• A research study, or clinical trial, for myeloproliferative neoplasms (MPNs) is a way to evaluate the safety and effectiveness of a potential new therapy. Some studies evaluate drugs that doctors cannot yet prescribe.
• Before deciding to join a research study, you will be provided with detailed information to help you make an informed decision about participating.
• Research studies may be an option for people with MPNs regardless of disease stage or risk for progression.

Participating in a research study may be a treatment option if you have a myeloproliferative neoplasm. Myeloproliferative neoplasms are a group of rare blood cancers in which bone marrow overproduces red blood cells, white blood cells, or platelets. There are three types of MPNs: primary myelofibrosis, essential thrombocythemia, and polycythemia vera. Common symptoms of myeloproliferative neoplasms include fatigue, weakness, abnormal bleeding, night sweats, and fever.

Currently, there are several treatments for myeloproliferative neoplasms, including phlebotomy, chemotherapy, and targeted therapy, as well as stem cell transplant in specific instances. The recommended treatment depends on your type of MPN, what symptoms you have, and the risk of disease progression. Your doctor may recommend a watch-and-wait approach if your condition is deemed low risk for progression. More aggressive interventions may be recommended if your condition is considered high risk.

In addition to currently available treatments for myeloproliferative neoplasms, research studies — also called clinical trials — may provide another option. Misunderstandings about what clinical trials involve and who can participate may make some people with MPNs less likely to consider a study. Below, we’ve highlighted some important facts about participating in research studies.

1. Participating in a research study may allow you to access new treatments.

As part of a clinical trial, you may have access to treatments that are in development, new combinations of treatments, or existing treatments being studied for a new use. The treatment being studied is called an investigational treatment or therapy.

By joining a trial, you can help ensure the study population includes people with your age, gender, and ethnic background. A diverse study population helps scientists understand how treatments work among people across demographics.

Participating in a research study can help scientists make advancements for all people with MPNs. The goals of clinical trials vary. They can help researchers answer the following questions:

• Is a new medication safe and effective?
• What dosage of an existing or new medication works best?
• Is a new treatment better than existing treatments?
• Does a new treatment cause fewer side effects than existing treatments?
• Does a new treatment help people who haven’t responded to other treatment options?

Read more about how research in MPNs leads to treatments that improve lives.

2. You will always be informed if there is a chance you will receive a placebo.

A placebo, sometimes called a “sugar pill,” is designed to look like the treatment being studied. However, it lacks the treatment’s active ingredient. To test the effectiveness of an investigational treatment, clinical trials may assign a group of participants a placebo. You will always be informed if there is a chance you will receive a placebo.

Placebos are rarely used alone in cancer clinical trials because it would be unethical to withhold treatment. In most cancer studies, an investigational treatment is compared to an existing treatment. A placebo may be used alone in a cancer trial only if no effective treatment currently exists.

Placebos may also be used when a clinical trial is testing an existing treatment in combination with a new treatment. One group of participants might receive the existing treatment plus a placebo, and the other group receives the existing treatment in combination with the investigational treatment.

In most trials, researchers will determine randomly who will receive the investigational treatment versus a placebo or existing treatment, to avoid bias. This process is called randomization. Many research studies are also double-blind, meaning neither the trial participant nor the researchers know which participant is receiving which treatment.

In some situations, if the investigational treatment appears to be much more effective than the treatment it’s being compared to, it may be made available to all participants after a certain period of time or under specific circumstances. When considering a clinical trial, you can ask questions about the study design and whether you will have access to the investigational treatment if you are assigned an existing treatment or a placebo.

3. There are measures in place to protect your safety and well-being when participating in MPN research.

The safety of research study participants is extremely important, and there are guardrails in place to protect you. These protections are a response to historical abuses when individuals’ rights were not protected in medical research.

International Review Board

In the United States, an ethical body called the Institutional Review Board (IRB) approves and monitors the majority of clinical trials. The IRB’s role is to protect the safety and rights of participants in research studies. The IRB will assess the study to ensure the possible benefits of the trial outweigh the risks and that appropriate precautions are in place to protect participants. If during the course of the trial, it is determined that it is no longer safe to proceed, the study will be discontinued.

Informed Consent

Before joining a research study, you will be given comprehensive information about the risks and potential benefits to ensure you can make an educated decision about participating. This process is called informed consent.

All treatments have potential risks. Even drugs that appear to be safe and effective must undergo testing. Clinical trials help researchers understand if a treatment is safe and if the potential benefits outweigh the potential risks. The informed consent process is there to ensure you understand and are comfortable with the potential risks and benefits of joining a research study.

You will also be given general information about the research study. The goals of clinical trials vary. Some trials are designed to test new treatments, while others test new combinations of existing treatments or test new uses for existing treatments. Some clinical trials don’t test treatments at all and instead evaluate ways to improve overall health or quality of life.

There are also different phases of clinical trials. Phase 1 trials focus primarily on the safety of a treatment, while phase 2 and phase 3 trials focus on efficacy in addition to safety. Understanding the phase of a trial can help you evaluate if it’s a good fit for you.

You can speak with family members, caregivers, trusted friends, your hematologist, or anyone else you turn to for guidance when deciding if a research study is right for you.

4. People with different types and stages of MPNs can participate in research.

A research study may be appropriate for anyone with primary myelofibrosis, essential thrombocythemia, or polycythemia vera, whether or not your condition is advanced. Each trial will have specific criteria about who is eligible. These criteria may include your disease stage and your response to other treatments you may have tried. The goal of research studies is to improve care and treatment, even if a person is feeling well or their disease has a low risk for progression.

There are several clinical trials for MPNs currently underway. Some of these trials focus only on individuals with advanced disease or individuals who have not seen improvement with other medications. Other trials include people with low-risk disease.

5. You are free to leave an MPN research study at any time.

You can withdraw from a research study at any time, for any reason, even if the study is still in progress. Signing consent documents and agreeing to participate in a trial is not a binding contract.

If you do choose to withdraw from a study, you should inform the research staff and follow the protocol for leaving the trial. Depending on the trial, you may need to follow special instructions or undergo monitoring when discontinuing the study treatment. If you are concerned about the trial or are considering withdrawing, reach out to the study team or your doctor.

6. You will likely still visit your regular MPN care providers throughout the course of a trial.

MPN studies may take place at special cancer centers with hematology and oncology research programs. However, these specialists do not necessarily replace your other health care.

Generally speaking, you should still see your regular health care providers when enrolled in a clinical study. The medical care you receive as part of the trial doesn’t replace comprehensive health care. Your doctors can coordinate with the research team to ensure the clinical trial treatment doesn’t interact negatively with any other treatments you are taking.

While you will likely continue seeing your existing health care providers, clinical trials usually require that you receive follow-up care at the facility where the trial is being conducted. Your doctor can help you understand how participating in a research study would impact your care.

Learn more about talking to your doctor about clinical trial participation.

Find Your Team

On myMPNteam, the social network for people with MPNs and their loved ones, members come together to ask questions, give advice, and share their stories with others who understand life with MPNs.

Have you considered a clinical trial for MPN treatment? Share your experience in the comments below, or start a conversation by posting on your Activities page.