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

Shandong Qilu Stem Cells Engineering

Status: Enrolling

Conditions: Short Bowel Syndrome; Bronchopulmonary Dysplasia; Hypoxic-Ischemic Encephalopathy

Treatments: Biological: Mononuclear cells; Procedure: Total parenteral nutrition; Device: Mild hypothermia therapy; Device: Breathing support technique

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT06427642

MNCs-2024

Details and patient eligibility

About

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.

Enrollment: 120 estimated patients

Sex: All

Ages: 1 to 28 days old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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120 participants in 6 patient groups

Experimental group for children with HIE

Experimental group

Description:

Intravenous infusion of UCB-MNCs is given within 24 hours of being identified as a high-risk patient

Treatment:

Biological: Mononuclear cells

Device: Mild hypothermia therapy

Control group for children with HIE

Active Comparator group

Description:

Mild hypothermia therapy is given for 72 hours to maintain anal temperature between 33.5°C and 34°C

Treatment:

Device: Mild hypothermia therapy

Experimental group for children with BPD

Experimental group

Description:

Intravenous infusion of UCB-MNCs is given within 24 hours of being identified as a high-risk patient

Treatment:

Biological: Mononuclear cells

Device: Breathing support technique

Control group for children with BPD

Active Comparator group

Description:

Clinical routine treatment

Treatment:

Device: Breathing support technique

Experimental group for children with SBS

Experimental group

Description:

Intravenous infusion of UCB-MNCs

Treatment:

Procedure: Total parenteral nutrition

Biological: Mononuclear cells

Control group for children with SBS

Active Comparator group

Description:

Clinical routine treatment

Treatment:

Procedure: Total parenteral nutrition