Clinical trials

This study aims to establish a standardized, synchronized data collection system for pediatric symptom questionnaires, cough sounds, and breath sounds, and to construct a multimodal database of pediatric respiratory diseases including both disease cases and healthy controls. Using the final research labels determined by clinicians' diagnoses, health status assessments, and research team review as the reference standard, this study will develop and validate a multimodal assisted diagnostic model for common pediatric respiratory diseases based on symptom questionnaires, cough sounds, and breath sounds. The study will primarily evaluate the diagnostic performance of the model in distinguishing healthy children from children with respiratory diseases, screening for asthma and asthma-related cough, and identifying pneumonia, tracheitis/bronchitis, upper airway-related diseases, and common causes of chronic cough. It will also assess the incremental value of cough sounds and breath sounds beyond symptom questionnaire information.

A Phase 3 multi-institutional study for treatment of children with newly diagnosed hepatoblastoma using a modified Paediatric Hepatic International Tumour Trial (PHITT) strategy incorporating a randomized assessment of sodium thiosulfate as auditory protection for children with localized disease, and response adapted therapy for patients with metastatic disease

Asthma is one of the most prevalent chronic respiratory diseases in children, and accurate phenotyping and disease monitoring remain challenging in routine clinical practice. This observational cohort study aims to investigate the clinical value of multimodal tongue and pulse information in the syndrome diagnosis and phenotypic characterization of pediatric asthma. Children aged 5-18 years with a confirmed diagnosis of asthma will be enrolled at Shanghai Children's Medical Center and followed in routine outpatient care. Standardized tongue images and pulse wave data will be collected using validated acquisition devices during visits when lung function testing is performed. Quantitative features extracted from tongue and pulse data will be integrated with clinical information, including asthma stage, lung function parameters, eosinophil counts, allergic sensitization status, and Asthma Control Questionnaire-5 (ACQ-5) scores. The primary objective is to evaluate the associations between tongue-pulse multimodal features and asthma clinical stages and pulmonary function. Secondary objectives include exploring their relationships with airway inflammation and asthma control status. This study seeks to establish a non-invasive, objective, and quantifiable approach to asthma phenotyping, providing evidence for integrating traditional diagnostic features with modern clinical data to support precision management of pediatric asthma.

This study aims to establish a novel personalized closed-loop NiBS/TMS therapeutic strategy and clinical protocol for children with Tic Disorders (TDs) through a series of scientific investigations. Additionally, it seeks to elucidate the underlying neural circuit mechanisms, enhance the therapeutic efficacy of TMS in pediatric TDs, and achieve precision neuromodulation for children with TDs.

Chronic cough is a common and burdensome condition in children, with complex and overlapping etiologies that often lead to delayed diagnosis, misdiagnosis, and inappropriate treatment. This prospective, controlled, observational study aims to develop and clinically validate a Wenjuanxing-based, parent-reported, multidimensional etiologic screening questionnaire for pediatric chronic cough. Children aged 3-18 years presenting with cough lasting ≥2 weeks will be enrolled in a tertiary pediatric respiratory clinic and allocated to either a routine-care group or a questionnaire-assisted group. All caregivers will complete the standardized electronic questionnaire, which generates an automated preliminary etiologic suggestion based on symptom patterns, triggers, and associated features. Diagnostic accuracy, treatment effectiveness, and symptom resolution will be evaluated through structured follow-up at two weeks. The primary outcome is the difference in diagnostic accuracy between physicians using routine assessment alone and those supported by the questionnaire. Secondary outcomes include treatment response and prevention of cough chronicity. This study seeks to provide evidence for a scalable, digital, and standardized screening tool to improve early etiologic identification and clinical decision-making in pediatric chronic cough.

Breakthrough chemotherapy-induced vomiting (CIV) is defined as CIV occurring after adequate antiemetic prophylaxis. Olanzapine is recommended for the treatment of breakthrough CIV in children, without adequate evidence. We conducted an open-label, single-center, phase 3 randomized controlled trial comparing the safety and efficacy of olanzapine and metoclopramide for treating breakthrough CIV.

Primary nocturnal enuresis (PNE), a prevalent pediatric disorder, suffers from therapeutic limitations characterized by low efficacy and high relapse rates. Targeting its core pathophysiology could significantly improve treatment outcomes. Growing evidence implicates circadian dysregulation in PNE pathogenesis. Our preliminary fMRI cohort identified abnormal functional connectivity between the suprachiasmatic nucleus (SCN, the central circadian pacemaker) and superior temporal gyrus in PNE patients, with clinical data confirming circadian realignment correlates with symptom remission. Small-scale pilot studies and clinical observations indicate that modulating central and peripheral circadian rhythms significantly alleviates PNE symptoms. This study will establish a circadian-focused PNE cohort to quantify therapeutic efficacy and elucidate underlying mechanisms, ultimately driving the development of mechanism-based therapies for PNE.

Urine storage and voiding are fundamental physiological processes. In clinical settings, many cases of lower urinary tract dysfunction (LUTD) are closely associated with abnormal conditioned reflexes formed in the central nervous system during the urine storage or voiding phases. Relaxation, as a core physiological and psychological state, has been shown to promote effective urine storage and facilitate smooth voiding. By repeatedly training individuals to establish a new conditioned reflex linking the sensation of urinary urgency with a state of relaxation, it may be possible to improve bladder storage capacity and voiding function. Based on this concept, the investigators have developed the world's first Micturition Desire-Relaxation Training Device (Chinese Patent No.: ZL 2020 1 0397789.4). This study aims to evaluate the clinical efficacy of this device in treating LUTD in children.

The goal of this clinical trial is to learn the plasma concentration of isavuconazole in pediatric patients. It will also learn about the relationship of isavuconazole plasma concentrations to efficacy and safety in pediatric patients. The main questions it aims to answer are: What is the plasma concentration after using isavuconazole in pediatric patients? What is the effective range of plasma concentration of isavuconazole in pediatric patients? What is the safe range of plasma concentration of isavuconazole in pediatric patients? Researchers will measure the plasma concentration of isavuconazole to see whether it is appropriate. Participants will: Take drug isavuconazole as prescribed by the doctor; 1mL of blood is drawn 30min before the next dose.

From January 2010 to December 2014, 150 children with MS-LCH were treated in our hospital following a LCH II (Arm B) based protocol. Treatment was based on a modification of the LCH-II (Arm B) based protocol. However, the continuation treatment was extended to 56 weeks and etoposide was omitted from the continuation treatment. For the 59 patients with RO involvement (RO+) (the lungs are not considered a RO in the current study), the rapid response rate (week 6) was 61.0% and the 3-year overall survival (OS) 73.4±5.9%. Rapid responders had a better 3-year survival rate than poor responders (90.9±5.0% vs. 45.7±11.0%, P\<0.001). The 3-year OS in the current study is 10\~20% lower than the rates reported by Gadner et al. and Morimoto et al.. We have not yet adopted effective salvage therapies for RO+ patients with recurrent disease. During the time of this study, cladribine was unavailable. Second-line therapy for non-responders or patients with disease reactivation was individualized treatment based on the physician's experience. An effective salvage therapy is essential for this high-risk group. For 91without RO involvement (RO-), 78 patients (85.7%) were rapid responders at week 6. The 3-year cumulative reactivation rate was 10.7% for RO- patients. No death occurred in this subgroup, with a 3-year OS of 100% in RO- patients. Compared to the LCH II and LCH III trials, the current study had a more intensive initial treatment regimen for RO- patients. However, the addition of etoposide to prednisone and vincristine in the initial therapy did not increase the 6-week response rate for RO- patients (85.7% in this study compared to 83% in the LCH II study and 86% in the LCH III study). Surprisingly, with a relatively intense initial treatment, a relatively low 3-year cumulative reactivation rate was observed in RO- patients in the current study. This result suggests that the initial treatment intensity and duration of continuation therapy both impact disease reactivation. The intensity of induction can affect the degree of disease resolution. Insufficient treatment intensity might lead to late relapse. Similarity to that observed has been in other childhood hematological malignancies. This finding deserves to be tested in prospective clinical trials with long-term follow-up. Cytarabine has been applied for patients with LCH but has never been evaluated in our hospital prospectively. In this study, we administer a cytarabine contained protocol to patients with multisystem involvement with or without risk organs involvement. The treatment results will be compared with our historical studies.

Establish a clinical diagnosis and treatment and long-term follow-up database of preterm infants, and analyze the effects of prenatal factors (including genetic characteristics, maternal diseases, etc.), postnatal diagnosis and treatment measures and family maintenance environment after discharge on preterm infant mortality and major diseases in the near and long term.