Efficacy Evaluation of UCB-MNCs in the Treatment of Refractory Neonatal Diseases
- Conditions
- Bronchopulmonary DysplasiaShort Bowel SyndromeHypoxic-Ischemic Encephalopathy
- Interventions
- Device: Breathing support techniqueBiological: Mononuclear cellsProcedure: Total parenteral nutritionDevice: Mild hypothermia therapy
- Registration Number
- NCT06427642
- Lead Sponsor
- Shandong Qilu Stem Cells Engineering Co., Ltd.
- Brief Summary
Hypoxic-ischemic encephalopathy (HIE), bronchopulmonary dysplasia (BPD), short bowel syndrome (SBS) are refractory in clinical treatment. Thus, how to better prevent such diseases is currently a key research topic in the international field. The use of cord blood-derived mononuclear cells may promote to save lives and improve patient outcomes.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 120
- For children with hypoxic-ischemic encephalopathy (HIE): meet the diagnostic criteria for HIE.
For children with bronchopulmonary dysplasia (BPD): 1) preterm infants with definite gestational age of 25-30 weeks; 2) birth weight 401-1249 g; 3) the risk of BPD was assessed to be greater than 60%. The scoring was based on the BPD high risk scoring system established by the NCHD Neonatal Cooperative Network; 4)parents read the subject's instructions, agreed to the treatment and signed the informed consent.
For children with short bowel syndrome (SBS): 1) postoperative short bowel syndrome caused by neonatal necrotizing enterocolitis and other causes (developmental malformations of the digestive tract: intestinal atresia, anal atresia, intestinal stenosis, etc.); 2) parents read the subject's instructions, agreed to the treatment and signed the informed consent.
- For children with HIE: unable or unwilling to provide informed consent or unable to comply with trial requirements.
For children with BPD: 1) with severe anemia, severe intracranial hemorrhage, pulmonary hemorrhage, congenital respiratory malformations (posterior nostril atresia, tracheoesophageal fistula, cleft palate, etc.), complicated congenital heart disease, diaphragmatic hernia, shock, other serious comorbidities or complications (congenital inherited metabolic diseases, endocrine diseases, severe congenital malformations and other diseases that affect lung development); 2) unable or unwilling to provide informed consent or unable to comply with trial requirements.
For children with SBS: unable or unwilling to provide informed consent or unable to comply with trial requirements.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Experimental group for children with SBS Total parenteral nutrition Intravenous infusion of UCB-MNCs Experimental group for children with HIE Mild hypothermia therapy Intravenous infusion of UCB-MNCs is given within 24 hours of being identified as a high-risk patient Control group for children with BPD Breathing support technique Clinical routine treatment Control group for children with HIE Mild hypothermia therapy Mild hypothermia therapy is given for 72 hours to maintain anal temperature between 33.5°C and 34°C Experimental group for children with BPD Mononuclear cells Intravenous infusion of UCB-MNCs is given within 24 hours of being identified as a high-risk patient Experimental group for children with SBS Mononuclear cells Intravenous infusion of UCB-MNCs Experimental group for children with BPD Breathing support technique Intravenous infusion of UCB-MNCs is given within 24 hours of being identified as a high-risk patient Experimental group for children with HIE Mononuclear cells Intravenous infusion of UCB-MNCs is given within 24 hours of being identified as a high-risk patient Control group for children with SBS Total parenteral nutrition Clinical routine treatment
- Primary Outcome Measures
Name Time Method Incidence of adverse reactions Within 12 hours after UCB-MNCs infusion Monitor oxygen, heart rate, temperature, rash, infection, etc
- Secondary Outcome Measures
Name Time Method Imaging test results 2 weeks and 6 months after UCB-MNCs infusion Children with HIE: Brain diffusion tensor imaging (DTI) and 18F-Fluorodeoxyglucose positron emission tomography (18F-FDG-PET/CT)
Biomarker of HIE 7 days after UCB-MNCs infusion pNF-H, marker of central nervous system axonal damage
Change of Gross Motor Function Measure (GMFM) 1, 3, 6 months after UCB-MNCs infusion GMFM is a standardized measurement tool for assessing motor function for children with HIE. It consists of lying \& rolling, sitting, crawling \& kneeling, standing, etc. Higher value means better gross motor function
Biomarker of BPD 7 days after UCB-MNCs infusion AGER, marker of lung epithelial cell damage
Incidence of complications a year Children with BPD: The incidence of various complications such as pneumothorax, necrotizing enterocolitis (NEC), intraventricular hemorrhage (grade 3 and above), persistent pulmonary hypertension (PPHN), retinopathy of prematurity (ROP)
Electroencephalography (EEG) results 7 days UCB-MNCs infusion Children with HIE: The frequency of seizures will be measured via EEG. Seizures appear on EEG as a sudden and transient rise in the lower and/or upper borders of amplitude
Ventilator supporting time 1 month after UCB-MNCs infusion Ventilator supporting time and oxygen demand will be recorded as important indications for clinical prognosis for children with HIE or BPD
Change of Gross Motor Performance Measure (GMPM) 1, 3, 6 months after UCB-MNCs infusion GMPM is a standardized measurement tool for assessing quality of movement for children with HIE. Higher value means better motor quality
Inflammatory indicators concentrations 7 days after UCB-MNCs infusion Serum IL-6, IL-8, TNF concentrations will be measured via ELISA for children with HIE, BPD or SBS
Trial Locations
- Locations (1)
Qilu Children's Hospital of Shandong University
🇨🇳Jinan, Shandong, China