Study of hCT-MSC in Newborn Infants With Moderate or Severe HIE

NCT03635450 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: Pro00100253
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

To determine the safety of single and repeated intravenous doses of hCT-MSC in newborn infants with HIE.

Conditions

Moderate to Severe Hypoxic-ischemic Encephalopathy

Keywords

hypoxic-ischemic encephalopathy; newborn infants; therapeutic hypothermia; hCT-MSC, an Umbilical Cord Tissue-Derived Mesenchymal Stromal Cell Product

Outcome Measures

Primary Outcomes (2)

• Incidence of infusion reactions

  for this study, infusion reactions are defined as anaphylactic or anaphylactoid reactions with clinical signs inclusive of skin rashes, bronchospasm, angioedema, myocardial infarcts, arrhythmias, and acute lung injury.

  24 hours after each infusion

• Incidence of Infections post-infusion

  for this study, infections recorded as safety endpoints will be defined as bacterial, viral or fungal infections identified by culture or molecular methodologies within two weeks after administration of hCT-MSC.

  Up to 2 Weeks

Secondary Outcomes (2)

• Survival

  Up to 6 months

• Neurodevelopmental Assessments

  Up to 16 postnatal months

Study Arms (2)

First cohort of 3 subjects enrolled

EXPERIMENTAL

The first cohort of three patients will receive a single dose in the first 48 postnatal hours.

Biological: Infusion of hCT-MSC

Second cohort of 3 subjects enrolled

EXPERIMENTAL

If there are no safety concerns after the first cohort of 3 subjects are infused then the second cohort of three patients will receive two doses, with the first dose given in the first 48 postnatal hours and the second dose given approximately two months after the first dose.

Biological: Infusion of hCT-MSC

Interventions

Infusion of hCT-MSC BIOLOGICAL

Infants who meet enrollment criteria for moderate to severe hypoxic ischemic encephalopathy will receive 1 infusion of hCT-MSC within the first 48 postnatal hours. hCT-MSCs are a product of allogeneic cells manufactured from digested umbilical cord tissue that is expanded in culture, cryopreserved and banked. hCT-MSCs are manufactured from umbilical cord tissue donated to the Carolinas Cord Blood Bank, an FDA-licensed, FACT-accredited, public cord blood bank at Duke University Medical Center, after written informed consent from the baby's mother. Cord tissue is harvested from the placentas of male babies delivered by elective C-section after a normal, full- term pregnancy.

First cohort of 3 subjects enrolled; Second cohort of 3 subjects enrolled

Eligibility Criteria

• Age: 0 Hours - 48 Hours
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• /7th weeks gestation or older at the time of delivery.
• Able to receive one dose of hCT-MSCs in the first 48 postnatal hours
• Willingness to return for one year assessments.
• Signs of encephalopathy within 6 hours of age

You may not qualify if:

• Major congenital or chromosomal abnormalities
• Severe growth restriction (birth weight \<1800 g)
• Opinion by attending neonatologist that the study may interfere with clinical treatment or safety of subject
• Moribund neonates for whom no further treatment is planned
• Infants whose mothers have unknown serologies for Hepatitis B or HIV
• Infants born to mothers are known to be HIV, Hepatitis B, Hepatitis C or who have active syphilis or CMV infection in pregnancy
• Infants suspected of overwhelming sepsis
• ECMO initiated or likely in the first 48 hours of life
• Mother suspected to have intraamniotic infection at time of birth.
• ALL blood gases (cord and postnatal) done within the first 60 minutes had a pH \> 7.15 AND base deficit \< 10 mEq/L (source can be arterial, venous or capillary)
• Mother with documented Zika infection during this pregnancy
• Availability of autologous cord blood collected and usable in the randomized trial of autologous volume- and red blood cell-reduced cord blood cells (Duke IRB Pro00066647; clinical trials.gov link: https://clinicaltrials.gov/ct2/show/NCT02612155 )

Sponsors & Collaborators

• Joanne Kurtzberg, MD: lead
• Duke Clinical and Translational Science Institute (CTSI), part of the NIH Clinical and Translational Science Awards (CTSA): collaborator

Study Sites (1)

Duke University

Durham, North Carolina, 27705, United States

Location

MeSH Terms

Conditions

Hypoxia-Ischemia, Brain

Condition Hierarchy (Ancestors)

Brain Ischemia; Cerebrovascular Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Hypoxia, Brain; Vascular Diseases; Cardiovascular Diseases; Hypoxia; Signs and Symptoms, Respiratory; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• Michael Cotten, MD

  Duke University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor Department of Pediatrics

Model Details: The first cohort of three patients will receive a single dose in the first 48 postnatal hours. If there are no safety concerns, the second cohort of three patients will receive two doses, with the first dose given in the first 48 postnatal hours and the second dose given approximately two months after the first dose.

Study Record Dates

• First Submitted: August 15, 2018
• First Posted: August 17, 2018
• Study Start: December 27, 2018
• Primary Completion: July 28, 2019
• Study Completion: December 28, 2020
• Last Updated: April 12, 2024

Record last verified: 2024-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)