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Many patients who receive bone marrow or cord blood transplants may be asked to join a clinical trial, also known as a research study. Clinical trials have led to greatly improved survival for transplant today. Clinical trials will continue to help doctors make important discoveries for future patients.

If you're asked to join a clinical trial, your doctors should tell you about: 

  • What they are trying to learn
  • What the treatment and tests are
  • Your rights as a patient

Ask your doctor to explain what the trial will mean for your treatment. If you choose to participate in a clinical trial, your doctor will give you a consent form to sign. The consent form will tell you more about the purpose of the study, the risks and benefits, other options available to you, and your rights as a participant in the study.

What is a clinical trial? 

A clinical trial is a way to gather information about how well a particular treatment, test, or approach to a disease works (efficacy) and what the side effects may be (safety). Some trials are done to learn how well a new medicine works. Others are to see if one way of treatment is better than another. Yet others look at a new test to see if the information from the test helps doctors better care for patients.

A clinical trial does not necessarily mean that the medicine or treatment is unproven or never before tested, although in some cases that may be true. 

Clinical trials may:

  • Look at a new use for a well-understood medicine given for other purposes
  • Test two different but well-known techniques for transplant to see if one technique is better than the other
  • Study the side effects and safety of an entirely new treatment for cancer

In each case, your doctor will explain the risks and benefits of joining the trial during the consent process. You will never be part of a clinical trial without your clear consent.

Many transplant patients join clinical trials that test drugs used before and after transplant. Others join trials that study different types of transplant for a given circumstance.

Your rights and safety

Clinical trials must follow strict rules to keep patients safe. In the U.S., clinical trials are regulated by the Office of Human Research Protections (OHRP). They are also sometimes regulated by the Food and Drug Administration (FDA). Each clinical trial is watched closely by an institutional review board (IRB). The IRB reviews the trial to be sure that it is ethical and that the rights of people who join are protected.

Throughout the trial, researchers report to the OHRP, FDA, and IRB. The trial can be shut down at any time to protect the people in it.

They will stop the trial if any unexpected risks, such as dangerous unknown side effects, are found.

The FDA and the OHRP require that patients give informed consent before joining a clinical trial. Informed consent means that the patient has been told about and understands the possible risks and benefits before agreeing to be in the trial.

Joining clinical trials is not required. If you are uncomfortable joining a trial, tell your doctor. Your doctor and health care team will take good care of you whether or not you decide to participate in a trial.

Finding a trial

Your transplant doctor will let you know if you are eligible to participate in a clinical trial at your transplant center. You can also find clinical trials by going to the U.S. National Institutes of Health Clinical Trials website at You should discuss any clinical trials with your health care team before moving forward with a study. Remember, participation in clinical trials will not affect the quality of care you will receive.

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