Efficacy Evaluation of UCB-MNCs in the Treatment of Refractory Neonatal Diseases

Shandong Qilu Stem Cells Engineering Co., Ltd.120 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

120

Conditions

Hypoxic-Ischemic Encephalopathy Bronchopulmonary Dysplasia Short Bowel Syndrome

Interventions

Eligibility

Sex: ALLMin age: 1 DayMax age: 28 Days

Medical Language ↔ Plain English

Inclusion Criteria: * 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. Exclusion Criteria: * 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.

Outcomes

Primary Outcomes

Incidence of adverse reactions

Monitor oxygen, heart rate, temperature, rash, infection, etc

Time frame: Within 12 hours after UCB-MNCs infusion

Secondary Outcomes

Incidence of complications

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)

Time frame: a year

Imaging test results

Children with HIE: Brain diffusion tensor imaging (DTI) and 18F-Fluorodeoxyglucose positron emission tomography (18F-FDG-PET/CT)

Time frame: 2 weeks and 6 months after UCB-MNCs infusion

Electroencephalography (EEG) results

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

Time frame: 7 days UCB-MNCs infusion

Ventilator supporting time

Ventilator supporting time and oxygen demand will be recorded as important indications for clinical prognosis for children with HIE or BPD

Time frame: 1 month after UCB-MNCs infusion

Change of Gross Motor Performance Measure (GMPM)

GMPM is a standardized measurement tool for assessing quality of movement for children with HIE. Higher value means better motor quality

Time frame: 1, 3, 6 months after UCB-MNCs infusion

Change of Gross Motor Function Measure (GMFM)

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

Time frame: 1, 3, 6 months after UCB-MNCs infusion

Biomarker of HIE

pNF-H, marker of central nervous system axonal damage

Time frame: 7 days after UCB-MNCs infusion

Biomarker of BPD

AGER, marker of lung epithelial cell damage

Time frame: 7 days after UCB-MNCs infusion

Inflammatory indicators concentrations

Serum IL-6, IL-8, TNF concentrations will be measured via ELISA for children with HIE, BPD or SBS

Time frame: 7 days after UCB-MNCs infusion

Efficacy Evaluation of UCB-MNCs in the Treatment of Refractory Neonatal Diseases

Overview

Conditions

Interventions

Eligibility

Locations (1)

Outcomes

Primary Outcomes

Secondary Outcomes

Central Contacts