect clinical event data. Safety and oversight An independent Data and Safety Monitoring Board (DSMB) will regularly review all safety information. The study has been approved by the ethics committees of all participating hospitals and is conducted in accordance with the Declaration of Helsinki and Chinese Good Clinical Practice guidelines. All enrolled children are covered by clinical trial liability insurance.

This study investigates the impact of letermovir prophylaxis on viral infections (including CMV, EBV, BKV, HHV-6/7, RSV, ADV, HSV, etc.) following allogeneic hematopoietic stem cell transplantation in pediatric patients with EBV-associated T/NK-cell lymphoproliferative diseases and refractory/relapsed EBV-related hemophagocytic lymphohistiocytosis. Additionally, we examine its effects on other transplantation complications, including engraftment failure, graft-versus-host disease (GvHD), disease relapse, thrombotic microangiopathy (TMA), overall survival (OS), post-transplant lymphoproliferative disorder (PTLD) incidence, and immune reconstitution.

Background: Currently in China, adolescent asthma care is characterized by both undertreatment and overtreatment. Patients in this age group may exhibit negative emotions toward disease treatment and long-term control. Insufficient disease knowledge, irregular lifestyle habits, and negative attitudes toward the disease can all reduce medication adherence, affect disease control outcomes, and thereby increase the risk of severe chronic respiratory conditions and disease burden in adulthood. Objectives: To assess disease control status in adolescent asthma patients, evaluate the psychological well-being of patients and their family members, and understand patients' self-management skills and readiness for transition to adulthood. Procedures: Eligible participants will complete a series of assessment questionnaires through the Youran Huxi mobile application, including questionnaires on asthma control, physical activity, psychological health, self-management behaviors, and transition readiness. The study period is 12 months; the baseline assessment involves one-time completion of written questionnaires with no interim follow-up visits. The study does not intervene in clinical diagnosis or treatment decisions.

The aim of this study is to comprehensively understand the disease control and management status of children with asthma in China, as well as the types of airway inflammation. The findings of this survey will help improve the asthma control level in Chinese children, facilitate future patient education, and guide rational medication use. Moreover, it will provide important evidence for health decision-making departments to better allocate and utilize medical resources.

The goal of this clinical trial is to learn if hetrombopag promotes platelet engraftment in neuroblastoma children undergoing autologous hematopoietic stem cell transplantation. The main question it aims to answer is: · Does drug hetrombopag promote platelet engraftment? Participants will take hetrombopag every day for 6 weeks or until platelet \> 100\*10\^9/L.

Children are sensitive to ionizing radiation, and there are significant regional variations in current pediatric CT radiation doses. Although there are studies on low-dose CT targeting single lesions, these studies require clear clinical indications and support from advanced equipment, making widespread promotion difficult. The establishment of low-dose CT protocols is mostly experience-based; when radiologists lack confidence in image quality, the credibility of diagnostic reports is compromised. Therefore, the development of low-dose protocols must meet the diagnostic confidence of most radiologists, enable clear visualization of key anatomical structures in clinical practice, and satisfy the requirements for disease diagnosis. The purpose of this study is to establish a pediatric low-dose CT scanning protocol using Low-dose Simulation of CT (LDS) technology, and conduct a multicenter randomized controlled trial (RCT) in clinical settings to verify the clinical feasibility of this low-dose CT scanning protocol.

In order to further observe the long-term safety and effectiveness of real-world polyethylene glycol-recombinant human growth hormone(PEG-rhGH) treatment of GHD, idiopathic short stature, and SGA in children, explore and analyze the factors affecting the efficacy of PEG-rhGH and the height prediction model after treatment, etc., collect and analyze more scientifically and rationally, and understand the situation of real-world PEG-GH treatment. A database registration study was developed.

To investigate whether the addition of recombinant humanized anti-CD25 monoclonal antibody to the conventional EBV-T/NK LPD conditioning regimen can prevent acute and chronic GVHD after transplantation, improve the severity of GVHD and have a corresponding impact on other related post-transplant complications such as poor engraftment, thrombotic microvascular disease, early EBV reactivation and relapse.

This study is based on the hypothesis that the pharmacokinetics of anti-tuberculosis drugs in TB children are different from adults. The investigators aim to study the population pharmacokinetics of children receiving the anti-tuberculsis drugs for treatment of TB. In this study, the investigators will detect drug concentration in plasma by using residual blood samples of blood gas analysis and other clinical tests and employ computers for constructing population pharmacokinetic models. In addition, the investigators also want to correlate use of anti-tuberculsis drugs with treatment effectiveness and incidence of adverse effects in children. This novel knowledge will allow better and more rational approaches to the treatment of TB in children. It will also set the foundation for further studies to improve anti-tuberculosis drug therapies for children.

This study is a multicenter, prospective cohort study of patients diagnosed with cystic fibrosis, the clinical information of recruited patients, including clinical manifestations, lung function, chest imaging, quality of life and other indicators, will be followed for 10 years.

This study is a multicenter, prospective cohort study of patients diagnosed with primary ciliary dyskinesia, the clinical information of recruited patients, including clinical manifestations, lung function, chest imaging, quality of life and other indicators, will be followed for 10 years.