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Congenital Heart DiseasePhase 1/2NCT03558269

Autologous Umbilical Cord Blood Treatment of Neonate With CHD

A Phase 1/2 study testing Autlogous UCB infusion in people with Congenital Heart Disease and Brain Injuries, run by Sheba Medical Center at 1 site in Israel.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial 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). Read more about this study

Who is it for?
Ages 3 to 14 days
What is being tested?
Autlogous UCB infusion
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
60 participants
Where
1 siteIsrael
Study dates
1 Feb 2019to 1 Apr 2023
Phase
Phase 1/2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 3 to 14 days
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

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

Who cannot join

  • Total nucleated cells (TNC) lower than 1X107 in the collected umbilical cord blood unit.
  • Infected umbilical cord blood unit.
  • Parents refusal to continue in the study at any stage.

Trial locations and contacts

1 site in 1 country.

Omer Bar-Yosef, M.D.-Ph.D. contact

972-3-5302687

omer.baryosef@sheba.health.gov.il

Amir Vardi, M.D. contact

972-3-5308010

amir.vardi@sheba.health.gov.il

Israel1 site, 1 recruiting

  • Sheba Medical Center

    Ramat Gan, 5621000

    Omer Bar-Yosef, M.D._Ph.D., +972-3-5302687, omer.baryosef@sheba.health.gov.il

    Omer Bar-Yosef, M.D.-Ph.D.

    Recruiting

Other trials in Israel

About this study

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

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

Read the 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

Being tested
Autlogous UCB infusion
Keywords
Autologous Umbilical cord blood infusion, Congenital Heart Disease, Brain Injuries, Low Cardiac Output Syndrome, Neonatal Disorder
Medical subject headings
Heart Defects, Congenital, Brain Injuries, Cardiac Output, Low, Infant, Newborn, Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Autlogous UCB infusionBiological

    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.

Study groups in NCT03558269
Study groupRoleWhat they receive
Study groupExperimentalThis group will receive UCB after the first palliative surgery
Receives: Biological: Autlogous UCB infusion
Control groupNo interventionThis group will not receive any treatment

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Neuroimaging of the brain before and after the surgery (ref 1)

    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.

    Measured: The time frame between MRI before and after surgery is 10-13 days

Secondary outcomes (3)

  1. Neuro-development at one month

    General movements assessment (ref 2)

    Measured: One month

  2. Neuro-development at six month

    Gross Motor Function Measure 66 (ref 3)

    Measured: 6 months

  3. Neuro-development at 12 month

    Gross Motor Function Measure 66 (ref 3)

    Measured: 12months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted15 Jun 2018
  2. Study start1 Feb 2019
  3. Primary completion1 May 2021 (estimated)
  4. Study completion1 Apr 2023 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
Single blind
Who is masked
Outcomes assessor
Enrolment
60 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03558269
Sponsor's protocol ID
3757-16-SMC
Status last verified
Jan 2021
First submitted
12 May 2018
Last updated
20 Jan 2021
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Undecided: By excel data base

Publications

  • Kwong AKL, Fitzgerald TL, Doyle LW, Cheong JLY, Spittle AJ. Predictive validity of spontaneous early infant movement for later cerebral palsy: a systematic review. Dev Med Child Neurol. 2018 May;60(5):480-489. doi: 10.1111/dmcn.13697. Epub 2018 Feb 22.

    PubMed 29468662

  • Alotaibi M, Long T, Kennedy E, Bavishi S. The efficacy of GMFM-88 and GMFM-66 to detect changes in gross motor function in children with cerebral palsy (CP): a literature review. Disabil Rehabil. 2014;36(8):617-27. doi: 10.3109/09638288.2013.805820. Epub 2013 Jun 26.

    PubMed 23802141

  • Dimitropoulos A, McQuillen PS, Sethi V, Moosa A, Chau V, Xu D, Brant R, Azakie A, Campbell A, Barkovich AJ, Poskitt KJ, Miller SP. Brain injury and development in newborns with critical congenital heart disease. Neurology. 2013 Jul 16;81(3):241-8. doi: 10.1212/WNL.0b013e31829bfdcf. Epub 2013 Jun 14.

    PubMed 23771484

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Autlogous UCB infusion in people with Congenital Heart Disease and Brain Injuries, run by Sheba Medical Center at 1 site in Israel.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: status not confirmed, as of the sponsor's last update on 20 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Israel. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

Contact Omer Bar-Yosef, M.D.-Ph.D. (972-3-5302687, omer.baryosef@sheba.health.gov.il), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03558269, which also lists 3 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jan 2021.

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