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Are you interested in participating in a clinical trial on myeloproliferative neoplasms (MPN)?

Updated: Jul 19, 2023

If so, you may wonder what a clinical trial entails, its benefits and risks, and what questions to ask before agreeing. This article explains some fundamental aspects of clinical trials for MPN, a type of blood cancer affecting the production of red and white blood cells and platelets.



Clinical trial on myeloproliferative neoplasms (MPN)


Content guide



What are clinical trials?


Clinical trials are scientific investigations that test the safety and efficacy of new treatments or interventions for a specific disease or condition.


Clinical trials are necessary to advance medical knowledge and offer better therapeutic options to patients. Clinical trials of MPN seek to find new drugs or drug combinations that will improve disease control, quality of life, and survival in patients with essential thrombocythemia (ET), myelofibrosis (MF), or polycythemia vera (PV).

There are also clinical trials investigating the causes and molecular mechanisms of MPNs, such as the discovery of JAK2, calreticulin, and other mutations.



Phases of a clinical trial


Clinical trials are divided into different phases, depending on the objective and the number of participants:

  • Phase I: a new treatment is tested in a small group of healthy volunteers or patients to evaluate its safety, dose, and possible side effects.

  • Phase II: A new treatment is tested in a larger group of patients to assess its efficacy and safety compared to a standard treatment or placebo.

  • Phase III: A new treatment is tested in a large and diverse group of patients to confirm its efficacy and safety compared to a standard or placebo treatment. This phase is usually the last one before applying for regulatory approval of the treatment.

  • Phase IV: it is carried out after the treatment has been approved and marketed to monitor its long-term effects, safety in different populations, and usefulness in clinical practice.



How long does a clinical trial last?


The length of each trial varies depending on the type of trial. You will know how long you must participate in a trial by reading the informed consent form and talking to the staff. They will explain to you what the trial involves and what your role is.


Eligibility for clinical trials


Not all patients with MPN can participate in any clinical trial. Each clinical trial has eligibility criteria that define who may or may not participate, depending on factors such as the type and stage of the disease, previous treatment, age, and condition.


What is expected in a clinical trial?


Clinical trials are research studies that test new treatments or procedures. They can help you access cutting-edge therapies that are not widely available. However, they also have some uncertainties and possible side effects. Before joining a clinical trial, you should learn as much as you can about the study and what it involves. You will have a team of health professionals who will guide you through the process and monitor your health closely. You can always ask them any questions or share any worries you have. Participating in a clinical trial is your choice. You can withdraw from the study anytime without affecting your regular care.



Best nine questions to ask if you are interested in participating in a clinical trial


If you are interested in participating in an MPN clinical trial, you should be well-informed about the trial's goals, procedures, benefits, and risks. You should also know your rights and responsibilities as a participant. Some questions you can ask the research team are:

  • What is the purpose of the clinical trial?

  • What treatment will be tried, and how is it administered?

  • What tests or visits are required during the clinical trial?

  • What benefits can I expect from the clinical trial?

  • What risks or side effects can I have from participating in the clinical trial?

  • What alternatives do I have if I do not participate in the clinical trial?

  • Who finances the clinical trial and supervises its ethical and scientific quality?

  • What rights do I have as a participant, and how can I withdraw from the clinical trial if I wish?

  • What follow-up will be done after the end of the clinical trial?



Will there be any cost to me if I participate in a clinical trial?


If you join a clinical trial, you may get some money back for your expenses because of the study (for example, travel, food). You need to ask for approval before you spend money on some things. It would help if you talked to your study doctor about the costs you can expect. You or your insurance company will not have to pay for these things: Study Drug(s) tested in the study for a new use, study visits, and any medical tests only for the analysis. You may have to pay for other things (for example, tests you would get any way for your condition), and your insurance may cover some of them.



What is a Clinical Trial Informed Consent Form (ICF)?


To participate in a clinical trial, you must give your informed consent in writing, which means that you have understood all the information about the clinical trial and voluntarily agree to participate. Informed consent is not a contract; you can withdraw it without negative consequences for your health care.

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