Systemic mastocytosis (SM) is a rare disease with no known cure. Ongoing research through clinical trials tests new potential treatments on animals, then people, to evaluate the safety and efficacy of a new medicine or treatment.
People living with SM may benefit from having access to innovative therapies that are still under research. These may potentially help treat symptoms, contribute to slowing disease progression or even provide a cure.
What is SM?
Systemic mastocytosis (SM) is a rare hematological disease characterized by mast cells that are overactive and accumulate in different parts of the body such as the bone marrow, liver, spleen, gastrointestinal tract and lymph nodes.
What are the purposes of clinical trials?
People living with SM may find the symptom burden can be hard to manage, with a significant effect on quality of life. As the disease progresses, it may lead to organ damage and, in severe cases, a reduced life expectancy. A clinical trial may be considered a way to receive a new treatment as early as possible.
Read more about SM treatment and care
Participating in a clinical trial can allow you to access potentially ground-breaking treatments before they become available. Most commonly, such trials will test new treatments or new approaches such as combinations of different treatments or targeted therapies. By joining a clinical trial, you will also contribute to scientific progress and help others who suffer from SM.
While the benefits can be substantial, there are also risks to be considered. The two main potential disadvantages include side effects from the new medicines being studied and the possibility that they may have no positive effect on symptoms or disease progression.
In addition, clinical trials often involve two groups: one testing the medicine and one that receives a placebo, which is not the medicine and is used as a comparison. You may find yourself in the group that doesn’t receive the new therapy.
How can you find out about clinical trials?
There are many different clinical trials running at any one time, and if you’d like to apply to join one, you’ll need to be well-informed. You can find out about them in the following ways.
Your health care team: When choosing your primary health care provider following diagnosis, ensure he or she is proactive about staying informed about upcoming clinical trials. Doctors play an important role in identifying eligible patients and helping them to navigate the recruitment and consent process. Your doctor will also be able to answer any questions you may have about what to expect from a trial.
Personal research: The website ClinicalTrials.gov is an excellent way to become familiar with the world of clinical trials, how they work and how to participate. It offers a comprehensive section of patient resources full of information that is easy to understand.
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