By The Marfan Foundation

The VEDS Movement Director Ryan Rodarmer is a genetic counselor and Type A dissection survivor. Here, Ryan shares his insights regarding advancing progress when it comes to VEDS.

Living with Vascular Ehlers-Danlos syndrome (VEDS) means navigating unique health challenges. This condition affects the body’s connective tissue – the “glue” that holds cells and organs together. Because connective tissue is found everywhere, from our hearts and blood vessels to our abdominal organs and eyes, this syndrome can cause many symptoms and serious health risks, especially involving the aorta, the body’s main artery. 

Finding better ways to diagnose, manage, and treat this condition is incredibly important for everyone affected. This is where clinical trials come in – they are the key to unlocking progress and offering hope for the future. 

What Exactly Are Clinical Trials? 

Think of clinical trials as carefully planned research studies involving people. Their main goal is to find out if new medical approaches – like medicines, devices, or ways to manage symptoms – are safe and effective. Before any new treatment can become widely available, it must go through several phases of clinical trials. 

• Phase 1: Usually involves a small group of people to test safety and dosage. 

• Phase 2: Involves a larger group to see if the treatment works and to further check its safety. 

• Phase 3: Involves an even larger group to confirm effectiveness, monitor side effects, compare it to existing treatments, and gather information that will allow the new approach to be used safely. 

Why a Placebo Group is Often Essential 

You might hear about ‘placebo-controlled’ trials, particularly in later phases. In these studies, some participants receive the active treatment being tested, while others receive a placebo – an inactive substance (like a sugar pill or saline injection) designed to look identical to the active treatment. This comparison group is critical for several reasons. Firstly, it helps researchers distinguish the actual effects of the experimental treatment from the “placebo effect” – where people sometimes feel better simply because they believe they are receiving treatment. Secondly, it helps account for the natural course of the condition; symptoms might improve or worsen over time regardless of treatment. By comparing the outcomes in the group receiving the active treatment to the group receiving the placebo, researchers can scientifically determine if the treatment is genuinely effective and causing the observed benefits, beyond psychological effects or natural changes. This rigor is crucial for proving a treatment truly works. 

Why Are Trials So Crucial for the VEDS Community? 

VEDS is considered a rare condition. While “rare” might sound isolating, it makes participation in clinical trials even more powerful. Here’s why: 

1. Overcoming Rarity Through Participation: Because VEDS is a rare condition, researchers face challenges gathering enough data to conduct meaningful studies. Every person with VEDS who joins a clinical trial provides invaluable information. This participation is essential for researchers to understand if a potential treatment works and is safe specifically for individuals with VEDS. Without sufficient participation from the VEDS community, studies can face significant delays or may not be possible, hindering progress towards potential breakthroughs. 
2. Addressing the Unique Biology of VEDS: VEDS is a distinct condition involving connective tissue, with a specific genetic cause and unique effects on the body. Because of these unique characteristics, a treatment that works well for the general population, or even one designed for other connective tissue conditions, might not be the most suitable or safest approach for managing VEDS specifically. This is why clinical trials focused directly on VEDS are so vital – they allow researchers to develop and test treatments carefully matched to the unique biology and challenges presented by VEDS. Furthermore, VEDS trials often narrow their focus, perhaps concentrating on specific age groups, particular genetic variations within VEDS, or certain clinical measurements. This necessary specificity means that not everyone living with VEDS may be eligible to participate in every VEDS trial. Clearly defining these participant groups is crucial for researchers to accurately determine if a potential treatment is truly safe and effective for the specific VEDS population it’s intended to help. 
3. Understanding VEDS Better: Understanding how VEDS naturally progresses over time (its natural history) is vital for developing and evaluating potential treatments. Specific studies, including observational studies as well as data collected during clinical trials, are crucial for building this knowledge base. Clinical trials allow researchers to test whether a potential therapy can alter the expected course of VEDS for the better. Every observation made during a trial contributes to this essential understanding, helping to refine research questions and guide the development of future therapies specifically for VEDS. 

Safety and Your Choice 

It’s natural to have questions about safety. Clinical trials are highly regulated. In the U.S., this involves oversight from bodies like the Food and Drug Administration (FDA), particularly for studies involving new drugs, new uses for existing drugs, or medical devices. The FDA reviews trial results on safety and effectiveness before deciding if a treatment can be approved and get a specific label for use. Additionally, before a trial starts locally, it must be approved by an Institutional Review Board (IRB) or ethics committee. These local groups ensure the study is ethical and that participant safety is protected throughout the trial. 

Everyone who considers joining a trial goes through a process called “informed consent.” This means the research team explains the study in detail – its purpose, procedures, potential risks, and potential benefits. You have the right to ask questions and decide whether or not to participate without any pressure. Participation is always voluntary, and you can choose to leave a trial at any time. 

How Can You Get Involved or Learn More? 

If you’re interested in learning about clinical trials relevant to VEDS: 

• Talk to your doctor: Your specialist is the best person to discuss whether any ongoing trials might be suitable for you or your family member. 
• Connect with patient advocacy groups: The VEDS Movement has information about current research and trials. Contact thevedsmovement.org/ask or learn more through our umbrella Foundation website Get Involved in Research – The Marfan Foundation
• Explore online databases: Websites like ClinicalTrials.gov list publicly and privately funded clinical studies conducted around the world. 
  

A Community Effort 

Progress in understanding and managing VEDS depends fundamentally on research, and that research relies entirely on people within the VEDS community willing to participate in clinical trials. By considering participation, you potentially gain access to new approaches being studied and, crucially, you contribute invaluable knowledge. This knowledge is essential for advancing care and can help countless others living with VEDS, both now and in the future – potentially including your own family and loved ones. Participating in research is a powerful way for the VEDS community to actively work together towards a brighter, healthier future. 

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