Clinical trial NCT03760900: Safety of Autologous Cord Blood Cells for Preterm Infants.
Official title: Safety of Autologous Cord Blood Cells for Preterm Infants.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: EARLY_PHASE1
Conditions: Safety Issues
Effect of Drugs
Neonatal Death
Keywords: Autologous Umbilical Cord Blood Stem Cells
Safety
Effect
neonates
Mechanism
Lead sponsor: Guangdong Women and Children Hospital
Sponsor class: OTHER
First posted: 2018-11-30
Enrollment: 15
Sex: ALL
Maximum age (years): 37
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: PREVENTION

Brief summary: To assess the safety of autologous volume- and red blood cell (RBC)-reduced non-cryopreserved umbilical cord blood (UCB) cell infusion to preterm infants.

Detailed description: Preterm birth complications are one of the leading causes of death among children under 5 years of age. Despite advances in medical care, many survivors face a lifetime of disability, including mental and physical retardation, and chronic lung disease. More recently, both allogenic and autogenic cord blood cells have been applied in the treatment of neonatal conditions such as hypoxic-ischemic encephalopathy (HIE) and bronchopulmonary dysplasia (BPD).

Objective-To assess the safety of autologous volume- and red blood cell (RBC)-reduced non-cryopreserved umbilical cord blood (UCB) cell infusion to preterm infants.

Method- This study was a phase I, open-label, single-arm, single center trial to evaluate the safety of autologous, volume- and RBC-reduced non-cyropreserved UCB cell (5×107cells/kg) infusion for preterm infants \<37 weeks gestational age. UCB cell characteristics, pre- and post- infusion vital signs, laboratory investigations were recorded. Clinical data including mortality rates and preterm complications were recorded.

Results-After processing, (22.67±4.05) ml UCB cells in volume, (2.67±2.00)×108 cells in number, with (22.67±4.05)×106 CD34+, and (3.72±3.25)×105colony forming cells (CFU-GM), (99.7±0.17%) vitality were infused to 15 preterm infants within 8 hours after birth. No adverse effects were noticed during treatment. All fifteen patients who received UCB infusion survived. The duration of hospitalization ranged from 4 to 65 (30±23.6) days. Regarding preterm complications, no BPD, necrotizing enterocolitis (NEC), retinopathy of prematurity (ROP) were observed. There were 1/15 (7%) infant with intraventricular hemorrhage (IVH), and 5/15 (33.3%) infants with ventilation-associated pneumonia, 10/15(66.67%) with anemia respectively.

Conclusions-Collection, preparation and infusion of fresh autologous UCB cells to preterm infants is feasible and safe. Adequately powered randomized controlled studies are needed.

Eligibility criteria: Inborn Infants admitted to the Neonatal Intensive Care Unit (NICU) of Guang Dong Women and Children's Hospital were eligible if they were: 1. Preterm: \<37 weeks gestation 2. Without congenital abnormalities; 3. Without maternal chorioamnionitis 4. had available UCB. 5.the mother was negative for hepatitis B (HBsAg and/or HBeAg) and C virus (anti-HCV), syphilis, HIV (anti-HIV-1 and -2) and IgM against cytomegalovirus, rubella, toxoplasma and herpes simplex virus.

Primary outcomes:
measure: Short-term safety of autologous umbilical cord blood stem cell infusion for preterm infants
timeFrame: during infusion
measure: Short-term safety of autologous umbilical cord blood stem cell infusion for preterm infants
timeFrame: 6 hours after infusion
measure: Long-term safety(2-3 years follow-up after discharge) of autologous umbilical cord blood stem cell infusion for preterm infants
timeFrame: 2years
Secondary outcomes:
Other outcomes:
Locations:
Study officials:
affiliation: Guangdong Women and Children Hospital and Healthy Institutes
name: jie Yang, PHD
role: PRINCIPAL_INVESTIGATOR
References: