Clinical trials

The study aims to conduct a real-world investigation of children with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) in China, assessing their diagnosis, treatment status, survival outcomes, and prognostic factors. The research will involve children diagnosed with Ph+ ALL between January 1, 2015, and December 31, 2024. Our objective is to understand the incidence and prognosis of CML-like ALL, compare the diagnostic and treatment differences between CML-like ALL and typical Ph+ ALL, and provide clinical evidence for standardized treatment of CML-like patients. Through a multicenter retrospective study, we hope to improve the diagnostic and treatment strategies for children with Ph+ ALL, enhancing patient survival rates and quality of life.

This is a single-arm, open-label, phase I clinical study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of RD06-05, a universal CD19/BCMA dual-targeting chimeric antigen receptor T-cell (CAR-T), in pediatric and adolescent patients with refractory autoimmune diseases, including systemic lupus erythematosus/lupus nephritis (SLE/LN), systemic sclerosis (SSc), idiopathic inflammatory myopathy (IIM), multidrug-resistant nephrotic syndrome (MDR-NS), and refractory IgA nephropathy (IgAN). Approximately 30 eligible patients will be enrolled and receive a single intravenous infusion of RD06-05 at an initial dose of 6×10⁶ CAR+ T cells/kg, with a potential dose escalation to 10×10⁶ CAR+ T cells/kg following review by a Safety Review Committee (SRC).

Advances in medical care have significantly improved survival among children with cancer. In China, the 5-year survival rate has reached 71.9%. Despite these improvements, many survivors continue to experience multiple co-occurring symptoms, such as fatigue, pain, sleep disturbance, and depression, which may adversely affect their quality of life. These symptoms often occur together as symptom clusters and may reflect shared underlying biological mechanisms. This study aims to characterize symptom clusters among childhood cancer survivors and to explore their potential biological basis. Participants will complete questionnaire assessments at multiple time points to evaluate symptom patterns and changes over time. In addition, stool samples will be collected to analyze gut microbiota composition and metabolite profiles. The study will examine the associations between symptom clusters and gut microbiota-metabolite features. Findings from this study are expected to improve understanding of symptom burden in childhood cancer survivors and to provide evidence for the development of targeted symptom management strategies.

This study was a cross-sectional, observational design without involving random grouping or intervention. From July 2026 to June 2027, 199 children with type 1 diabetes who had received insulin treatment for at least 6 months and their main family caregivers were recruited by a continuous enrollment method from the endocrinology department of a tertiary children's hospital in Zhejiang Province. Qualified investigators conducted on-site distribution and collection of structured questionnaires, collecting general information of the children, the occurrence of skin complications, and the insulin injection behaviors of the caregivers. At the same time, personnel with ultrasound qualifications used a wireless handheld ultrasound combined with a skin lipid caliper to uniformly measure the subcutaneous fat thickness at 8 sites including the abdomen, buttocks, arms, and thighs.

The goal of this clinical trial is to evaluate the efficacy and safety of Ruxolitinib Phosphate Cream in treating non-segmental vitiligo in children aged 2 to 11 years under real-world conditions. The main question\[s\] it aims to answer are: Primary Efficacy: What is the improvement rate (e.g., proportion of participants achieving F-VASI75) after 24 weeks of treatment with Ruxolitinib Phosphate Cream in this population? Safety Profile: What is the safety profile of the treatment over 24 weeks, specifically regarding the incidence of Application Site Acne, Application Site Pruritus, and other adverse events? Participants will: Apply Ruxolitinib Phosphate Cream topically to vitiligo lesions as prescribed by the investigator. Attend scheduled clinic visits at Weeks 4, 8, 12, and 24 for efficacy and safety assessments. Undergo standardized clinical photography and severity scoring of their vitiligo lesions at each visit. Report any adverse events or skin reactions experienced during the study period to the research team.

The goal of this clinical trial is to learn whether a Nintendo Switch-based exergame physical activity program can help children and adolescents with congenital heart disease increase their physical activity. It will also learn about the safety, acceptability, and feasibility of this program. The study will include children and adolescents aged 8 to 18 years who have congenital heart disease, have received surgical or interventional treatment, and are clinically stable. The main questions it aims to answer are: Does the exergame-based program increase the amount of time participants spend in moderate-to-vigorous physical activity each day? Is the exergame-based program safe, acceptable, and feasible for children and adolescents with congenital heart disease? All participants will receive the same exergame-based physical activity program. Researchers will use different baseline observation periods to help understand whether changes in physical activity happen after the program starts. Participants will: Wear an activity monitor to measure daily physical activity. Complete a baseline observation period lasting 7, 14, or 21 days. Take part in a 12-week Nintendo Switch-based exergame physical activity program with guidance, goal setting, self-monitoring, feedback, and caregiver support. Complete a 2-week observation period after the program to see whether physical activity changes are maintained. Complete study assessments at screening, the end of baseline, week 4, week 8, week 12, and the end of the observation period. Researchers will also collect information on step counts, energy expenditure, self-reported physical activity, exercise capacity, quality of life, adherence, acceptability, and adverse events.

This is an exploratory, open-label, single-arm clinical study designed to evaluate the safety, tolerability, and preliminary efficacy of QT-219CX. QT-219CX is a universal allogeneic chimeric antigen receptor T-cell (CAR-T) product targeting both CD19 and BCMA. The study targets subjects with refractory B-cell-related autoimmune diseases, including systemic lupus erythematosus (SLE), multi-drug resistant nephrotic syndrome (NS), IgA nephropathy (IgAN), systemic sclerosis (SSc), and ANCA-associated vasculitis (AAV) .The research is divided into two phases: a dose-escalation phase and a dose-expansion phase. Dose Escalation: Utilizes a standard "3+3" design to evaluate potential recommended dose(RD) and identify dose-limiting toxicities (DLTs) .Treatment Procedure: Eligible subjects will receive a lymphodepleting conditioning regimen followed by a single intravenous infusion of QT-219CX .Primary Objectives: The primary goals are to evaluate the safety profile, including the incidence of cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS), and to assess clinical response rates at 90 days post-infusion .Follow-up: Subjects will be monitored for pharmacokinetics (cell expansion), pharmacodynamics (B-cell depletion), and long-term safety for up to two years .

The goal of this clinical trial is to evaluate whether a Just-in-Time Adaptive Intervention (JITAI)-based digital exercise rehabilitation program can improve postoperative recovery in children after congenital heart disease (CHD) surgery. The study will examine whether this system can: Improve cardiopulmonary endurance, cardiac function, and muscle strength Increase rehabilitation adherence and enhance training experience while reducing exercise-related fear Researchers will compare children receiving the adaptive digital exercise rehabilitation program with those receiving standard postoperative rehabilitation to determine whether the digital program provides additional clinical and behavioral benefits. Participants will: Wear non-invasive monitoring devices to collect real-time physiological data Engage in daily structured exercise rehabilitation sessions guided by the digital system Receive adaptive adjustments in exercise intensity based on real-time physiological feedback Complete questionnaires on recovery, emotional status, and rehabilitation experience

Based on the biopsychosocial (BPS) medical model, this study focuses on HPV vaccination behavior among adolescent females. It aims to explore the influence of multidimensional factors-including biological characteristics, psychological factors, and family and social environments-on vaccination behavior, and to evaluate the effectiveness of a comprehensive HPV vaccination intervention program designed for joint participation by adolescents and their parents. This study employs a prospective, multicenter, randomized, open-label, parallel-group design. Participants-girls and adolescents aged 9-17 who are scheduled to receive or have not yet completed HPV vaccination, along with their primary caregivers-were recruited from our hospital and collaborating pediatric/maternal and child health institutions both domestically and internationally. Participants were randomly assigned in a 1:1 ratio to an intervention group and a control group. In addition to routine vaccination clinic counseling, the intervention group received a comprehensive HPV vaccination intervention program based on the BPS model, including: structured health education materials (illustrated booklets/short videos); structured communication and shared decision-making support in the clinic setting; continuous information dissemination and vaccination reminders via platforms such as WeChat; and personalized follow-up and Q\&A sessions for families with high vaccine hesitancy; The control group received standard routine education and vaccination services. The primary outcome was the proportion of adolescents who completed the first dose of the HPV vaccine within 3 months of enrollment; secondary outcomes included the proportion completing the full vaccination series within 6 months, changes in vaccine hesitancy levels and HPV-related knowledge, changes in anxiety/depression levels among adolescents and caregivers, and changes in the quality of parent-child communication regarding health and vaccination as well as family decision-making patterns. This study is expected to identify key bio-psycho-social determinants of HPV vaccination behavior among adolescent females, validate the effectiveness of the comprehensive BPS intervention in increasing vaccination rates and improving decision-making experiences and psychosocial outcomes, and provide evidence-based guidance and scalable practical pathways for pediatric and related specialty clinics to implement adolescent vaccination health promotion and family shared decision-making services.

Study Purpose: To develop a pulmonary rehabilitation intervention program suitable for school-age children with asthma, and to evaluate its effectiveness in improving asthma control in children through a prospective randomized controlled trial. Study Methods: This is a single-center, prospective, randomized, open-label, parallel-controlled trial. Eligible subjects who meet all inclusion criteria and none of the exclusion criteria, together with their families, will be randomly assigned in a 1:1 ratio to either the intervention group (family-empowered pulmonary rehabilitation intervention) or the control group (conventional pulmonary rehabilitation care) after signing the informed consent form. Both groups will receive a 12-week core intervention period, with follow-up assessments conducted at baseline, end of intervention (12 weeks), and at 24 and 48 weeks after intervention.

This is an exploratory, open-label, single-arm Phase 1 clinical study designed to evaluate the safety, tolerability, and preliminary efficacy of QT-219C. QT-219C is a universal allogeneic chimeric antigen receptor T-cell (CAR-T) product targeting both CD19 and BCMA. The study targets subjects with refractory B-cell-related autoimmune diseases, including systemic lupus erythematosus (SLE), multi-drug resistant nephrotic syndrome (NS), IgA nephropathy (IgAN), systemic sclerosis (SSc), and ANCA-associated vasculitis (AAV) .The research is divided into two phases: a dose-escalation phase and a dose-expansion phase. Dose Escalation: Utilizes a standard "3+3" design to evaluate potential recommended dose(RD) and identify dose-limiting toxicities (DLTs) .Treatment Procedure: Eligible subjects will receive a lymphodepleting conditioning regimen followed by a single intravenous infusion of QT-219C .Primary Objectives: The primary goals are to evaluate the safety profile, including the incidence of cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS), and to assess clinical response rates at 90 days post-infusion .Follow-up: Subjects will be monitored for pharmacokinetics (cell expansion), pharmacodynamics (B-cell depletion), and long-term safety for up to two years .

Cardiopulmonary bypass-associated pulmonary injury is a common complication after infant cardiac surgery and may contribute to impaired oxygenation, prolonged mechanical ventilation, and longer intensive care stay. Lidocaine has anti-inflammatory and membrane-stabilizing properties and may attenuate perioperative lung injury. This investigator-initiated, randomized, placebo-controlled, double-blind trial will evaluate whether perioperative intravenous lidocaine reduces postoperative pulmonary injury in infants undergoing corrective non-palliative congenital cardiac surgery with cardiopulmonary bypass.

Primary monosymptomatic nocturnal enuresis (PMNE) is one of the most common urinary system problems in childhood. The exact pathological mechanism has not been fully elucidated yet, but excessive nocturnal urination is considered one of the core pathogenic mechanisms. Studies have found that a considerable number of PMNE patients have abnormal secretion of arginine vasopressin (AVP) at night. Currently, the "gold standard" for diagnosing nocturnal polyuria is through a voiding diary, which is cumbersome and the records may contain errors, and there are many inconveniences in clinical implementation. Therefore, finding objective and simple biomarkers to assist in diagnosis and classification is a current clinical research hotspot. Copeptin is the C-terminal fragment of the precursor protein of AVP and is released into the blood simultaneously at the same molar ratio as AVP, making it an ideal alternative biomarker for AVP. This study aims to systematically and deeply explore the diagnostic value of serum copeptin in PMNE, especially in its different subtypes (NP-PMNE vs. NNP-PMNE), analyze its correlation with clinical severity, and explore more precise detection strategies, in order to provide new and objective biological tools for the clinical management of PMNE.

A single arm, open-label pilot study is designed to determine the safety and effectiveness of anti-CD19/BCMA CAR-NK cell injection in patients with refractory pediatric rheumatic diseases.

This study aims to construct a structuralized follow-up model for survivors of childhood hematological malignancies in China. Using a multicenter prospective cohort design, it will identify the burden and risk factors of late effects. The study hypothesizes that a standardized follow-up path managed by an electronic platform will improve follow-up compliance and reduce the missed diagnosis rate of late effects.

This is a prospective, single-center clinical study to evaluate the safety and efficacy of a personalized 6-mercaptopurine (6-MP) dosing strategy guided by NUDT15 and TPMT genotypes in children with Acute Lymphoblastic Leukemia (ALL) during maintenance therapy. The study compares this gene-guided strategy with historical controls to assess if it reduces the incidence of Grade ≥3 neutropenia and infection events.

This study is a Study to Understand How to Better Match Allergy Shots to a Child's Unique Mite Allergy Profile 1. Why is this study being done? For many children, tiny house dust mites are a major cause of persistent allergies, leading to uncomfortable symptoms like a stuffy nose, sneezing, and even asthma. A common long-term treatment is allergy shots, also known as allergen immunotherapy (AIT). These shots work like a vaccine, slowly training the body's immune system to stop overreacting to mites. However, not every child responds to this treatment in the same way. The investigators now know that children can be allergic to different parts of the dust mite. While most treatments focus on the most common parts (called "major allergens"), some children are allergic to other, less common parts (called "intermediate or minor allergens"). The EXPLORER study wants to find out if a child's unique allergy profile-meaning which specific mite proteins they are allergic to-affects how well they respond to allergy shots. Our goal is to move away from a "one-size-fits-all" approach and toward more personalized care for children with dust mite allergies. 2. Who is this study for? The investigators are looking for children and teenagers (ages 5 to 18) in China who have a confirmed dust mite allergy and are planning to start allergy shots. 3. What will happen during the study? This is an "observational" study, which means the investigators will simply observe and track the progress of children who are already receiving a standard, approved allergy shot treatment as part of their regular medical care. The investigators won't be testing a new drug. Here's what participation involves: A Detailed Allergy Test: At the beginning, participants will have a special blood test. This test will look for allergies to nine different specific parts of the dust mite, giving us a very detailed map of their allergy. Grouping: Based on the results of this test, participants will be placed into different groups according to their specific allergy patterns. Tracking Progress: The investigators will follow their treatment journey for three years. The investigators will collect information at regular check-ups (at 0, 3, 6, 12, 24, and 36 months) on: How well their symptoms are controlled. How much allergy medication they need. Their overall quality of life. Any side effects from the shots. Annual Blood Tests: Once a year, the investigators will repeat the detailed blood test to see how their immune system is changing in response to the treatment. 4. What do the investigators hope to learn? The investigators have a main theory, or hypothesis: Children who are allergic to many different parts of the dust mite (including the minor ones) will show greater improvement from the allergy shots, compared to children who are only allergic to the most common parts. By studying a large group of 1,000 children, the investigators hope to provide clear evidence that can help doctors choose the right treatment for the right child. This will help fill a current gap in medical guidelines and bring us closer to truly personalized treatment for pediatric allergies. 5. Why is this study important? This is the first large-scale study of its kind in a real-world setting. The results could change how doctors diagnose and treat dust mite allergies in children. Instead of just knowing a child is allergic to mites, the investigators will be able to see the full picture of how they are allergic. This knowledge will help ensure that every child receives the treatment that gives them the best chance for long-term relief.

Under conventional treatment regimens, pediatric ocular myasthenia gravis (OMG) is prone to relapse and is associated with corticosteroid-related adverse effects, indicating an unmet clinical need. In May 2025, the targeted B-cell biologic agent Telitacicept was approved for use in adult patients with acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (GMG) and subsequently initiated in national multicenter clinical trials for adult OMG. Our center published a retrospective study in the Chinese Journal of Evidence-Based Pediatrics in August 2025, which was the first report both domestically and internationally on the efficacy and safety of Telitacicept in four pediatric OMG patients. This study plans to conduct a prospective, multicenter, open-label, single-arm clinical trial aimed at evaluating the effectiveness and safety of Telitacicept in pediatric OMG.

Objective: To explore the safety and tolerability of different doses of EXG110 with Fabre disease

This study aims to evaluate the efficacy and safety of inhaled xenon for the treatment of children with autism spectrum disorder (ASD). The primary objective is to determine whether short-term inhalational xenon therapy can improve social functioning in children with ASD, as measured by changes in the Social Responsiveness Scale (SRS). Safety and tolerability of xenon inhalation in the pediatric population will also be assessed. In this randomized, placebo-controlled trial, participants will receive either inhaled xenon or a placebo gas (medical air without xenon) to compare treatment effects. Participants will: Inhale 25% xenon or placebo for 10 minutes per day for 10 consecutive days Attend two clinical visits: one immediately after completion of the intervention and one at 3 months post-intervention for follow-up assessments and safety evaluations

1. Main objectives: ① To determine the optimal timing for bronchoscopic lung lavage in treating severe pneumonia in children, providing evidence-based guidance for the standardized clinical implementation of this technique. ② To establish a multidimensional efficacy evaluation system encompassing clinical symptoms, blood gas analysis, and inflammatory markers. 2. Secondary objectives: ① To assess the impact of different intervention timings on clinical outcomes, including the time to temperature normalization, resolution of lung rales, length of hospital stay, hospitalization costs, and mortality rate. ② To evaluate the therapeutic effects at the pathophysiological level by measuring inflammatory factors (e.g., IL-6, IL-8), epithelial/endothelial injury biomarkers (e.g., SP-D, angiopoietin-2), and indicators of pathogen clearance, thereby clarifying the treatment's effects on inflammation control, lung injury repair, and pathogen elimination. ③ To enhance the accumulation of safety data by calculating the incidence of adverse events related to bronchoscopic procedures (such as airway bleeding and hypoxemia) and further defining the safety profile of this technique in the pediatric population.

The goal of this clinical trial is to evaluate whether supplementing with pasteurized donor human milk (pHDM) or preterm formula (PTF) when own mother's milk (OMM) is insufficient can improve outcomes in very preterm infants born before 29 weeks of gestation. It also aims to assess whether routine use of human milk fortifiers benefits this population. The main questions it aims to answer are: Does supplementing OMM with pHDM or PTF improve survival without surgery-requiring necrotizing enterocolitis (NEC) by 34 weeks corrected gestational age? Is routine fortification of human milk better than selective fortification based on growth faltering? Researchers will compare: pHDM vs. PTF to see which better supports survival without severe NEC. Routine fortification vs. selective fortification to assess the impact on growth and long-term neurodevelopment. Participants will: Be randomized twice: * First, within the first week of life to receive either pHDM or PTF when OMM is insufficient * Second, in the second week of life to receive either routine fortification or selective fortification only if growth faltering occurs Receive feeding and care as per standard clinical practice Complete neurodevelopmental assessment at 2 years corrected age using the PARCA-R tool (no additional study visits required) This multicenter, double-randomized, open-label randomized controlled trial is embedded in routine neonatal care and uses real-world data to assess both short- and long-term outcomes. COLLABORATE-China is being run in partnership with the UK-wide COLLABORATE trial sponsored by Imperial College London.

The goal of this clinical trial is to investigate whether there are significant differences in the efficacy of infliximab monotherapy and combined azathioprine therapy in treating pediatric Crohn's disease, as well as whether there are differences in the safety of these two treatment regimens during long-term use. The main questions it aims to answer are: Is infliximab combined with azathioprine superior to monotherapy in inducing and maintaining remission of Crohn's disease in children? In long-term treatment, can infliximab combined with azathioprine more effectively reduce disease activity and the occurrence of complications, but the incidence of adverse reactions is not higher than that of monotherapy? Researchers will compare the treatment of infliximab combined with azioprine with that of infliximab monotherapy to assess the efficacy and safety of both in inducing and maintaining remission of Crohn's disease in children. Participants will: Take drug ABC or a placebo every day for 4 months Experimental group: Infliximab, administered intravenously at 5mg/kg every 8 weeks at weeks 0, 2, 6 and thereafter, along with azathioprine, taken orally at 1.5-2.5mg/kg daily. Control group: Infliximab (Remicade), administered intravenously at 5mg/kg every 8 weeks at weeks 0, 2, 6 and thereafter. A comprehensive disease assessment will be conducted at the hospital in the 14th and 54th weeks. Record their symptoms, signs and test results.

This study aims to address the high prevalence and recurrence rate of severe early childhood caries (S-ECC) by breaking away from the traditional single biological factor perspective and introducing the theories of "24-hour activity behavior" and "family psychological stress". By collecting clinical and behavioral data from 1,200 preschool children and their parents, it explores the association pathways between parental burnout, children's executive function, sleep/dietary behaviors and S-ECC, and builds a high-precision risk prediction model to provide evidence-based support for the clinical development of personalized prevention strategies.

The correction of ventral curvature in hypospadias follows a stepwise principle. Clinically, in some cases of hypospadias, residual severe ventral curvature (VC ≥ 30°) persists even after thorough skin degloving and transection of the urethral plate, due to the unbalanced development of the ventral and dorsal tunica albuginea of the corpus cavernosum. In such cases, ventral tunica albuginea incision and corporoplasty with a graft are mandatory. Although the currently commonly used pedicled Tunica Vaginalis Flap (TVF) corporoplasty can effectively correct the curvature, it requires additional dissection of the scrotum and tunica vaginalis sac. This prolongs the operative time and poses risks of donor-site complications, such as testicular retraction and scrotal hematoma. The novel Urethral Plate Flap (UPF) corporoplasty utilizes local pedicled urethral plate tissue for homologous repair. This study adopts a prospective, single-center, randomized, controlled, double-blind, non-inferiority trial design, enrolling 90 subjects. The aim is to verify that the therapeutic efficacy of the UPF technique in correcting such residual severe ventral curvature is non-inferior to that of TVF, while demonstrating significant advantages in surgical efficiency and donor-site safety. This study aims, through a single-center, double-blind, RCT design, and under the strict indication of "residual severe ventral curvature after urethral plate transection," to verify efficacy via a "non-inferiority" hypothesis, and to verify safety and efficiency via a "superiority" hypothesis. The goal is to provide Level I evidence for the update of hypospadias guidelines, while simultaneously exploring the establishment of postoperative imaging evaluation standards.