Join Clinical Trials for Pediatric Cardiovascular Disorders
Unfortunately, children and adolescents are not exempt from experiencing heart-related problems. The two major types of heart problems in pediatric populations are congenital and acquired.
Congenital heart defects are birth defects of the heart, which occur during gestation when the baby’s heart is developing. Such birth defects are relatively common, with six to eight of every 1,000 children being born with a congenital heart defect. Fortunately, many of these defects are mild and do not require treatment. Others are more severe and necessitate treatment in order to save the child’s life.
The second type of heart problem experienced by children – acquired heart disease – is one that appears after birth and is usually caused by damage to the heart from a certain disease, or infection with a bacteria or virus. Among acquired heart diseases, the most common is caused by rheumatic fever.
The National Heart, Lung, and Blood Institute (NHLBI) has acknowledged that heart disease in infants, children, and adolescents is a significant and under-appreciated problem in public health today. And, in early 2001, the NHLBI convened a task force to identify top research priorities for addressing pediatric cardiovascular conditions.
As a result, the task force recommended that research focus on three far-reaching areas: 1) basic research on the development of the cardiovascular system and the ultimate causes of cardiovascular disease; 2) research that works to improve the clinical outcomes of pediatric cardiovascular disease patients; and 3) research that evaluates strategies to reduce cardiovascular disease in adults by modifying risk factors that are present in childhood.
To accomplish these research objectives, studies are currently underway in pediatric patients to evaluate new and improved technologies to visualize the heart, advance methods of repairing birth defects of the heart (both during gestation and after delivery), better manage and treat acquired heart disease, and improve methods to identify and treat heart disease risk factors in children.
What Will Pediatric Cardiovascular Disease Trials Be Like?
The types of tests and assessments used in pediatric cardiovascular disease clinical trials will ultimately depend on the specific nature of the study and the particular cardiovascular condition being investigated. Provided below is a list of frequent procedures and tests used to evaluate the heart, lungs, and blood vessels, many of which may be incorporated for use in clinical trials:
- Physical exam
- Detailed family history cardiovascular disease
- Genetic testing
- Blood tests to evaluate the effectiveness or chemical properties of a medication, if you are participating in a clinical trial that is investigating the use of a new drug.
- Blood tests to evaluate the type and number of specific blood cells present in the blood.
- Echocardiography (“echo”): a painless procedure that uses ultrasound to create moving pictures of your heart, which allow doctors to see its size, shape, and how well it is working.
- Electrocardiogram (EKG, or ECG): a straightforward and painless procedure that records the electrical activity of the heart.
- Chest x-ray
- Computed tomography (CT scan, or “CAT scan”) or magnetic resonance imaging (MRI) scans: these are non-invasive imaging procedures, similar to an x-ray, that allow doctors to take detailed pictures of your heart.
- Stress test: a test performed while you exercise (usually by walking or running on a treadmill, or pedaling a stationary bicycle), which allows doctors to evaluate how your heart works during episodes of physical stress.
Typical Pediatric Cardiovascular Disease Clinical Trial Protocol:
Specific examples of clinical trials for pediatric cardiovascular disease might include the following:
- A study in which tissue samples are collected from infants undergoing surgery to correct heart birth defects and then analyzed to determine how a child’s heart develops during the first year of life. The goal of a study such as this would be to identify treatments that are geared more specifically toward pediatric heart patients rather than using modified versions of treatments developed and intended for adults.
- A randomized clinical trial in newborns with congenital heart defects to determine if those who receive pre-operative breast milk feedings prior to surgery show improved intestinal function post-surgery when compared to newborn infants who received nothing by mouth prior to surgery.
- An observational study in which standard cardiac MRI is compared to an enhanced form of cardiac MRI to determine which is more effective at visualizing complex congenital heart diseases in children.
- A randomized clinical trial in pediatric endocarditis patients to determine if standard treatment plus a new drug is more effective at preventing heart-muscle damage than standard treatment plus placebo. A trial such as this would evaluate the efficacy of the new drug in preventing heart muscle damage.
- A study to determine if a dietary intervention (i.e., specific Mediterranean-style diet) plus supplementation with prescription-strength fish oil preparation is effective at lowering cholesterol among children with high cholesterol who are deemed to be at risk for early-onset coronary heart disease.
A brief word about randomized trials and placebos:
Many clinical trials involve the comparison of an investigational treatment to a “standard” treatment. Some studies determine which therapy a patient receives through a process known as randomization, in which patients are randomly assigned to receive either the investigational treatment or the standard treatment.
On occasion, a trial will investigate the use of a standard treatment plus a new drug compared to standard treatment plus a placebo (such as the fourth clinical trial example provided above). Placebos are inactive or “sham” treatments that are identical in appearance to the active treatment but have no therapeutic value.
Placebos are necessary to help determine if adverse effects that occur during the clinical trial are the result of the investigational treatment or due to some other factor. They also allow researchers to measure the effects of the active treatment and observe what would have happened without it.
In rare instances where no standard therapy exists, or when a new drug or therapy is being investigated, the investigational treatment might be compared to a placebo alone. In these types of trials, those patients who are randomized to the placebo group do not receive an active treatment.
It is important to know that placebo-only trials are only conducted when scientifically necessary and when patients have been adequately informed that they may end up receiving the placebo rather than the active treatment. It is very important to note, however, that no one should ever participate in such a placebo trial when there is a widely available and highly effective standard treatment already in existence for their particular disease or condition.
Trial Eligibility and Medical Information Needed:
The type of clinical trial you may be eligible for often depends on many factors. Therefore, it is important to know many details pertaining to your specific diagnosis when searching for clinical trials. Examples of the details you may want to have on hand include:
- Your specific diagnosis of acquired or congenital heart disease (e.g., endocarditis, Tetralogy of Fallot [a common cardiac birth defect])
- Your prior history of treatment for heart disease (including any surgeries, procedures, and medications)
- Your current medications (including aspirin), vitamins, and dietary supplements
Suggested Search Terms:
Once you are ready to begin your search for pediatric cardiovascular disease clinical trials, the following keywords may be useful when combined with the phrase “pediatric heart”: “transplant,” “failure,” “surgery,” “disease,” “murmur,” and “defect.”
Additionally, the following search phrases may also be helpful: “congenital heart disease,” “congenital heart defect,” “congenital heart defect surgery,” “congenital heart defect treatment,” “family history congenital heart defect,” “congenital heart defect outcomes,” and “congenital heart defect quality of life.”
Current Search Term:
“Pediatric Cardiovascular Disorders”
General Clinical Trial Information