Safety of Autologous Cord Blood Cells for Preterm Infants.

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

Study Overview

• Status: Completed
• Conditions: Safety Issues; Neonatal Death; Effect of Drugs
• Intervention / Treatment: Combination product: Autologous Umbilical Cord Blood Stem Cells Therapy

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.

• Study Type: Interventional
• Enrollment (Actual): 15
• Phase: Early Phase 1

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 8 months (CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: PREVENTION
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: infusion group; Autologous Umbilical Cord Blood Stem Cells Therapy	Combination product: Autologous Umbilical Cord Blood Stem Cells Therapy; Autologous Umbilical Cord Blood Stem Cells Therapy,the dose is 5×107cells/kg, within 24 hours after birth

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Short-term safety of autologous umbilical cord blood stem cell infusion for preterm infants	vital signs; blood gas analysis; blood routine; liver and kidney function before and after infusion will be compared	during infusion
Short-term safety of autologous umbilical cord blood stem cell infusion for preterm infants	vital signs; blood gas analysis; blood routine; liver and kidney function before and after infusion will be compared	6 hours after infusion
Long-term safety(2-3 years follow-up after discharge) of autologous umbilical cord blood stem cell infusion for preterm infants	1. The growth and development curve will be drawn and compared	2years

Collaborators and Investigators

• Sponsor: Guangdong Women and Children Hospital
• Investigators: Principal Investigator: jie Yang, PHD, Guangdong Women and Children Hospital and Healthy Institutes

Study record dates

Study Major Dates

• Study Start (ACTUAL): January 1, 2009
• Primary Completion (ACTUAL): January 1, 2010
• Study Completion (ACTUAL): June 5, 2016

Study Registration Dates

• First Submitted: November 23, 2018
• First Submitted That Met QC Criteria: November 29, 2018
• First Posted (ACTUAL): November 30, 2018

Study Record Updates

• Last Update Posted (ACTUAL): June 2, 2020
• Last Update Submitted That Met QC Criteria: May 29, 2020
• Last Verified: May 1, 2020

More Information

Terms related to this study

• Keywords: Safety; Effect; neonates; Mechanism; Autologous Umbilical Cord Blood Stem Cells
• Additional Relevant MeSH Terms: Pathologic Processes; Pregnancy Complications; Death; Perinatal Death
• Other Study ID Numbers: Guang dong WCH

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No