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

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ClinicalTrials.gov Identifier: NCT03635450
Recruitment Status : Active, not recruiting
First Posted : August 17, 2018
Last Update Posted : November 14, 2019
Sponsor:
Collaborator:
The Duke Clinical and Translational Science Institute (CTSI), part of the National Institutes of Health's Clinical and Translational Science Awards (CTSA)
Information provided by (Responsible Party):
Joanne Kurtzberg, MD, Duke University

Tracking Information
First Submitted Date  ICMJE August 15, 2018
First Posted Date  ICMJE August 17, 2018
Last Update Posted Date November 14, 2019
Actual Study Start Date  ICMJE December 27, 2018
Actual Primary Completion Date July 28, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 15, 2018)
  • Incidence of infusion reactions [ Time Frame: 24 hours after each infusion ]
    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.
  • Incidence of Infections post-infusion [ Time Frame: Up to 2 Weeks ]
    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.
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03635450 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: August 16, 2018)
  • Survival [ Time Frame: Up to 6 months ]
    Death prior to discharge from initial hospitalization
  • Neurodevelopmental Assessments [ Time Frame: Up to 16 postnatal months ]
    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.
Original Secondary Outcome Measures  ICMJE
 (submitted: August 15, 2018)
  • Survival [ Time Frame: Up to 6 months ]
    Death prior to discharge from initial hospitalization
  • Neurodevelopmental Assessments [ Time Frame: Up to 16 postnatal months ]
    6. 1 year (12 - 16 postnatal months) Bayley III assessments in cognitive, language and motor development
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study of hCT-MSC in Newborn Infants With Moderate or Severe HIE
Official Title  ICMJE A Phase I Study of hCT-MSC, an Umbilical Cord-Derived Mesenchymal Stromal Cell Product, in Newborn Infants With Moderate or Severe Hypoxic- Ischemic Neonatal Encephalopathy.
Brief Summary To determine the safety of single and repeated intravenous doses of hCT-MSC in newborn infants with HIE.
Detailed Description

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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:
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.
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Moderate to Severe Hypoxic-ischemic Encephalopathy
Intervention  ICMJE 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.
Study Arms  ICMJE
  • Experimental: First cohort of 3 subjects enrolled
    The first cohort of three patients will receive a single dose in the first 48 postnatal hours.
    Intervention: Biological: Infusion of hCT-MSC
  • Experimental: Second cohort of 3 subjects enrolled
    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.
    Intervention: Biological: Infusion of hCT-MSC
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: August 15, 2018)
6
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE July 28, 2020
Actual Primary Completion Date July 28, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • 36 0/7th 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

Exclusion Criteria:

  • 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 )
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 48 Hours   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03635450
Other Study ID Numbers  ICMJE Pro00100253
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Joanne Kurtzberg, MD, Duke University
Study Sponsor  ICMJE Joanne Kurtzberg, MD
Collaborators  ICMJE The Duke Clinical and Translational Science Institute (CTSI), part of the National Institutes of Health's Clinical and Translational Science Awards (CTSA)
Investigators  ICMJE
Principal Investigator: Michael Cotten, MD Duke University
PRS Account Duke University
Verification Date November 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP