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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 helped evaluate treatments and therapies for many diseases and conditions.

The NHLBI continues to support research on various treatments, including blood and marrow stem cell transplants. For example, NHLBI-sponsored research includes studies that:

  • Explore how well stem cell transplants using lower doses of chemotherapy work for patients with certain types of cancer.
  • Provide long-term evaluation and followup care for people who have had stem cell transplants.
  • Examine whether new stem cell transplant methods can help reduce complications of the procedure.
  • Explore the safety and effectiveness of using both peripheral and cord blood stem cells to treat people who have severe aplastic anemia or myelodysplastic syndrome. (Peripheral stem cells come from the bloodstream. Cord blood stem cells come from a baby's umbilical cord.)
  • Explore how well stem cell transplants using lower doses of chemotherapy work for children who have sickle cell disease.
  • Examine ways to improve the safety of stem cell transplants and how well they work.

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 stem cell transplants, talk with your doctor. You also can visit the following Web sites to learn more about clinical research and to search for clinical trials:

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

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Blood and Marrow Stem Cell Transplant Clinical Trials

Clinical trials are research studies that explore whether a medical strategy, treatment, or device is safe and effective for humans. To find clinical trials that are currently underway for Blood and Marrow Stem Cell Transplant, visit www.clinicaltrials.gov.


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The NHLBI updates Health Topics articles on a biennial cycle based on a thorough review of research findings and new literature. The articles also are updated as needed if important new research is published. The date on each Health Topics article reflects when the content was originally posted or last revised.