National Institutes of Health
National Heart, Lung, and
Bethesda, Maryland 20892
National Heart, Lung, and Blood Institute (NHLBI)
Participation in Clinical Research
on Sickle Cell Disease (SCD)
The only way to know for certain whether a new treatment will help or harm people with a particular condition is to conduct a clinical trial. This is a formal way of looking at treatments to determine whether and how well they work, for whom they are appropriate, and how many side effects they cause. The NHLBI has been the home for SCD clinical research since the late 1960s, and has conducted a number of landmark trials that demonstrated the effectiveness of:
• penicillin begun soon after birth to prevent life-threatening infections
• transcranial Doppler to detect children at high risk of stroke
• blood transfusions to prevent stroke
• hydroxyurea to reduce pain crises, acute chest syndrome, the need for blood transfusions, and deaths.
Clinical research is complex and expensive. It requires a dedicated and coordinated effort not only by the patients and their families who volunteer to be part of the studies, but also by a host of others including the following:
• Investigators who design and conduct studies in people are scientists, many of whom are physicians, as well as experts in other fields such as statistics. Many are on the faculty of universities or in community practices where they see patients with conditions under study. Investigators come up with most of the ideas for clinical studies, and their passion to advance the science and improve the lives of their patients is essential.
• Sponsors who support clinical trials can come from industry, private foundations, or the federal government. The federal National Institutes of Health (NIH), of which the NHLBI is a part, conducts and supports research with the goal of improving the health of individuals and the public as a whole. Taxpayer money is appropriated by the U.S. Congress for this purpose.
• Data and Safety Monitoring Boards are composed of scientists, ethicists, and patient representatives who oversee a study as it is being conducted to safeguard the health and well-being of participants. They are independent of the investigators and the sponsors of the study to ensure an unbiased focus on the welfare of the patients who participate.
The NHLBI, in partnership with other NIH components and the National Marfan Foundation, Institutes, recently unveiled a comprehensive Web site, Children in Clinical Studies, to give parents and health-care providers the information they need to understand clinical research in children and make informed decisions about participating in a study. It includes a video that may be useful for adults thinking about participating in a clinical trial as well.
A database of all open clinical trials, whether they are sponsored by the NIH or by some other entity, is available at http://clinicaltrials.gov. You can search the site for studies of any condition, including SCD, and find out whom to contact if you are interested in participating. The site also includes studies that are no longer enrolling participants so that you can find out about clinical trials that have recently been completed.
Examples of NIH-sponsored trials in SCD that are currently recruiting participants are provided to the left.