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Clinical Trials in Depth

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Clinical trials, also called research studies, may offer good treatment options for some people with bone marrow failure diseases like aplastic anemia, MDS and PNH. This section helps you and your family understand clinical trials and gives you some things to consider when deciding whether a clinical trial is a good option for you.

A clinical trial is a research study that involves volunteers who participate in carefully conducted investigations aimed at discovering better ways to treat, prevent, diagnose, and understand human disease. Many clinical research studies test new treatments and therapies. Or they may compare existing treatments to new treatment options.

Doctors and other health professionals run the clinical trials, which are conducted in phases. Clinical research is done according to a detailed plan known as a protocol. The protocol is carefully designed to safeguard the participants’ health and answer specific research questions. A protocol describes the following:

  • Who is eligible to participate in the trial
  • Details about tests, procedures, medications and dosages
  • The length of the study and what information will be gathered

A clinical study is usually led by a principal investigator (PI), who is often a doctor. Members of the research team regularly monitor the participants’ health to determine the study’s safety and effectiveness.

When you volunteer to be in a study, you might be trying a new treatment or drug that may or may not be better than one that is already in use. The doctors and researchers don't know ahead of time how the study will turn out. That is why they are doing the research. Each clinical trial in the United States must be approved and monitored by an Institutional Review Board (IRB) to ensure that the risks are minimal and are worth any potential benefits. An IRB is an independent committee that consists of physicians, statisticians and members of the community who ensure that clinical trials are ethical and that the rights of participants are protected. Federal regulation requires that all U.S. institutions conducting or supporting biomedical research with humans work with an IRB to initially approve and periodically review their research.

Being part of a research study can mean playing a more active role in your health care. It can also:

  • Help you gain access to new treatments before they are widely available to the public
  • Offer expert care at leading health care facilities during the trial
  • Help others by contributing to medical research

Being in a clinical trial is not for everyone. The information in this section will help you learn more about research studies before you decide if this option is right for you.

Treatment options are often limited for rare diseases. These studies help doctors find new and better ways to find, prevent, diagnose, treat and control rare health problems.

Research studies help doctors determine the proper dose and dose schedule to follow when giving a treatment. This improves a treatment's success rate. Studies are also a great way to find better treatments with fewer side effects.

Past research studies have led to the approval of many effective treatments for bone marrow failure diseases. These include:

  • decitabine (Dacogen)
  • deferasirox (Exjade)
  • lenalidomide (Revlimid)
  • eculizumab (Soliris)
  • azacitidine (Vidaza)
  • eltrombopag (Promacta)

Research has also helped develop new procedures, such as bone marrow transplants. Clinical trials explore and measure ways to improve the comfort and quality of life for patients with a chronic illness through supportive care.

Before you join a clinical research study, you should know that:

  • The treatment being tested may not work as well as the standard treatment. The term "standard treatment" means a treatment that has been tested and found helpful and that is commonly used.
  • There is a chance that the side effects of the treatment being tested may be more serious than those of the standard treatment. The risks depend on the type of treatment you receive. The study researchers will explain all known risks to you throughout the study. This is known as informed consent.
  • You may have more doctor visits and tests than if you were not in the study.

These studies test whether new products are safe and effective treatments. A research team may include doctors, social workers and other health care professionals. They will check your health at the start of the study and give you instructions for being in the trial. They will check you carefully during the entire study and may stay in touch with you when the study is done.

Some studies require that neither you nor your doctor know whether you are getting the new study treatment, the standard treatment, or a placebo. Clinical trials may compare a new medical approach to a standard one that is already available or to a placebo that contains no active ingredients or to no intervention. Some clinical trials compare interventions that are already available to each other.

Volunteers in a clinical trial are free to withdraw from the study completely or to refuse particular treatments or tests at any time. In some cases, this might make them ineligible to continue the study.

Before a research study begins, the lead researcher creates a study plan called a "clinical trial protocol." The plan lists exactly what will happen during the study and why.  Most protocols include:

  • The specific research questions the study hopes to answer
  • Who may take part in the study (eligibility)
  • How many participants are needed
  • How long the study will last
  • The results the doctors hope to see in response to the treatment
  • What information will be gathered about the participants

Protocols also address the medicine and treatment and include:

  • Details about tests, procedures, medications and dosages
  • How the medicine or treatment will be given
  • Any lab tests that will be done
  • Known side effects of the treatment
  • Possible benefits and risks of the treatment

The protocol must be reviewed and approved by a special review committee, called an Institutional Review Board, or IRB for short. Doctors, scientists and people from the community make up an IRB. These IRB members review the study plan to make sure that the rights of volunteers in the study are protected. They also check to see that the risks of being in the study are reasonable in comparison to the expected good outcomes.

Some research studies look for people with certain health problems to be studied. Other studies need only healthy participants. Research procedures with healthy volunteers are designed to develop new knowledge, not to provide direct benefit to study participants. Every clinical trial has a set of guidelines about who can be part of that study. These guidelines are also called eligibility requirements. The factors that allow someone to participate in a clinical study are called inclusion criteria. Those factors that disqualify someone from participating are called exclusion criteria. These are based on things such as age, gender, the type and stage of a disease, previous treatment history and other medical conditions. In other words, you must qualify for the study before you can participate in the clinical trial.

Before you are allowed to be in a study, you'll be asked to fill out a form to see if you meet the guidelines. The guidelines help the researchers make sure people with the health condition being studied are in the trial. These guidelines also protect people who have certain health problems that may make it dangerous for them to be part of the study.

Guidelines are often set so that people in the study are as much alike as possible. This way, the doctors and researchers can be sure that the results from the study are due to the treatment.

You may be asked if you:

  • Have a certain type or stage of illness
  • Have had a certain kind of therapy in the past
  • Are in a certain age group

Can children be part of a clinical trial?

According to the National Heart, Lung and Blood Institute, many medicines given to children have not been studied and approved by the Food and Drug Administration (FDA) for use in children. This is known as "off-label" use. Most of the time, this works well, but because children are not merely little adults, there is a chance that the dose used could be ineffective or even harmful. Without research studies on children with aplastic anemia, the healthcare team may not have the level of information needed to understand the way medicines and other treatments work in a child’s body over time, or how those therapies impact a child’s growth and development. The only way to examine the scientific value of a new therapy for children, is by conducting a pediatric clinical trial. Should you enroll your child in a clinical trial? Learn more about clinical trials and children, then speak with your child’s doctor.

For more information, see our Clinical Trials and Children page.

  • Treatment trials test new treatment options. For example, treatment trials for bone marrow failure may study a new medicine. Or, the study may look to find a new use for one or more drugs that are already being used. Treatment trials make sure a treatment is safe and effective before it is approved for wider use.
  • Diagnostic trials test and evaluate new ways to diagnose a disease.
  • Screening trials test the best way to detect a disease or health problem.
  • Quality-of-life trials are also called supportive care trials. They study ways to improve the comfort and life quality of people with a long-term illness.
  • Prevention trials look for better ways to prevent disease in people who have never had the disease. They may also explore ways to stop a disease from coming back. These trials may include using medicines, vaccines, or vitamins, or making changes in lifestyle.

Clinical trials are carried out in phases, each designed to find out certain information. These phases are defined by the U.S. Food and Drug Administration (FDA). A new drug treatment must first be tested in a lab or in animals before it is tested in people. Then, before a drug can become part of standard treatment, it must first go through 3 or 4 clinical trial phases. Each phase has a different purpose and helps scientists answer different questions.

  • Phase I – Is the drug safe? These studies are often conducted with healthy volunteers. Safety is important in these studies. Phase 1 trials typically have between 20 and 80 participants.
  • Phase II – Does the drug treatment work? Does the new drug have a positive effect on people with a certain disease or condition? It is usually in Phase II trials when the researchers find out if the drug is not working as planned or has toxic effects. Phase II trials typically have between 100 and 300 participants.
  • Phase III – Is the new drug treatment better than the standard treatment? These studies look at the safety and effectiveness of a new treatment compared to the standard treatment for a disease or condition. It is after a Phase III trial that most drugs are able to get approved by the FDA to treat a specific disease. Phase III trials often have between 1,000 and 3,000 participants.
  • Phase IV – What is the long-term impact of the new drug treatment? How safe and effective is the new drug or treatment once it has been approved to enter the market by the FDA? These trials can have thousands of participants.

You do not have to take part in all the phases. The early phases of a research study make sure the drug or treatment is safe. The later phases of the study show if it works better than the standard treatment used.

They can be sponsored or funded by:

  • Medical or health institutions
  • Foundations
  • Pharmaceutical companies
  • Voluntary groups
  • Federal agencies, such as the National Institutes of Health (NIH)

In the U.S., there are strict guidelines to make sure that people who agree to be in a clinical trial are treated as safely as possible. These guidelines apply to all medical research involving human participants. The guidelines require the following:

  • An institutional review board, also called an IRB, must review and approve every clinical trial plan. Every group conducting clinical trials must work with an IRB. This review helps make sure that the risks of the study are small and are worth the possible benefits.
  • Informed consent must be given by a patient before being in the study. Informed consent can be confusing. It means that you agree (consent) to be in the study and you understand all the facts about being in the study, including any possible harm it may cause you.

An institutional review board, also called an IRB, is a committee that makes sure a clinical trial is safe for patients in the study. Each medical center, hospital, or research facility doing clinical trials must have an active IRB.

Each IRB includes a diverse group of doctors, faculty, staff and students at a specific institution. An IRB must also have at least one person from the community who is not connected to the institution. This board goes over each study plan and makes sure that the highest standards are met to protect the rights of the study participants.

Informed consent is a process in which researchers provide potential and enrolled participants with information about a clinical study. This information helps people decide whether they want to enroll, or continue to participate, in the study. The informed consent process is intended to protect participants and should provide enough information for a person to understand the risks, potential benefits and alternatives to the study. Informed consent for a clinical trial is much more than just reading and signing a piece of paper. It involves two essential parts:

  • An informed consent document
  • A process that includes many conversations with the medical team doing the clinical trial

The informed consent document reviews all the information about the clinical trial. It includes:

  • The purposes of the research study
  • How long the study will last
  • The tests and treatments you will get
  • The possible risks
  • The potential benefits of being in the study
  • Your rights as a participant. For example, you have the right to leave the study at any time

If there is even a small risk of your getting injured by being part of the research study, the informed consent document must also include:

  • Who to contact if you are injured by being in the study
  • A list of health treatments to treat an injury and how to get more information about these treatments
  • Whether or not you are able to get any money (compensation) if you are injured and how this money can be obtained

Reading the informed consent document begins the informed consent process. This process includes a series of conversations between you and the research team. During these conversations the study researchers may also explain:

  • Other treatments, if any, that you might use instead of the treatment being used in the study (This allows you to know all your treatment options before joining the research study.)
  • How the records identifying you will be kept private
  • Who can answer your questions about the research

Informed consent continues after you are in a clinical trial. It includes ongoing talks with the doctors and nurses doing the clinical trial. These conversations will help you make good decisions about whether to stay in the trial. Before, during and even after the trial, you can ask questions and raise concerns. So informed consent is an ongoing and interactive process. It is not just a one-time information session or a form you read and sign.

You will want to look at all the pros and cons of any research study before choosing to be part of the study. Take the time to talk about the risks and benefits with others. Talk to your doctors and family to decide if being in the study is right for you.

Kristin Dodd, RN, a member of the AAMDSIF Nursing Network from the Cleveland Clinic Foundation, shared some reasons for patients to consider becoming involved in clinical trials. First, patients in a clinical trial are likely to be monitored more closely.  There is an increase in lab draws and tests such as EKGs, CT scans or MRIs as well as an increased number of clinic visits with the doctor and nurse, compared to the standard of care visits when not involved in a clinical trial. Because of this more intense schedule and level of monitoring, clinical trial participants often indicate they feel more secure. 

During a clinical trial, participants receive the study drug at no cost to them.  All of the study-related services are often covered in full by the sponsor conducting the clinical trial.  Participants may even be able to recoup out-of-pocket expenses, such as parking, travel and food or hotel costs.  Some patients experience a sense of relief when these burdens of cost are lifted from their shoulders. Contrary to what many think, standard-of-care aspects of a clinical trial are still covered by a patient’s health insurance.

When a patient participates in a clinical trial, other available FDA-approved therapies are still available later on, in case a trial medication doesn’t work. Some trials may exclude patients previously treated with currently available drugs. In this case, if a patient participates in a clinical trial that does prove effective, he or she can then move on to another clinical trial or pursue FDA-approved treatment. 

In conclusion, having the opportunity to participate in clinical trials enables you to make informed decisions about your health. You may get access to experimental drugs that may not become widely available for years, and if the trial drug does not work, there are other trials out there.  If clinical trials are not a good fit for you, then you can turn to FDA-approved therapies.  To find out where to look for clinical trials in your area, contact our patient educator, ask your doctor, contact any major medical center in your area, or visit

Take this information with you when you talk with the research team. Use the questions below as you decide.

Below is a list of questions you may want to ask if you are considering a clinical trial. Print out the list and take it with you when you speak with the clinical trial coordinator.

About the study:

  • What is the purpose and phase of this study?
  • Who is going to be in this study?
  • Why do researchers believe the new treatment being tested may be effective? Has it been tested before, and if so, what were the findings?
  • Who is sponsoring this study?
  • Who has reviewed and approved the study?
  • How long will the study last?
  • Who will be in charge of my care during the study?
  • Will I have to travel to be part of this study? If so, who will pay for travel?

About tests or treatments in the study:

  • What kinds of tests and treatments are involved?
  • What tests will I get as part of this study?
  • Will I have to pay for any treatment or test?
  • How do the possible drawbacks and benefits compare with those of my current treatment?
  • What are the known short-term and long-term side effects of the treatment?
  • How might being in this study affect my daily life?
  • Will I need to go into the hospital to get the treatment or drug? If so, how often and for how long?
  • How will I know that the treatment is working? Will results of the study be shared with me?
  • If the treatment being studied is found to be harmful or not working, will I stop taking it?

After the study is over:

  • Will I be able to still get this treatment after the study ends?
  • What type of long-term follow-up care is part of this study?