Clinical Trials

The National Heart, Lung, and Blood Institute (NHLBI) is strongly committed to supporting research aimed at preventing and treating heart, lung, and blood diseases and conditions and sleep disorders.

NHLBI-supported research has led to many advances in medical knowledge and care. For example, this research has uncovered some of the causes of blood diseases, as well as ways to prevent and treat these diseases.

Many questions remain about blood diseases and conditions, including sickle cell anemia. The NHLBI supports an extensive research program to learn more about sickle cell anemia and find better ways to treat the disease and prevent complications. For example, NHLBI-supported research on sickle cell anemia includes studies that explore:

• The safety and effectiveness of blood and marrow stem cell transplants in children who have sickle cell disease
• How genetics play a role in sickle cell pain crises
• The best ways to control pain and treat other complications of sickle cell anemia
• Approaches that could be used to lower the risk of stroke in children
• Whether changes in blood flow cause pain crises in people who have sickle cell anemia
• How certain medicines and other therapies can help treat sickle cell anemia and improve quality of life for people who have the disease

Much of this research depends on the willingness of volunteers to take part in clinical trials. Clinical trials test new ways to prevent, diagnose, or treat various diseases and conditions.

For example, new treatments for a disease or condition (such as medicines, medical devices, surgeries, or procedures) are tested in volunteers who have the illness. Testing shows whether a treatment is safe and effective in humans before it is made available for widespread use.

By taking part in a clinical trial, you can gain access to new treatments before they're widely available. You also will have the support of a team of health care providers, who will likely monitor your health closely. Even if you don't directly benefit from the results of a clinical trial, the information gathered can help others and add to scientific knowledge.

If you volunteer for a clinical trial, the research will be explained to you in detail. You'll learn about treatments and tests you may receive, and the benefits and risks they may pose. You'll also be given a chance to ask questions about the research. This process is called informed consent.

If you agree to take part in the trial, you'll be asked to sign an informed consent form. This form is not a contract. You have the right to withdraw from a study at any time, for any reason. Also, you have the right to learn about new risks or findings that emerge during the trial.

For more information about clinical trials related to sickle cell anemia, talk with your doctor. You also can visit the following Web sites to learn more about clinical research and to search for clinical trials:

• http://clinicalresearch.nih.gov
• www.clinicaltrials.gov
• www.nhlbi.nih.gov/studies/index.htm
• www.researchmatch.org

For more information about clinical trials for children, visit the NHLBI's Children and Clinical Studies Web page.

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