Today, we will be looking into the latest research and clinical trials focused on congenital heart disease.

The following list has been carefully curated by evaluating the ongoing phase 3 trials for congenital heart disease, targeting both adults and children in the United States. Please note that the dates provided are approximate and subject to change. This compilation primarily features studies that have released updates within the past 12 months.

This series aims to offer a glimpse into upcoming innovations in the field and how the outcomes of these studies could potentially influence clinical guidelines related to the topic.

Now, let’s go ahead and explore the list of Congenital Heart Disease Clinical Trials!

Quick View Table of Congenital Heart DiseaseClinical Trials
Study TitlePhaseEnrollmentStart DateLast Update Posted
Comparative Effectiveness of Gadopiclenol for Evaluation of Adult Congenital Heart Anatomy and HemodynamicsPHASE 31605/9/20245/9/2024
Fetal Cerebrovascular Autoregulation in Congenital Heart Disease and Association With Neonatal NeurobehaviorPHASE 3150 12/17/2021 5/8/2024
Nitric Oxide Effect on Brain and Kidney in Pediatric Patients Undergoing Cardiopulmonary BypassPHASE 3504/25/2022 8/14/2024
Acute Normovolemic Hemodilution in Complex Cardiac Surgery (ANH)PHASE 3632/28/2022 6/5/2024
Training in Exercise Activities and Motion for Growth (TEAM 4 Growth) RCT (T4G RCT)PHASE 31442/23/2021 7/3/2024
NO During CPB in Neonates to Reduce Risk of AKIPHASE 3401/10/2023 3/22/2024
Phase 3 Triiodothyronine Supplementation for Infants After Cardiopulmonary Bypass (TRICC-2)PHASE 322011/1/20144/9/2024

Phase 3 Clinical Trials:

Comparative Effectiveness of Gadopiclenol for Evaluation of Adult Congenital Heart Anatomy and Hemodynamics

Study Details | Source

  • Sponsor: University of California, San Diego
  • The goal of this clinical trial is to learn how well and at what doses gadopiclenol, a new intravenous (IV) contrast agent used for MRI, works to produce high-quality MRI images of the heart, in patients with a history of congenital heart disease, when compared to gadobenate dimeglumine, the IV contrast agent that is normally used at our institution for heart (cardiac) MRI. The main questions it aims to answer are:
    • Does using gadopiclenol result in similar or superior image quality, similar signal-to-noise ratio (SNR), and similar flow measurements with 4-dimensional (4D) flow cardiac MRI when compared to gadobenate dimeglumine?
    • At what dose(s) does gadopiclenol result in similar image quality (using the above metrics) for cardiac MRI when compared to gadobenate dimeglumine?
  • Researchers will compare cardiac MRI images obtained after administration of gadopiclenol to cardiac MRI images obtained after administration of gadobenate dimeglumine (called the standard of care treatment) to see if the images are of similar or superior quality.
  • Participants will:
    • Be randomized to receive either gadopiclenol at one of three different doses or gadobenate dimeglumine before their congenital heart cardiac MRI
    • Undergo their congenital heart cardiac MRI as they would during the course of normal clinical care.
  • Interventions:
    • Drug: Gadopiclenol
    • Drug: Gadobenate dimeglumine
  • Primary Outcomes Measures:
    • Image Quality
      • Images will be qualitatively scored by three attending radiologists for quality of contrast enhancement on a 5-point likert Scale
      • Image Quality will be assessed on a rolling basis during the study by all radiologists and will be completed within one month of the conclusion of enrollment.
    • Signal-to-Noise Ratio (SNR)
      • Signal-to-noise will be measured from the blood pool in the aorta and main pulmonary artery and compared against myocardial signal for contrast-to-background calculations in contrast-enhanced MRA and 4D Flow signal images. This ca be readily performed by placing a region of interest (ROI) within the vessel of interest and calculating the standard deviation of signal intensities relative to mean signal intensity.
      • SNR will be assessed on a rolling basis during the study by the study team and will be completed within one month of the conclusion of enrollment.
    • Standard Deviation of Volumetric Flow Measurements
      • We will measure standard deviation of volumetric flow measurements from the aorta and main pulmonary artery from ROIs that are typically used in our clinical routine to measure systemic and pulmonary blood flow. The standard error in these measurements will be compared between each of the four randomized conditions.
      • Standard deviation of flow measurements will be assessed on a rolling basis during the study by all attending radiologists and will be completed within one month after the conclusion of enrollment.

Fetal Cerebrovascular Autoregulation in Congenital Heart Disease and Association With Neonatal Neurobehavior

Study Details | Source

  • Sponsor: University of Utah
  • Determine 1) the impact of abnormal fetal cerebrovascular physiology with neurodevelopmental delay (ND) outcomes and 2) how this relationship is modified by patient and environmental factors such as chronic congenital heart disease (CCHD) lesion, maternal-fetal environment, and social determinants of heath (SDOH) in a diverse population using a multicenter design.
  • Pregnant women will be approached during one of their fetal cardiology clinic visits.
  • Interventions:
    • Procedure: Single Arm
    • Procedure: Neonatal Neurobehavioral Scale
  • Primary Outcomes Measures:
    • Pre-operative Neonatal Network Neurobehavioral Scale (NNNS) attention scores
      • The NeoNatal Neurobehavioral Scale (NNNS-II) examines the neurobehavioral organization, neurological reflexes, motor development - active and passive tone, and signs of stress and withdrawal of the at-risk and drug-exposed infant
      • Time frame - <=30 days of life
    • Baseline MCA-PI and change in MCA-PI with Maternal Hyperoxia
      • The fetal middle cerebral artery (MCA) pulsatility index (PI)
      • Time frame - <=30 days of life

Nitric Oxide Effect on Brain and Kidney in Pediatric Patients Undergoing Cardiopulmonary Bypass

Study Details | Source

  • Sponsor: Vanderbilt University Medical Center
  • The goals of this study are:
    • To evaluate the neuroprotective effect of nitric oxide by measuring glial fibrillary acid protein (GFAP) before and after surgery. GFAP will be analyzed via an enzyme-linked immunosorbent assay (ELISA) kit. Patients will also be monitored post-operatively for delirium in the intensive care unit (ICU).
    • To evaluate the renal protective effect of nitric oxide by measuring neutrophil gelatinase-associated lipocalin (NGAL) before and after surgery. NGAL will also be analyzed via an ELISA kit. Patient creatinine will be monitored post-operatively.
    • To evaluate effect of nitric oxide on other ICU outcomes (invasive mechanical ventilation, days to extubation, ICU and hospital length of stay, and blood product administration).
  • Interventions:
    • Drug: Nitric Oxide (NO) 20 part per million (ppm)
    • Other: Standard of care cardiopulmonary bypass
  • Primary Outcomes Measures:
    • Level of glial fibrillary acid protein (GFAP)
      • GFAP will be measured via blood sample
      • Time frame - baseline to peak rewarming temperature of blood (approximately 3 hours)
    • Level of neutrophil gelatinase-associated lipocalin (NGAL)
      • NGAL will be measured via blood sample
      • Time frame - baseline to 2 hours after CPB initiation (approximately 2 hours)

Acute Normovolemic Hemodilution in Complex Cardiac Surgery (ANH)

Study Details | Source

  • Sponsor: University of California, Los Angeles
  • Postoperative bleeding in cardiac surgery is a frequent complication, and cardiac surgery utilizes 15-20% of the national blood supply. Packed red blood cells (pRBCs) are associated with worse short and long term outcomes. For each unit transfused, there is an additive risk of mortality (death) and cardiac adverse events. Despite current guidelines and numerous approaches to bleeding reduction, >50% of the patients undergoing cardiac surgery receive transfusions. Acute normovolemic hemodilution (ANH), a blood conservation technique that removes whole blood from a patient immediately prior to surgery, could be a valuable method to reduce transfusion in complex cardiac surgery. At the University of California, Los Angeles (UCLA), ANH is routinely utilized in patients who refuse allogenic blood transfusions such as Jehovah's Witnesses. ANH has been shown to be safe with minimal risk to patients. ANH has been studied in simple cardiac surgery, such as coronary artery bypass grafting, however it has not been studied in complex cardiac surgery, such as aortic surgery and adult congenital heart disease. ANH has been demonstrated to reduce pRBC transfusion in lower risk cardiac surgery without any significant complications. Complex heart surgery utilizes more blood products. This study could identify the benefits of ANH in a higher risk surgical group.
  • Interventions:
    • Biological: Acute normovolemic hemodilution (ANH)
  • Primary Outcomes Measures:
    • Packed red blood cells (pRBC)
      • Total transfusion of pRBC units administered 24 hours post-operatively.
      • Time frame - 24 hours
    • Packed red blood cells (pRBC)
      • Total transfusion of pRBC units administered 48 hours post-operatively.
      • Time frame - 48 hours
    • Packed red blood cells (pRBC)
      • Total transfusion of pRBC units administered 72 hours post-operatively.
      • Time frame - 72 hours

Training in Exercise Activities and Motion for Growth (TEAM 4 Growth) RCT (T4G RCT)

Study Details | Source

  • Sponsor: Carelon Research
  • This is a Phase III randomized controlled trial of a passive ROM exercise program that will be performed in infants with HLHS and other single right ventricle anomalies following the Norwood procedure at PHN and Auxiliary Centers.
  • Interventions:
    • Other: passive range of motion exercise therapy
  • Primary Outcomes Measures:
    • weight-for-age z-score
      • weight-for-age z-score
      • Time frame - 4 months of age or superior cavopulmonary connection (SCPC) evaluation clinic visit, whichever comes first

NO During CPB in Neonates to Reduce Risk of AKI

Study Details | Source

  • Sponsor: Children's Hospital Medical Center, Cincinnati
  • Acute kidney injury (AKI) following cardiac surgery for congenital heart defects (CHD) in children affects up to 60% of high risk-patients and is a major cause of both short- and long-term morbidity and mortality. Despite effort, to date, no successful therapeutic agent has gained widespread success in preventing this postoperative decline in renal function. Nitric oxide is an intricate regulator of acute inflammation and coagulation and is a potent vasodilator. The investigators hypothesize that nitric oxide, administered during cardiopulmonary bypass (CPB), may reduce the incidence of AKI.
  • Interventions:
    • Drug: Nitric Oxide
    • Drug: Oxygen
  • Primary Outcomes Measures:
    • AKI
      • Incidence of AKI in the first 72 hours postoperative as defined by the Kidney Disease Improving Global Outcomes (KDIGO) diagnostic classification
      • Time frame - 72 hours

Phase 3 Triiodothyronine Supplementation for Infants After Cardiopulmonary Bypass (TRICC-2)

Study Details | Source

  • Sponsor: Seattle Children's Hospital
  • This is a study to determine the safety and efficacy of liothyronine sodium/triiodothyronine (Triostat), a synthetic thyroid hormone, when given to infants with congenital heart disease during cardiopulmonary bypass surgery. Funding Source - FDA OOPD.
  • Interventions:
    • Drug: Triostat
    • Drug: Placebo
  • Primary Outcomes Measures:
    • Time To Extubation
      • time on mechanical ventilation after aortic cross clamp removal
      • Time frame - 30 Days

Potential Guidelines That May Be Affected Include:

There you have it - a list of phase 3 clinical trials for congenital heart disease as of February 2025. Stay tuned, for our next Guidelines+ Trials Rundown. Sign up for alerts and stay informed on the latest published guidelines and articles.

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