Autologous Umbilical Cord Blood Treatment of Neonate With CHD
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03558269|
Recruitment Status : Not yet recruiting
First Posted : June 15, 2018
Last Update Posted : June 15, 2018
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|
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:
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 (Clinical Trial)|
|Estimated Enrollment :||60 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Single (Outcomes Assessor)|
|Masking Description:||The outcome assessor will not be exposed to the participant group|
|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|
|Estimated Study Start Date :||June 15, 2018|
|Estimated Primary Completion Date :||May 1, 2020|
|Estimated Study Completion Date :||April 1, 2022|
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
- 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.
- Neuro-development at one month [ Time Frame: One month ]General movements assessment (ref 2)
- Neuro-development at six month [ Time Frame: 6 months ]Gross Motor Function Measure 66 (ref 3)
- Neuro-development at 12 month [ Time Frame: 12months ]Gross Motor Function Measure 66 (ref 3)
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
|Contact: Omer Bar-Yosef, M.D.-Ph.D.||email@example.com|
|Contact: Amir Vardi, M.D.||firstname.lastname@example.org|
|Sheba Medical Center||Not yet recruiting|
|Ramat Gan, Israel, 5621000|
|Contact: Omer Bar-Yosef, M.D._Ph.D. +972-3-5302687 email@example.com|
|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.|