Ongoing Clinical Trials for Congenital Heart Disease
Currently, there are 3 ongoing clinical trials exploring new treatments and protective strategies for children and young people with congenital heart disease. These studies are testing medications to prevent brain injury during surgery, improve heart protection, and enhance exercise capacity in patients who have undergone specific heart procedures.
Clinical trial locations
- Germany
- Netherlands
- Norway
Study on Allopurinol Sodium for Brain Injury Prevention in Newborns with Critical Congenital Heart Disease Undergoing Heart Surgery
This trial is investigating whether allopurinol sodium can protect the brain in newborns with critical congenital heart disease who need heart surgery within their first four weeks of life. The surgery requires the use of a heart-lung machine, called cardiopulmonary bypass, which can sometimes lead to reduced blood flow or oxygen to the brain.
Who can participate: Newborn babies, both male and female, who have been diagnosed with critical congenital heart disease either before or after birth. The heart condition must be serious enough to require surgery with cardiopulmonary bypass within the first month of life.
Who cannot participate: Babies without critical congenital heart disease, those who do not need surgery with a heart-lung machine, newborns not at risk of brain injury related to their heart condition, and babies outside the specified age range for the study.
What the trial involves: The study will test allopurinol sodium, given through a vein, compared to a placebo to see if it can reduce brain injury. Babies will receive the medication shortly after birth and around the time of surgery. Researchers will monitor brain function during and after surgery using various methods including MRI scans, electroencephalogram, and near-infrared spectroscopy. The study will also follow the children as they grow, assessing their movement and cognitive development at 3 months and 24 months of age.
Investigational drug: Allopurinol sodium works by blocking an enzyme called xanthine oxidase, which reduces the production of uric acid and oxidative stress that can damage brain tissue. The medication is administered as a solution through a vein.
Study Comparing Custodiol-N and Custodiol for Heart Surgery in Children with Congenital Heart Defects
This study is comparing two different solutions used to protect the heart during surgery in children with structural heart problems present from birth. Both Custodiol-N and Custodiol are used to safely stop the heart during surgery and preserve its function while repairs are being made.
Who can participate: Children from birth to less than 18 years old who need surgery for congenital heart problems. The surgery must involve cardiopulmonary bypass and cardioplegia, which is a method to protect the heart during the operation. Written consent from the patient or their legal guardians is required, and guardians must be able to understand what the study involves.
Who cannot participate: Patients with other serious health conditions that could interfere with the study, those who recently had surgery that might affect results, patients currently in another clinical trial, those unable to follow study procedures, patients with allergies to study medications, pregnant or breastfeeding patients, those with a history of drug or alcohol abuse, certain infections, patients on interfering medications, or those with certain unrelated heart conditions.
What the trial involves: During heart surgery, one of the two protective solutions will be used. The study will monitor safety by observing any unexpected medical problems for up to 30 days after surgery. Heart protection will be assessed by measuring specific enzymes in the blood, like CK-MB, which indicate heart muscle damage. Researchers will also evaluate heart function, hospital stay duration, vital signs, and the need for medications that support heart function.
Investigational drugs: Both Custodiol-N and Custodiol are cardioplegic solutions given intravenously during surgery. They work by reducing the heart’s metabolic demands and preserving cellular energy, which helps maintain heart tissue health during periods when blood flow is reduced. These solutions help to safely stop the heart and protect it during surgical procedures.
Study on Theophylline for Improving Exercise Capacity in Adolescents with Univentricular Heart Disease and Fontan Circulation
This trial is testing whether theophylline can help improve exercise capacity in adolescents and young adults who have a specific type of heart condition where only one ventricle is functional. These patients have previously undergone a Fontan procedure, a surgical technique that directs blood flow from the veins directly to the lung arteries, bypassing the heart.
Who can participate: Young people between 16 to 25 years old with univentricular congenital heart disease who have had Fontan-type surgery. Participants must be able to perform all necessary tests, particularly a cardiopulmonary exercise test on a stationary bike. They must have recent liver imaging results showing no more than mild liver disease, have a BMI between 18.5 and 34.9 kg/m², be able to safely take theophylline, and provide signed informed consent (with parental consent if under 18). Female participants must use contraception and have a negative pregnancy test.
Who cannot participate: Patients without the specific heart condition and Fontan surgery, those outside the 16-25 age range, patients who cannot safely take theophylline, and those unable to undergo remote dosage adjustment and heart rhythm monitoring.
What the trial involves: Participants will take Theo-Dur 200mg prolonged-release tablets orally for 12 weeks. The dosage will be adjusted remotely based on individual needs, and heart rhythm will be monitored throughout. The study will assess changes in exercise capacity, oxygen uptake during physical activity, quality of life through questionnaires, heart function using echocardiography, breathing capacity, and sleep patterns through a home-based sleep study. Regular monitoring will track any side effects and changes in health status.
Investigational drug: Theophylline is a bronchodilator that relaxes and opens airways in the lungs, making breathing easier. It also has effects on the heart and blood vessels, potentially improving blood flow and oxygen delivery during physical activity by increasing the strength of heart muscle contractions.
Summary
These three clinical trials represent focused efforts to address different challenges faced by patients with congenital heart disease across various age groups. The research is geographically distributed across three European countries: the Netherlands, Germany, and Norway, with each country hosting one specialized study.
The trials target distinct patient populations and clinical needs. The Dutch study focuses on the most vulnerable patients, newborns requiring early surgical intervention, investigating brain injury prevention. The German trial addresses a critical aspect of pediatric cardiac surgery by comparing heart protection solutions used during operations in children from birth to age 18. The Norwegian study takes a different approach, looking at quality of life and physical capacity in adolescents and young adults who have already undergone corrective surgery.
Two of the three studies involve medications during or around the time of surgery (allopurinol sodium and cardioplegic solutions), while the third explores longer-term medical treatment to improve daily functioning. This range of approaches reflects the complex and lifelong nature of managing congenital heart disease, from initial surgical interventions through childhood and into young adulthood.
