Autologous Cord Blood and Human Placental Derived Stem Cells in Neonates With Severe Hypoxic-Ischemic Encephalopathy (HPDSC+HIE)

• ClinicalTrials.gov Identifier: NCT02434965
• Recruitment Status: Withdrawn
• First Posted: May 6, 2015
• Last Update Posted: February 23, 2021
• Sponsor: New York Medical College
• Collaborator: Celgene
• Information provided by (Responsible Party): New York Medical College

Study Description

Brief Summary:

The purpose of this study is to investigate the safety and effectiveness of autologous human placental-derived stem cells (HPDSC) in combination with autologous cord blood in neonates with severe hypoxic-ischemic encephalopathy.

Condition or disease	Intervention/treatment	Phase
Severe Hypoxic-ischemic Encephalopathy	Drug: HPDSC; Drug: Cord blood	Phase 2

Detailed Description:

The primary aim of this study is to determine the safety, tolerability and feasibility of intravenous administration of autologous cord blood (CB) and autologous human placental derived stem cells (HPDSC) in neonates with severe hypoxic-ischemic encephalopathy (HIE). It is hypothesized that the administration of autologous CB and autologous HPDSC will be safe and well tolerated in neonates with severe HIE.

Additionally, postnatal neuro-developmental outcomes in neonates with HIE after autologous CB and HPDSC therapy will be measured; HIE injury to the neonate/infant brain post autologous CB and HPDSC therapy by imaging will be characterized; the pluripotent stem cell properties of CB and HPDSC will be characterized; serum levels of selected circulating cytokine and neurotrophic factors in neonates with HIE before and after autologous CB and HPDSC therapy will be compared and immune cell phenotype and function in neonates with HIE before and after autologous CB and HPDSC therapy will be compared.

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 0 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Safety and Feasibility Study of Autologous Cord Blood (CB) and Human Placental Derived Stem Cells (HPDSC) in Neonates With Severe Hypoxic-Ischemic Encephalopathy (HIE)
• Estimated Study Start Date: December 2019
• Estimated Primary Completion Date: January 2021
• Estimated Study Completion Date: January 2022

Arms and Interventions

Arm

Intervention/treatment

Experimental: Autologous Cord Blood and HPDSC; Autologous cord blood and placental blood will be collected after birth of child and administered in divided aliquots during the first week of life.

Drug: HPDSC; Autologous HPDSC collected after birth will be infused in aliquots. one-half of the HPDSC infused on Day 2; one-half of the collected HPDSC will be infused on Day 8.; Drug: Cord blood; Autologous Cord Blood collected after birth will be infused in aliquots. One-third of the collected cord blood will be infused within the first 24 hours after birth (Day 0); one-third of the collected cord blood will be infused on day 3; and one-third of the collected cord blood unit will be infused on Day 7.

Outcome Measures

Primary Outcome Measures :

1. Number of subjects with infusion reaction as a measure of safety and tolerability [ Time Frame: within the first 30 days ]
   Any infusion reaction to autologous human placental-derived stem cells (HPDSC) administered in conjunction autologous cord blood in neonates with severe hypoxic-ischemic encephalopathy will be assessed for safety and tolerability

Secondary Outcome Measures :

1. Improvement in neurological condition [ Time Frame: 2 years post HPDSC infusion ]
   Improvement in neurological condition as shown on head MRI, DTI and neurological development by Sarnat testing.

Eligibility Criteria

• Ages Eligible for Study: 1 Minute to 6 Hours (Child)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

• Gestational age ≥ 36 weeks
• Birth weight ≥ 1800 grams
• Postnatal age after birth of less than 6 hours
• Autologous cord blood and HPDSCs available for infusion
• Plus one or more of the following criteria: Apgar ≤ 5 at 10 minutes of postnatal age, or Continued need for resuscitation ≥10 min after birth, or Acidosis-cord blood pH or arterial blood pH within 60 minutes of birth ≤ 7.0 pH, or Base deficit ≥ minus 16mEq in cord blood and within 60 min of birth.
• Plus Moderate to Severe Altered State of Consciousness, by one or more of the following: Hypotonia, or Abnormal reflexes, or Absent/weak suck.

Exclusion Criteria:

• Major life-threatening or surgical anomalies
• Polycythemia (hematocrit > 65%)
• Congenital infection based on antenatal diagnosis of TORCH infection
• Parental refusal for study
• Infant expected to live < 24h, medical care is considered futile and no additional therapy will be offered by the attending neonatologist

Contacts and Locations

Locations

United States, New York

New York Medical College

Valhalla, New York, United States, 10595

Sponsors and Collaborators

New York Medical College

Celgene

Investigators

• Principal Investigator: Mitchell S Cairo, MD; New York Medical College

More Information

• Responsible Party: New York Medical College
• ClinicalTrials.gov Identifier: NCT02434965
• Other Study ID Numbers: NYMC-554
• First Posted: May 6, 2015
• Last Update Posted: February 23, 2021
• Last Verified: September 2019

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No
• Plan Description: Study not enrolling

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by New York Medical College:

Neonates; hypoxic-ischemic encephalopathy; human placental-derived stem cells; autologous

Additional relevant MeSH terms:

Brain Diseases; Brain Ischemia; Hypoxia-Ischemia, Brain; Ischemia; Hypoxia; Pathologic Processes; Central Nervous System Diseases; Nervous System Diseases; Signs and Symptoms, Respiratory; Cerebrovascular Disorders; Vascular Diseases; Cardiovascular Diseases; Hypoxia, Brain