LEMS and Clinical Trials
While no cure currently exists for Lambert-Eaton myasthenic syndrome (LEMS), researchers are working on developing new treatments. Clinical trials are essential steps in this process.
Here is information that may help you to decide whether you would like to join a LEMS clinical trial.
What is a clinical trial?
The goal of clinical trials is to determine whether a new device, procedure, or pharmaceutical therapy works, and if it is safe. Clinical trials may also test new ways of using existing treatments, and assess other aspects of care. In an observational study, they simply record daily life with a disease over time.
Trials are controlled and monitored to ensure they are carried out as intended, and that any issue or potential risk is identified as soon as possible. They are regulated by law, and require governmental approval of their approach and goals before they can begin.
Is a trial an experiment?
Yes, it is. Scientists who design a trial aim to gather enough scientific evidence, meaning evidence that is clinically meaningful and reproducible, to support an application to a regulatory body — in the U.S., the Food and Drug Administration (FDA) — for approval of what they are testing.
What are the different phases of a clinical trial?
New medications typically undergo three trial “phases.” In the first phase, a relativity small number of participants are drawn from the general population to test the proposed treatment for safety, tolerability, and other properties. Phase 2 involves testing in the intended patient population. Tests here are for safety and early evidence of effectiveness in a generally small number of patients. A Phase 3 trial aims to show scientifically — with a statistically valid and representative patient group, to minimize scientific bias — that the treatment is of benefit for its target population.
What are the potential benefits?
By enrolling in a clinical trial, you can:
- Gain access to new treatments before they are widely available
- Receive regular and careful medical attention from a research team that includes physicians and other health professionals
- Help others by contributing to knowledge about new treatments or procedures
Are there any risks?
Any experimental treatment can carry unpleasant, serious, or even life-threatening risks. Additionally, the trial may require more time and attention than standard care would, including study site visits, repeat blood tests, and extensive procedures that might require hospital stays. There may also be stringent and sometimes complex dosing schedules.
Questions to ask
If you are considering registering for a LEMS trial, you should feel free to ask any questions or broach any issues concerning the trial at any time. Make a list of your questions or concerns, so you are sure to address all of them.
Here are some general questions you might want to ask:
- What is the purpose of the study?
- Who will fund the study?
- How long will the study last?
- Who will inform me of the study results?
- What are possible benefits and risks?
- Are there other options available?
- What kinds of therapies, procedures, or tests will I undergo?
- Does this trial have a placebo, or sham treatment, group?
- Who will be in charge of my care?
- How might taking part in this trial affect my daily life?
- Can I talk to other people in the trial?
- Will I have to pay for any part of this trial, including travel to and from a test site?
Who can enroll in a clinical trial?
Clinical studies have standards, called eligibility criteria, outlining who can participate. These are based on characteristics such as age, gender, the disorder’s type and stage, the patient’s previous treatment history, and the presence of other medical conditions.
A number of LEMS trials are taking place at universities and medical centers globally. You can also contact the Muscular Dystrophy Association (MDA) about ways to stay apprised of current and upcoming studies.
Doctors treating you for LEMS could discuss a trial’s details, like eligibility requirements, and may be able to help advise you as to which trials are best suited to you.
Last updated: July 20, 2020
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