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

Bronchopulmonary Dysplasia Clinical Trial

Official title:

Efficacy Evaluation of Umbilical Cord Blood-derived Mononuclear Cells in the Treatment of Refractory Neonatal Diseases

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06427642
• Other study ID #: MNCs-2024
• Status: Recruiting
• Phase: N/A
• Start date: April 1, 2022
• Est. completion date: April 30, 2025

Study information

• Verified date: May 2024
• Source: Shandong Qilu Stem Cells Engineering Co., Ltd.
• Contact: Yujie Han, MD
• Phone: +86 187 5414 6336
• Email: 410358192[@]qq.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 120
• Est. completion date: April 30, 2025
• Est. primary completion date: May 30, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Day to 28 Days

Eligibility

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.

Related Conditions & MeSH terms

• Brain Diseases
• Brain Ischemia
• Bronchopulmonary Dysplasia
• Hypoxia-Ischemia, Brain
• Hypoxic-Ischemic Encephalopathy
• Infant, Newborn, Diseases
• Short Bowel Syndrome

Intervention

Biological:
• Mononuclear cells
UCB-MNCs are obtained from umbilical cord blood by density gradient centrifugation

Device:
• Mild hypothermia therapy
Mild hypothermia therapy via hypothermia therapy apparatus
• Breathing support technique
Breathing support via ventilator

Procedure:
• Total parenteral nutrition
Liquid nutrition injected directly into the bloodstream

Locations

Country	Name	City	State
China	Qilu Children's Hospital of Shandong University	Jinan	Shandong

Sponsors (2)

Lead Sponsor	Collaborator
Shandong Qilu Stem Cells Engineering Co., Ltd.	Qilu Children's Hospital of Shandong University

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of adverse reactions	Monitor oxygen, heart rate, temperature, rash, infection, etc	Within 12 hours after UCB-MNCs infusion
Secondary	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)	a year
Secondary	Imaging test results	Children with HIE: Brain diffusion tensor imaging (DTI) and 18F-Fluorodeoxyglucose positron emission tomography (18F-FDG-PET/CT)	2 weeks and 6 months after UCB-MNCs infusion
Secondary	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	7 days UCB-MNCs infusion
Secondary	Ventilator supporting time	Ventilator supporting time and oxygen demand will be recorded as important indications for clinical prognosis for children with HIE or BPD	1 month after UCB-MNCs infusion
Secondary	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	1, 3, 6 months after UCB-MNCs infusion
Secondary	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	1, 3, 6 months after UCB-MNCs infusion
Secondary	Biomarker of HIE	pNF-H, marker of central nervous system axonal damage	7 days after UCB-MNCs infusion
Secondary	Biomarker of BPD	AGER, marker of lung epithelial cell damage	7 days after UCB-MNCs infusion
Secondary	Inflammatory indicators concentrations	Serum IL-6, IL-8, TNF concentrations will be measured via ELISA for children with HIE, BPD or SBS	7 days after UCB-MNCs infusion