Autologous Umbilical Cord Blood Treatment of Neonate With CHD

NCT03558269 | Unknown Phase 1

• 1 other identifier: 3757-16-SMC
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 1

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

Conditions

Congenital Heart Disease; Brain Injuries; Low Cardiac Output Syndrome; Neonatal Disorder

Keywords

Autologous Umbilical cord blood infusion; Congenital Heart Disease; Brain Injuries; Low Cardiac Output Syndrome; Neonatal Disorder

Outcome Measures

Primary Outcomes (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.

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

Secondary Outcomes (3)

• Neuro-development at one month

  One month

• Neuro-development at six month

  6 months

• Neuro-development at 12 month

  12months

Study Arms (2)

Study group

EXPERIMENTAL

This group will receive UCB after the first palliative surgery

Biological: Autlogous UCB infusion

Control group

NO INTERVENTION

This group will not receive any treatment

Interventions

Autlogous UCB infusion BIOLOGICAL

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 group

Eligibility Criteria

• Age: 3 Days - 14 Days
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Sheba Medical Center: lead

Study Sites (1)

Sheba Medical Center

Ramat Gan, 5621000, Israel

RECRUITING

Related Publications (3)

• 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.

  PMID: 29468662 BACKGROUND

• 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.

  PMID: 23802141 BACKGROUND

• 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.

  PMID: 23771484 RESULT

MeSH Terms

Conditions

Heart Defects, Congenital; Brain Injuries; Cardiac Output, Low; Infant, Newborn, Diseases

Condition Hierarchy (Ancestors)

Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Craniocerebral Trauma; Trauma, Nervous System; Wounds and Injuries; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Central Study Contacts

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

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Masking Details: The outcome assessor will not be exposed to the participant group
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER GOV
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: May 12, 2018
• First Posted: June 15, 2018
• Study Start: February 1, 2019
• Primary Completion: May 1, 2021
• Study Completion: April 1, 2023
• Last Updated: January 20, 2021

Record last verified: 2021-01

Locations

IL (1)