There currently is no cure for Ehlers-Danlos syndrome (EDS), and not many options for treating the genetic disease’s symptoms, which can range from loose joints to fragile skin to cardiac problems. None of the treatments available address the disease’s underlying cause. Participating in clinical trials is a way to help researchers develop new and better therapies.
The following information will give you better insight into what clinical trials are, how they work, and what questions you may wish to ask if you are considering taking part in one.
What are clinical trials?
Clinical trials are part of clinical research and are at the heart of all medical advances. The goal of clinical trials is to determine whether a device, procedure, or pharmaceutical therapy works and is safe. These trials also may test new ways of using existing treatments, assess other aspects of care, or in the case of an observational study, simply record the daily lives of those living with a disease over time. Such observational studies can provide important data for researchers and clinicians, particularly about real-life symptoms and challenges.
People with the disease and healthy volunteers, known as controls, may be enrolled in clinical trials. All trials are controlled to ensure they are carried out as intended, and that all participants are monitored so that any issue or potential risk is identified as soon as possible. Clinical trials are regulated by law and require governmental approval before they can begin.
Is it an experiment?
Yes. Scientists who design the trial aim to gather enough scientific evidence — evidence that is clinically meaningful and reproducible — to support an application to a regulatory body for approval of what they are testing. In the U.S., all applications are submitted to the Food and Drug Administration, known as the FDA, for approval.
It is important to remember that a new treatment that researchers are testing may have serious side effects that they cannot foresee.
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 healthy participants are drawn from the general population to test the proposed treatment for safety, tolerability, and other properties. Phase 2 involves testing the medication in the intended patient population. Tests here are for safety and early evidence of effectiveness, and generally include a small number of patients. A Phase 3 trial aims to show scientifically that the medicine is of benefit for its target population. This phase usually requires a statistically valid and representative patient group, to minimize scientific bias. Oftentimes a Phase 3 trial will have a larger number of participants than the earlier studies.
Who can enroll in clinical trials?
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.
What are the potential benefits?
By enrolling yourself or your child 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
Questions to ask
If you are considering registering for an EDS 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.
Some general questions you might want to ask include:
- 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 the possible benefits and risks?
- What kinds of therapies, procedures, or tests will I (or my child) undergo?
- Who will be in charge of my (child’s) care?
- Where will the study be held?
EDS clinical trials
There are ongoing needs for new therapies, interventions, and tests as a means to prevent, detect, treat, or manage EDS, which occurs in about 1 in 5,000 births worldwide. Notably, many of EDS’ types are far rarer. Under the current 2017 classification system, there are 13 types of EDS.
A number of EDS clinical trials are taking place at universities and medical centers globally. Each study summary provides a list of where it runs, and whether it is recruiting. To participate in a study, it’s often necessary to contact the study coordinator of the participating institution. Your healthcare team may help you find trials that may be of interest to you.
You may find a list of current EDS studies on the website of the U.S. National Library of Medicine. Current trials range from the evaluation of compression garments in EDS (NCT03986229) to a study (NCT03596437) involving ultra-high frequency ultrasound in vascular EDS.
The Ehlers-Danlos Society also can help keep you apprised of current research needs and opportunities.
Last updated: Dec. 2, 2020
Ehlers-Danlos News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.
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