Autologous Umbilical Cord Blood Treatment of Neonate With CHD

Sheba Medical Center

Status and phase: Unknown; Phase 2; Phase 1

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

Treatments: Biological: Autlogous UCB infusion

Study type: Interventional

Funder types: Other

Identifiers

NCT03558269

3757-16-SMC

Details and patient eligibility

About

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

Full 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

Enrollment: 60 estimated patients

Sex: All

Ages: 3 to 14 days old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: Single Blind

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60 participants in 2 patient groups

Study group

Experimental group

Description:

This group will receive UCB after the first palliative surgery

Treatment:

Biological: Autlogous UCB infusion

Control group

No Intervention group

Description:

This group will not receive any treatment