Clinical Trials

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. 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?

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