This guide, which will be published as a series of articles over the next couple of weeks, is intended to help clarify the clinical trial process and to answer common questions so that you can decide whether a clinical trial is the right option for you. The guide will explain the different types of clinical trials, address risks and benefits associated with participating in clinical trials, explain the details of participating in clinical trials, and provide resources for finding a clinical trial

For patients who are considering participating in a clinical trial, it is important to be entirely comfortable with the decision. This article will provide resources for finding a clinical trial as well as explain details of participating in them.

Where can I find a clinical trial that is right for me?

MDS patients can actively seek clinical trials that best fit them through several methods.

Patients can first ask their doctors if they know of currently recruiting clinical trials that may be appropriate for them.

In addition, the clinical trial registry of the United States National Institutes of Health allows patients to search for MDS-specific studies conducted in the U.S. and worldwide.  Similar lists can also be found at eCancerTrials.com, CenterWatch.com, ClinicalTrialsSearch.org, and the National Cancer Institute website.

Organizations like the Leukemia & Lymphoma Society (LLS) and Aplastic Anemia & MDS International Foundation (AA&MDSIF) have also compiled their own lists of studies for MDS patients.

Finally, patients may want to look for studies by location, such as those being conducted at specific hospitals or clinical trial centers.  Examples of hospital-based clinical trials for MDS can be found for City of Hope, Memorial Sloan-Kettering Cancer Center, Mayo Clinic, and the Fred Hutchinson Cancer Research Center.

What should I do after I find a clinical trial?

It is important for patients to learn as much as possible about the clinical trial before deciding to participate.

First, patients should ask their doctors about the clinical trial.  Physicians who know the possible risks and benefits of the treatment under investigation can offer individualized insight for their patients, ensuring that it is appropriate for them.

Patients should then meet with the study’s research coordinator, who is responsible for clearly explaining what participation will entail.  Patients are welcome to bring a family member or friend to the meeting for support.

At the meeting, patients should learn details of the study that will help them decide if they want to participate.  Meeting with the research coordinator does not mean that a patient is eligible for the trial or that they have agreed to participate.  In addition, they are not required to make a decision at the meeting.

Patients should come with questions and not be timid about asking for any clarification.  It is recommended that patients take notes or ask the research team for permission to record the meeting for later reference.  Possible questions to ask may include:

• What is the purpose and phase of this study?
  • What is the treatment?
• Why do you believe the treatment being tested is effective?
• Has it been tested before?
  • If so, what were the results?
  • Who can participate in this study?
  • How long will this study last?
  • How will the treatment be administered?
  • How much will I be given, and how often?
  • What are the potential risks and benefits of this treatment?
    • Are there any potential side effects?
  • What are the potential short- and long-term effects?
  • Am I required to undergo lab tests?
    • If so, which ones?
  • Who will oversee my treatment, my local physician or the study’s investigator?
  • What are the expected effects on daily living?
  • How will you measure if the treatment is successful?
  • What happens if the treatment is ineffective or harmful?

Other important questions that may not be associated with the actual treatment include:

• Will I be informed about the results of the study?
• Does participation require time commitments, such as extra travel, doctors’ visits, or hospitalization?
• Who will pay for the treatment?
  • Will I be compensated for travel and/or meal costs?

Patients should also consider what will happen after the study is completed:

• Will the treatment still be available to me after the specified length of the trial?
• Are you planning to do long-term follow-up after completing the study?

How much will it cost to participate in a clinical trial?

The research organization that sponsors the clinical trial usually pays for expenses related to conducting the trial, including the treatment, personnel, and analysis of results.

Other costs related to patient care may or may not be covered by a patient’s insurance. Patients should ask their doctor about coverage.  Routine care costs that relate to doctors’ visits, hospital stays, lab tests, and x-rays are usually covered regardless of trial participation.  However, extra costs related to tests that are not part of standard care may not be included.

Patients should also consider travel and food expenses that accompany extra doctors’ visits and tests.  These costs are sometimes covered by trial sponsors. Organizations such as the AA&MDSIF may offer to financially reimburse participants for travel costs.

What happens after the clinical trial ends?

The research team may follow participants for a time specified in their protocol after the treatment period ends to monitor any short and long-term side effects.  Patient results will usually be categorized as having no response, partial response, or complete response leading to disease-free or progression-free survival.

Depending on the level of response, patients may choose to remain on the treatment by enrolling in the extension arm of the study, which is sometimes made available by the sponsoring organization.  Patients who participate in the extension arm continue to receive treatment at no cost.

If this option is unavailable, patients may also choose to purchase the drug, if it has been approved by the FDA by that time.  Insurance coverage and payment methods may vary by treatment type.

Finally, if patients are unresponsive, they can choose to return to their previous treatment or try another treatment after talking with their doctor.  Patients may also be eligible for other clinical trials investigating different therapies; however, they must usually wait a specified amount of time before enrollment.

What if I am currently ineligible for ongoing clinical trials?

Patients who do not meet the criteria to participate in clinical trials may consider expanded access protocols.  In these studies, the FDA allows manufacturers to distribute experimental treatments to patients who may benefit from the treatment but are ineligible for ongoing clinical trials.

However, there must be existing evidence that the treatment is potentially effective for patients. Evidence can be provided through ongoing or completed clinical trials.  Patients may consider participating in expanded access studies if there are no good treatment alternatives available and their disease is life-threatening.

Results from expanded access studies can contribute more information about the safety of the investigational treatment.  However, because expanded access protocols are not under the same strict guidelines as clinical trials, patients may be at risk for unforeseen safety issues.  Expanded access studies can be found at the clinical trial registry of the U.S. National Institutes of Health.

Who will benefit from my participation?

It is often difficult to envision the names and faces of future MDS patients who will be treated or cured through participation in a clinical trial.  However, it is important to remember that every MDS patient who currently receives treatment or has been cured is indebted to years of research in the past.

Hopeful reminders for MDS patients can be found through stories of survival and cure, such as those on the AA&MDSIF or Fred Hutchinson Cancer Research Center websites.  Individualized resources, such as the LLS-based My Personal MDS Journey, may help patients know that they are not alone.

Participating in clinical trials is just one way that patients can partner with the medical community to take small steps toward preventing, treating, and curing diseases like MDS.

For more information about clinical trials for MDS patients, please see Part 1: Learning About Clinical Trials and Part 2: Benefits Ands Risks Of Clinical Trials.