Autologous Cord Blood Cell Therapy for Neonatal Encephalopathy

Phase 1

Completed

• Conditions: Neonatal Encephalopathy; Hypoxic-ischemic Encephalopathy
• Interventions: Other: Autologous umbilical cord blood cells

• Registration Number: NCT02256618
• Lead Sponsor: Neonatal Encephalopathy Consortium, Japan

Brief Summary

This is a pilot study to test feasibility and safety of intravenous infusion of autologous umbilical cord blood cells in the first 72 hours after birth if a neonate is born with signs of encephalopathy.

Detailed Description

This is a multicenter pilot study to evaluate the feasibility and safety of intravenous infusions of autologous (the patient's own) umbilical cord blood cells in term gestation newborns with neonatal encephalopathy (hypoxic-ischemic encephalopathy). If a neonate is born with signs of moderate to severe encephalopathy and cooled for the encephalopathy, the neonate can receive their own non-cryopreserved volume- and red blood cell-reduced cord blood cells. The cord blood cells are divided into 3 doses and infused at 12-24, 36-48, and 60-72 hours after the birth. Infants will be followed for safety and neurodevelopmental outcome up to 18 months.

Study Timeline

• Study Start: 2014-08
• Study First Submitted: 2014-10-01
• Study First Submitted QC: 2014-10-01
• Study First Posted: 2014-10-03
• Primary Completion: 2017-10
• Study Completion: 2019-07
• Status Verified: 2019-10
• Last Update Submitted: 2019-10-28
• Last Update Posted: 2019-10-29

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 6

Inclusion Criteria

Infants are eligible if they meet all the following inclusion criteria except 4.

1. ≥36 weeks gestation
2. Either a 10-minute Apgar score ≤5, continued need for resuscitation for at least 10 minutes, or severe acidosis, defined as pH <7.0 or base deficit ≥16 mmol/L in a sample of umbilical cord blood or any blood during the first hour after birth
3. Moderate to severe encephalopathy (Sarnat II to III)
4. A moderately or severely abnormal background amplitude-integrated EEG (aEEG) voltage, or seizures identified by aEEG, if monitored
5. Up to 24 hours of age
6. Autologous umbilical cord blood available to infuse within 3 days after birth
7. A person with parental authority must have consented for the study.

Exclusion Criteria

1. Known major congenital anomalies, such as chromosomal anomalies, heart diseases
2. Major intracranial hemorrhage identified by brain ultrasonography or computed tomography
3. Severe growth restriction, with birth-weight less than 1800 g
4. Severe infectious disease, such as sepsis
5. Hyperkalemia
6. Outborn infants (Infants born at hospitals other than the study sites)
7. Volume of collected cord blood <40 ml
8. Infants judged critically ill and unlikely to benefit from neonatal intensive care by the attending neonatologist

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Cell therapy	Autologous umbilical cord blood cells	Infants who are born at the study sites, have moderate to severe encephalopathy, and have cord blood available for infusion

Primary Outcome Measures

Name	Time	Method
Adverse event rates	first 30 postnatal days	Adverse event rates (combined rate of death, continuous respiratory support, and continuous use of vasopressor) will be compared between the cell recipients and historical controls at 30 days of age.

Secondary Outcome Measures

Name	Time	Method
Efficacy	18 months	Neuroimaging at 12 months of age and neurodevelopmental function at 18 months of age will be compared between the cell recipients and historical controls.

Trial Locations

Locations (6)

Nagoya University Hospital; 🇯🇵 Nagoya, Aichi, Japan

Yodogawa Christian Hospital; 🇯🇵 Osaka, Japan

Kurashiki Central Hospital; 🇯🇵 Kurashiki, Okayama, Japan

Saitama Medical University; 🇯🇵 Kawagoe, Saitama, Japan

Osaka City General Hospital; 🇯🇵 Osaka, Japan

Osaka City University; 🇯🇵 Osaka, Japan