Autologous Umbilical Cord Blood Treatment of Neonate With CHD

January 17, 2021 updated by: Dr. Omer Bar-Yosef, Sheba Medical Center

Pilot Study to Assess the Efficacy of Autologous Umbilical Cord Blood Treatment of Brain and Heart Injury in Neonates With Congenital Heart Defect

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

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Anticipated)

60

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Israel
      • Ramat Gan, Israel, 5621000
        • Recruiting
        • Sheba Medical Center
        • Contact:
        • 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.

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

3 days to 2 weeks (CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NON_RANDOMIZED
  • Interventional Model: PARALLEL
  • Masking: SINGLE

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
The time frame between MRI before and after surgery is 10-13 days

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Neuro-development at one month
Time Frame: One month
General movements assessment (ref 2)
One month
Neuro-development at six month
Time Frame: 6 months
Gross Motor Function Measure 66 (ref 3)
6 months
Neuro-development at 12 month
Time Frame: 12months
Gross Motor Function Measure 66 (ref 3)
12months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sheba Medical Center

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

February 1, 2019

Primary Completion (ANTICIPATED)

May 1, 2021

Study Completion (ANTICIPATED)

April 1, 2023

Study Registration Dates

First Submitted

May 12, 2018

First Submitted That Met QC Criteria

June 13, 2018

First Posted (ACTUAL)

June 15, 2018

Study Record Updates

Last Update Posted (ACTUAL)

January 20, 2021

Last Update Submitted That Met QC Criteria

January 17, 2021

Last Verified

January 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 3757-16-SMC

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

IPD Plan Description

By excel data base

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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