Clinical trials are how new treatments advance and become available to help patients. In rare diseases like systemic mastocytosis (SM), a clinical trial can mean another option for a disease with limited treatments. But they don’t happen overnight: Clinical trials take year of effort from researchers and patients. In brief, here is how it all works.
What is a clinical trial?
A clinical trial is a research study on a new medication, medical device or approach to care in patients. These medical studies are carefully controlled to test how well an experimental therapy works and if it’s safe before the treatment is approved and made available to the wider patient population.
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How does a clinical trial work?
There are four phases of a clinical trial, but not every trial proceeds through all four phases. If at any stage researchers observe unexpected or dangerous side effects, that a new treatment isn’t working or that or the study’s objectives are not being met, the trial will be stopped.
Phase 1: This phase is the first time a new treatment is tested in humans. It uses small groups of patients to learn more about if it is safe, if it causes side effects and which doses work best.
Phase 2: If safety is confirmed in phase 1, phase 2 tests the treatment on a larger groups of patients. Hoe effective the treatment is, its safety and side effects are all studied.
Phase 3: In phase 3, the study is expanded to a much larger group of patients, with a more diverse population and different doses. If this phase is successful, the treatment will be approved by the Food and Drug Administration (FDA).
Phase 4: Following approval of the drug or treatment, researchers continue to study its long-term effects on patients.
What is being in a clinical trial like?
Th experience of being in a clinical trial will very much depend on the specific study. In general, though, being in a clinical trial means many extra medical appointments so the researchers can carefully monitor your health.
For some patients, this may mean regular travel to the hospital or center where the trial is being conducted. Other patients might be able to get their care close to home.
For many, being in a clinical trial means the exact care they’re getting could be a mystery for months or years: Many clinical trials are “blinded,” which means patients don’t know if they are receiving the new therapy or a placebo. Some studies are single-blinded — meaning the researchers know which patients are receiving the experimental treatment — while others are double-blinded, meaning neither the researchers nor the patients know who is receiving the actual treatment until after the study concludes.
Read one patient’s reflection on participating in a clinical trial: “Bidding a tearful goodbye to a life-changing clinical trial”
How can you join a clinical trial?
Your healthcare team is your first point of contact if you’re interested in joining a clinical trial. They have access to information on clinical trials that are looking for patients; with your consent, they can share your medical history and patient profile to have your eligibility assessed.
Different clinical trials will have their own criteria for who they accept. For example, a trial on a new on SM therapy may be specially looking for patients with indolent SMIndolent SM A subtype of nonadvanced SM caused by the abnormal accumulation of mast cells in the bone marrow and other organs. Indolent SM accounts for around 90% of SM cases., or an advanced subtype.
There are also online clinical trial registers such as ClinicalTrials.gov and the EU Clinical Trials Register that provide up to date information on ongoing trials.
Before joining a clinical trial, it’s important to have in-depth conversations with both your regular care team and the clinical trial team about the trial. You’ll want to know what the risks are, how long it will last, if you’ll need to stop taking your current medications and how the trial will impact your daily life, among many other questions. Clinical trials carry many pros and cons that every individual needs to weigh for themselves.
