Feasibility and Safety of Umbilical Cord Blood Transfusion in the Treatment of Neonatal Cerebral Ischemia and Anemia

Umbilical Cord Blood Mononuclear Cell Bank in Hong Kong and Treatment of Neonatal Cerebral Ischemia and Anemia - Part IV Clinical Trial

The study is to investigate the feasibility and safety of autologous umbilical cord blood transfusion to treat the newborn infants with presence of clinical indications of neonatal hypoxic-ischemia encephalopathy (HIE) and anemia. Umbilical cord blood (UCB) is collected following labor and is transfused intravenously within 48 hours after the birth. Newborn infant without UCB available recieves the standard care will be enrolled as control group.

Following the autologous UCB transfusion in the study group or standard care in the control group, HIE subjects will be followed for 2 years for survival and neurodevelopmental outcomes and anemia subjects will be followed for 6 months to assess the survival and change of hematocrit and hemoglobin levels.

Study Overview

• Status: Unknown
• Conditions: Anemia, Neonatal; Hypoxic-Ischemic Encephalopathy; Hypoxia Neonatal; Cerebral Ischemia of Newborn
• Intervention / Treatment: Biological: autologous umbilical cord blood (UCB); Procedure: standard care

• Study Type: Interventional
• Enrollment (Anticipated): 40
• Phase: Phase 1

Contacts and Locations

Study Locations

• Hong Kong
  • Sha Tin, Hong Kong
    • Recruiting
    • The Chinese University of Hong Kong
    • Contact: Simon Lam, M.D.; Phone Number: +852 35052851
    • Principal Investigator: Simon Lam, MD
    • Sub-Investigator: Ronald Wang, MD
    • Sub-Investigator: Chi Kong Li, MD
    • Sub-Investigator: Eva Fung, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 2 days (CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• evidence of asphyxiation, defined by 5-minute Apgar score ≤ 5;
• evidence of HIE, defined by UCB pH <7.15 or base excess ≤ 10mM;
• subjects with HIE confirmed by clinical features and initial investigations;
• subjects with evidence of anemia, defined by hematocrit < 40% or hemoglobin ≤ 13g/dL within the first 96 hours of life;
• obtain the informed consent from parents

Exclusion Criteria:

• congestive cardiac failure;
• microcephaly, anencephaly, encephalocele, or other abnormality
• conjoint twins;
• chromosomal disorders
• fetal alcohol syndrome
• spinal bifida or other neural tube defects
• subjects have other neurological deficit conditions
• polycythemia
• congenital hematological malignancy
• investigator decision

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NON_RANDOMIZED
• Interventional Model: PARALLEL
• Masking: NONE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Study Group; autologous UCB transfusion	Biological: autologous umbilical cord blood (UCB); autologous UCB transfusion to the newborn infants presence of HIE and/or anemia within 48 hours after the birth
Other: Control Group; standard care	Procedure: standard care; standard care procedure to the newborn infants presence of HIE and/or anemia

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
HIE: Mortality	Mortality Rate of the HIE Subjects	6 months
Anemia: Change from Baseline Hematocrit	Change from Baseline Hematocrit of the Anemia Subjects	48 hours, 1 week, 3 months, 6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
HIE: HINE	Hammersmith Infant Neurological Examination (HINE) of the HIE Subjects	6 months, 1 year and 2 years
HIE: HNNE	Hammersmith Neonatal Neurological Examination (HNNE) of the HIE Subjects	-1 day, 3 months (before discharge)
HIE: GMDS	Griffiths Mental Development Scale (GMDS) of the HIE Subjects	6 months, 1 year and 2 years
HIE: CBCL	Child Behavior Checklist for Attention Deficit of the HIE Subjects	2 years
HIE: Q-CHAT	Quantitative Checklist for Autism in Toddlers of the HIE Subjects	2 years
Anemia: hemoglobin	Change from Baseline hemoglobin of the Anemia Subjects	48 hours, 1 week, 3 months and 6 months
Anemia: Oxygenation level	Change from Baseline SpO2 of the Anemia Subjects	48 hours, 1 week
Anemia: Oxidative Stress Level	Change of Baseline Isoprostane of the Anemia Subjects	48 hours, 1 week, 3 months, and 6 months
Anemia: Requirements of Packed Cell Transfusion	Frequency of Requirements of Packed Cell Transfusion by the Anemia Subjects	6 months

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of Adverse Event	Safety outcomes are Incidence of Adverse Events	2 years

Collaborators and Investigators

• Sponsor: Mononuclear Therapeutics Ltd.
• Collaborators: Chinese University of Hong Kong; China Spinal Cord Injury Network
• Investigators: Principal Investigator: Simon Lam, MD, Chinese University of Hong Kong

Study record dates

Study Major Dates

• Study Start (Actual): April 16, 2018
• Primary Completion (Anticipated): June 30, 2020
• Study Completion (Anticipated): December 30, 2020

Study Registration Dates

• First Submitted: November 15, 2017
• First Submitted That Met QC Criteria: November 21, 2017
• First Posted (Actual): November 24, 2017

Study Record Updates

• Last Update Posted (Actual): June 7, 2018
• Last Update Submitted That Met QC Criteria: June 5, 2018
• Last Verified: June 1, 2018

More Information

Terms related to this study

• Keywords: umbilical cord blood
• Additional Relevant MeSH Terms: Pathologic Processes; Necrosis; Cardiovascular Diseases; Vascular Diseases; Cerebrovascular Disorders; Central Nervous System Diseases; Nervous System Diseases; Hematologic Diseases; Infant, Newborn, Diseases; Signs and Symptoms, Respiratory; Infarction; Stroke; Brain Infarction; Hypoxia, Brain; Brain Ischemia; Ischemia; Brain Diseases; Anemia; Cerebral Infarction; Hypoxia; Anemia, Neonatal; Hypoxia-Ischemia, Brain
• Other Study ID Numbers: UCB-HIEA-01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

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