Clinical Trials

Clinical Research Educational Site

Be sure to visit Clinical Research Trials and You. This NIH site is intended to inform and educate the public and healthcare providers about clinical trials. You can review personal stories of trial participants as well as researchers who conduct trials. Educational materials specific to children, seniors, and Spanish-speaking audiences are featured.

Clinical research is at the heart of all medical advances, identifying new ways to prevent, detect, or treat disease.  If you have a bone marrow failure disease, you may want to consider taking part in a clinical trial, also called a research study. These studies offer good treatment options for some people with bone marrow failure diseases like aplastic anemia, MDS and PNH. People participate in clinical trials for a variety of reasons. Healthy volunteers may participate to help others and to contribute to moving science forward. Patients with an illness or disease participate to help others, but also to possibly receive the newest treatment and to have the additional care and attention from the clinical trial staff. Clinical trials offer hope for many people and an opportunity to help researchers find better treatments for others in the future.  This section provides you and your family information about clinical trials and gives you some things to consider when deciding whether a clinical trial is a good option for you.

Clinical Trial Basics

  • What is a clinical trial?
  • Why participate in a clinical trial?
  • Why are clinical trials important for rare diseases?
  • What are some risks of being in a clinical trial?
  • What happens in a clinical trial?
  • What is a clinical trial protocol?
  • Who can be part of a clinical trial?
  • Are children able to participate in clinical trials?

More Information

  • What are the different types of clinical trials?
  • What are the phases of clinical trials?
  • Who sponsors or provides funds for clinical trials?
  • How is my safety protected if I join a clinical trial?
  • What is an Institution Review Board (IRB)?
  • What is Informed Consent?
  • How do I decide whether or not to participate?
  • Questions to ask about a clinical trial

Finding a Clinical Trial

  • Government resources
  • AA&MDSIF