A Phase 1 interventional study of Infusion of hCT-MSC in Moderate to Severe Hypoxic-ischemic Encephalopathy, sponsored by Joanne Kurtzberg, MD. Completed at 1 site in United States. Open to participants aged 0 Hours to 48 Hours. Per ClinicalTrials.gov, last updated 2024-04-12.
Sponsored by Joanne Kurtzberg, MD · Phase 1, Interventional, and Treatment
To determine the safety of single and repeated intravenous doses of hCT-MSC in newborn infants with HIE.
The purpose of this study is to assess the safety of one and two intravenous infusions of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC), the first administered in the first 48 postnatal hours, and the second at two months postnatal age, in term and near term infants with moderate to severe neonatal hypoxic-ischemic encephalopathy (HIE). This is a phase I, prospective, open-label trial designed to assess the safety of one or two intravenous doses of hCT-MSC in newborn infants with moderate to severe HIE who are recipients of therapeutic hypothermia. Infants born at 36 0/7 weeks gestation or later who have moderate to severe hypoxic-ischemic encephalopathy and are receiving therapeutic hypothermia will be eligible to participate. Investigators project an accrual of 6 patients. All infants will receive intravenous infusion(s) of hCT-MSCs. The first cohort of three infants will receive a single dose in the first 48 postnatal hours. If there are no safety concerns, the second cohort of three infants 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.
The potential risks associated with infusion of MSCs include a reaction to the product (rash, shortness of breath, wheezing, difficulty breathing, hypotension, swelling around the mouth, throat or eyes, tachycardia, diaphoresis), transmission of infection, and HLA sensitization. Another risk of this study is loss of confidentiality or privacy. Every effort will be made to keep the infant's medical record confidential. The results will be summarized using descriptive statistics and statistical testing as appropriate. Continuous secondary endpoints will be summarized using mean, standard deviation, CV%, median, minimum, and maximum.
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This study's enrollment of 6 is below the median of 58 across 454 interventional studies indexed under Brain Diseases.
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Exclusion Criteria:
The first cohort of three patients will receive a single dose in the first 48 postnatal hours.
Biological: Infusion of hCT-MSC
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
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.
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.
Time frame: 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.
Time frame: Up to 2 Weeks
Survival
Death prior to discharge from initial hospitalization
Time frame: Up to 6 months
Neurodevelopmental Assessments
1 year (12 - 16 postnatal months) Bayley Scales of Infant and Toddler Development, Third Edition (Bayley III) assessments in cognitive, language and motor development. Moderate to Severe CP will be assigned with cognitive score ,70, motor score ,70 and with Gross Motor Function Classification System \>=2.
Time frame: Up to 16 postnatal months
Plan to share: No
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This study is completed, as verified in Apr 2024. You cannot join it, but the record below documents what was studied.
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Joanne Kurtzberg, MD