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Autologous Umbilical Cord Blood Treatment of Neonate With CHD

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ClinicalTrials.gov Identifier: NCT03558269
Recruitment Status : Recruiting
First Posted : June 15, 2018
Last Update Posted : January 20, 2021
Sponsor:
Information provided by (Responsible Party):
Dr. Omer Bar-Yosef, Sheba Medical Center

Study Description
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Brief Summary:

The aim of the study is to evaluate the feasibility and safety and efficacy of collecting and infusing autologous umbilical cord blood (UCB) in newborn infants with hypoplastic left heart syndrome (HLHS) and transposition of great arteries (TGA).

Rationale: Neonates with HLHS and TGA have significant brain injury as demonstrated by peri-surgical MRI. Moreover, there a substantial tendency to suffer from chronic cardiac condition as low cardiac output state and valvular insufficiency. Treatment of neonates after hypoxic ischemic injury at birth with autologous UCB was shown to safe and improved developmental outcome. The effect of UCB is most likely achieved by reduction of free radicals injury and pro-inflammatory and apoptotic process.

Hypothesis: Treatment with UCB immediately after the first cardiac surgey, with in the first week life will reduce the brain injury demonstrated by MRI and reduce the choronic cardiac problems


Condition or disease Intervention/treatment Phase
Congenital Heart Disease Brain Injuries Low Cardiac Output Syndrome Neonatal Disorder Biological: Autlogous UCB infusion Phase 1 Phase 2

Detailed Description:

The aim of this pilot study is to evaluate the safety and feasibility of infusion of autologous umbilical cord blood (UCB) in neonates with with hypoplastic left heart syndrome (HLHS) or transposition of great arteries (TGA).

This is a prospective, matched control study, Phase I-II trial to evaluate the safety and efficacy of autologous UCB infusion in neonates with HLHS or TGA. The study group will consist of neonate that were diagnosed by prenatal fetal ultrasound with HLHS or TGA and their UCB was collected at the time of delivery.

The study group will include the patients with UCB and the control group will be patients without UCB.

All patients will have their surgery within 2 weeks from birth. The UCB will be infused to the patients in study group as soon as the patient has stabilized after the surgery and not more than 7 days after surgery.

Both groups will be followed similarity:

During hospitalization:

Neurological and cardiac evaluation a day before surgery, 7 days after surgery and at discharge.

Blood tests for immune and growth factors a day before surgery, at the time of infusion (or intended infusion for the control group) ,1 and 7 days after infusion.

have a cardiac and brain MRI before the surgery and within 14 days after surgery.

Brain and cardiac MRI before surgery, 7-14 days after surgery.

Ambulatory follow up (similar to routine follow up):

Cardiac and neuro-developmental evaluation at 1,6,12 month Blood tests for immune and growth factors at 1 month

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: Single (Outcomes Assessor)
Masking Description: The outcome assessor will not be exposed to the participant group
Primary Purpose: Treatment
Official Title: Pilot Study to Assess the Efficacy of Autologous Umbilical Cord Blood Treatment of Brain and Heart Injury in Neonates With Congenital Heart Defect
Actual Study Start Date : February 1, 2019
Estimated Primary Completion Date : May 1, 2021
Estimated Study Completion Date : April 1, 2023
Arms and Interventions
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Arm Intervention/treatment
Experimental: Study group
This group will receive UCB after the first palliative surgery
 Biological: Autlogous UCB infusion
Infants delivered with hypoplastic left heart syndrome and transposition of great arteries undergoing surgery in the 1st 2 weeks of life will be treated with autologous UCB after the surgery. Each group will be followed by MRI before (1-3 days) and after the surgery between at 10 POD. Developmental follow up will be done at 1, 6 and 12 months. Another brain MRI will be done at 6 months of age.
No Intervention: Control group
This group will not receive any treatment


Outcome Measures
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Primary Outcome Measures :
  1. Neuroimaging of the brain before and after the surgery (ref 1) [ Time Frame: The time frame between MRI before and after surgery is 10-13 days ]
    Change in MRI of the brain before (between 1-3 days) and after (10 days) the surgery between the intervention and control group. The change will be quantified by MRI brain injury severity (BIS) score quantifying white matter injury, stroke and brain hemorrhage. Diffusion tensor imaging measures will also be quantified by using average diffusivity and fractional anisotropy calculated from frontal, parietal, temporal and occipital white matter.


Secondary Outcome Measures :
  1. Neuro-development at one month [ Time Frame: One month ]
    General movements assessment (ref 2)

  2. Neuro-development at six month [ Time Frame: 6 months ]
    Gross Motor Function Measure 66 (ref 3)

  3. Neuro-development at 12 month [ Time Frame: 12months ]
    Gross Motor Function Measure 66 (ref 3)


Eligibility Criteria
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Ages Eligible for Study:   up to 14 Days   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. At least 35 weeks of gestation
  2. Prenatal diagnosis of HLHS including mitral stenosis/atresia, aortic stenosis/atresia, arch hypoplasia and the presence of hypoplastic left ventricle with or without VSD.
  3. Norwood procedure will take place within 14 days from birth.
  4. Treatment with cord blood should be given within 7 days after surgery.
  5. Parental informed consent for collection of umbilical cord blood.

Exclusion Criteria:

  1. Total nucleated cells (TNC) lower than 1X107 in the collected umbilical cord blood unit.
  2. Infected umbilical cord blood unit.
  3. Parents refusal to continue in the study at any stage.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03558269


Contacts
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Contact: Omer Bar-Yosef, M.D.-Ph.D. 972-3-5302687 omer.baryosef@sheba.health.gov.il
Contact: Amir Vardi, M.D. 972-3-5308010 amir.vardi@sheba.health.gov.il

Locations
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Israel
Sheba Medical Center Recruiting
Ramat Gan, Israel, 5621000
Contact: Omer Bar-Yosef, M.D._Ph.D.    +972-3-5302687    omer.baryosef@sheba.health.gov.il   
Sub-Investigator: Omer Bar-Yosef, M.D.-Ph.D.         
Sub-Investigator: Amir Vardi, M.D.         
Sub-Investigator: Elad Jacobi, M.D.         
Sub-Investigator: Tal Tirosh, M.D.         
Sub-Investigator: Yishai Salem, M.D.         
Sub-Investigator: David Mishali, M.D.         
Sponsors and Collaborators
Sheba Medical Center
More Information
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Publications of Results:

Other Publications:

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Responsible Party: Dr. Omer Bar-Yosef, Principal Investigator, Sheba Medical Center
ClinicalTrials.gov Identifier: NCT03558269    
Other Study ID Numbers: 3757-16-SMC
First Posted: June 15, 2018    Key Record Dates
Last Update Posted: January 20, 2021
Last Verified: January 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Plan Description: By excel data base

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Dr. Omer Bar-Yosef, Sheba Medical Center:
Autologous Umbilical cord blood infusion
Congenital Heart Disease
Brain Injuries
Low Cardiac Output Syndrome
Neonatal Disorder
Additional relevant MeSH terms:
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Brain Injuries
Heart Diseases
Heart Defects, Congenital
Cardiac Output, Low
Infant, Newborn, Diseases
Wounds and Injuries
Cardiovascular Diseases
 Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Craniocerebral Trauma
Trauma, Nervous System
Cardiovascular Abnormalities
Congenital Abnormalities