MPN Clinical Trials: What You Need to Know

What is a clinical trial?
What is the purpose of a clinical trial?
Why participate in a clinical trial?
Things you should know about participating in a clinical trial.
Useful questions to ask before participating in a clinical trial.

What is a Clinical Trial?

By definition, a clinical trial is a comparison test of a medication or other medical treatment (such as a medical device), versus a placebo (inactive look-a-like), other medications or devices, or the standard medical treatment for a patients’ condition. The number of patients can range from as few as 30 to hundreds or thousands. There are two kinds of clinical trials:


Participants are observed by researchers as to their outcomes.


Participants are assigned a treatment as researchers observe their outcomes.

The ethical and legal codes that govern medicine also apply to clinical trials. There are many checks and balances in place to ensure the safety of clinical trial participants and the study plan is thoroughly vetted before testing in human subjects is allowed.

What is the purpose of a Clinical Trial?

  • To assess the safety and effectiveness of a new medication or device on a specific patient
  • Assess the safety and effectiveness of a different dose of medication than is commonly used
  • Assess whether the new medication or device is more effective for the patients’ condition than the already used, standard medication or device
  • Compare the effectiveness in patients with a specific disease of two or more already approved or common interventions for that disease.

Why participate in a clinical trial?

Help medical science to advance treatments and better understand disease

Trials make new research treatments available to participants before they are widely available or fully approved

Help researchers test treatments and observe the outcome for disease in a diverse patient population. This is important so scientists understand how a disease works within different demographic groups (like gender, age, race). This also affords the researchers the ability to observe differences in reaction among patient groups to new treatments options. Click here to learn why diversity in clinical trials is important.

Some things you should know about participating in a MPN clinical trial

It is important to meet with your hematologist to decide whether a MPD clinical trial exists that is appropriate for your stage of disease, and whether the logistics of participation with that specific trial works for you.

All MPN clinical trials have guidelines about who can participate. These Inclusion / Exclusion criteria vary for each trial, and are based on factors such as age, gender, the type and stage of a disease, and other medical conditions. Your hematologist can help you determine whether you are qualified for the study.

The process of Informed Consent occurs before during and after participation in a clinical trial. Study details (including risks and potential benefits) are explained by doctors and nurses and are also provided in writing. If your native language is not English translations are available.

As you are taking this step be prepared to be actively involved in the process. Jot down any questions you have and visit the clinic or investigation site prepared to take notes. Feel free to bring a tape recorder with you.

There could be costs involved which may or may not be covered by your insurance. Be sure to check with your doctor, the researchers and your insurance when considering the financial ramifications.

Useful questions to ask before participating:

  • What is the purpose of the study?
  • Who is going to be in the study?
  • Why do researchers believe the experimental treatment being tested may be effective?
  • Has this been tested before?
  • What kinds of tests and experimental treatments are involved?
  • How do the possible risks, side effects, and benefits in the study compare with my current treatment?
  • How might this trial affect my daily life?
  • How long will the trial last?
  • Will hospitalization be required?
  • Who will pay for the experimental treatment?
  • Will I be reimbursed for other expenses?
  • What type of long-term follow up care is part of this study?
  • Will results of the trials be provided to me?
  • Will my current physicians (hematologist/primary care) be made aware of the results?
  • Can a participant leave a clinical trial after it has begun?

Additional sources:

The National Institutes of Health (NIH) provides information about clinical trials at Since the database is a repository of trials for all diseases, we recommend you obtain MPN-specific trial information by typing the specific disease (eg. polycythemia vera) in the Search Clinical Trials box.

The National Cancer Institute (NCI) provides information on clinical trials for all cancers from around the world at We recommend you obtain MPN-specific trial information by selecting “myeloproliferative disorders” in the Type of Cancer box. In the Subtype of Cancer box, select the specific MPN (e.g. essential thrombocythemia).

This information is provided to patients as a valuable resource and is not meant as an endorsement of any trials, institutions or physicians. Please consult your physician for advice before agreeing to join any trial.

Take the steps you need to help change your prognosis.



Get free, timely information on living with an MPN.