Clinical trial NCT06427642: Efficacy Evaluation of UCB-MNCs in the Treatment of Refractory Neonatal Diseases
Official title: Efficacy Evaluation of Umbilical Cord Blood-derived Mononuclear Cells in the Treatment of Refractory Neonatal Diseases
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Hypoxic-Ischemic Encephalopathy
Bronchopulmonary Dysplasia
Short Bowel Syndrome
Lead sponsor: Shandong Qilu Stem Cells Engineering Co., Ltd.
Sponsor class: INDUSTRY
Collaborators: name: Qilu Children's Hospital of Shandong University
First posted: 2024-05-24
Enrollment: 120
Sex: ALL
Minimum age (years): 1
Maximum age (years): 28
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Incidence of adverse reactions
timeFrame: Within 12 hours after UCB-MNCs infusion
Secondary outcomes:
measure: Incidence of complications
timeFrame: a year
measure: Imaging test results
timeFrame: 2 weeks and 6 months after UCB-MNCs infusion
measure: Electroencephalography (EEG) results
timeFrame: 7 days UCB-MNCs infusion
measure: Ventilator supporting time
timeFrame: 1 month after UCB-MNCs infusion
measure: Change of Gross Motor Performance Measure (GMPM)
timeFrame: 1, 3, 6 months after UCB-MNCs infusion
measure: Change of Gross Motor Function Measure (GMFM)
timeFrame: 1, 3, 6 months after UCB-MNCs infusion
measure: Biomarker of HIE
timeFrame: 7 days after UCB-MNCs infusion
measure: Biomarker of BPD
timeFrame: 7 days after UCB-MNCs infusion
measure: Inflammatory indicators concentrations
timeFrame: 7 days after UCB-MNCs infusion
Other outcomes:
Locations:
city: Jinan
country: China
facility: Qilu Children's Hospital of Shandong University
state: Shandong
Study officials:
affiliation: Qilu Children's Hospital of Shandong University
name: Xiaoying Li, MD
role: PRINCIPAL_INVESTIGATOR
References: