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The DiSCOVER TRIAL FOR VEDS

Decentralized Study Evaluating Celiprolol on Vascular Ehlers-Danlos Syndrome (VEDS) Related Events.

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CRITERIA FOR ELIGIBILITY IN CELIPROLOL CLINICAL TRIAL

To pre-qualify for this clinical research study, you must meet the following criteria:

  • Must be greater than or equal to 15 years old
  • Willing to obtain a magnetic resonance angiogram (MRA) image
  • During screening, must have a genetic test performed, via a saliva sample, to confirm the presence of the COL3A1 mutation which will confirm a VEDS diagnosis; you will need a positive test result for Vascular Ehlers-Danlos Syndrome with a COL3A1 gene mutation.
  • Must be able and willing to discontinue use of beta blockers under the care of your primary physician
  • Should not have had an arterial rupture or dissection, uterine rupture, and/or intestinal rupture within the last 6 months
  • Must be willing to complete all study procedures

Please note this is a summary of the main criteria to assess pre-eligibility and is not an exhaustive list.

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PROCESS
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If you are interested in learning more and would like to be considered for this Vascular Ehlers-Danlos Syndrome treatment study, please click on the orange “Learn More” button at the top right of the page. This button will take you to a new page where we will verify your name and contact information. We will also ask you a few questions that will determine if you may qualify for the Acer Vascular Ehlers-Danlos Syndrome treatment study with celiprolol. If you pre-qualify, the final step of the sign-up process will be Science 37 asking you to complete our Medical Record Release form so Science 37 can request your medical records to further assess your eligibility for this important study.

If you are eligible to participate in this Vascular Ehlers-Danlos Syndrome treatment study, you will participate entirely from home. You will have study visits where a registered nurse will visit your home and study visits conducted with a registered nurse online through a telehealth visit. You will be provided compensation for study visits completed. You will be provided instructions for taking the investigational drug which will be shipped directly to your home. During your participation in the study you will continue your regularly scheduled visits with your primary doctor(s).

VEDS RELATED FAQ

Vascular Ehlers-Danlos Syndrome (VEDS) is a rare disorder that weakens the body’s connective tissue. The connective tissue is responsible for supporting and strengthening the body’s bones, organs, and other tissues. When this tissue is weakened due to a genetic mutation in the gene COL3A1, it can cause a variety of symptoms and health complications. Type 4 EDS, also known as Vascular EDS (VEDS), is a particularly rare and serious form of EDS that can be life-threatening.

VEDS is caused by a mutation in the COL3A1 gene, which provides instructions for making a protein called type III collagen. This protein is a major component of the connective tissue in blood vessels, skin, and other organs. Mutations in the COL3A1 gene can result in the production of abnormal type III collagen, which can weaken the walls of blood vessels and other tissues, leading to potentially life-threatening complications.
Data on file estimates there are about 5,000 Americans with vEDS, and approximately 2,000 cases are known at this time.

Science 37 is a company that was established in 2014 to help accelerate clinical research and enable more representative participation. They help to operationalize, support, and execute clinical research studies. like the DiSCOVER Trial for Vascular Ehlers-Danlos Syndrome.

The core focus of Science 37 is to make the clinical research experience better for patients — they use a site-less, decentralized approach to conducting research that puts participants first. That’s why they make sure that any part of the research study that can be done remotely comes to you in your home. People join research studies to help move science forward, and their model allows you to spend more time participating in the research, not traveling to a research site.

Research studies help us determine whether new investigational drugs are safe and effective. The government has established many rules to help ensure that research studies are conducted in the most ethical and safe way possible and that participants are protected throughout. Participants receive specific interventions — which may be investigational medications, devices, or procedures— according to a research plan (protocol) developed by the scientists, physicians, and statisticians.

Volunteers are part of the next wave of science and can influence the future of medicine. Advances in research have allowed people to get personalized cancer treatment, be vaccinated for polio, have less invasive tests, manage their diabetes, and countless other developments. These breakthroughs wouldn’t be possible without volunteers. People volunteer to help others by contributing to medical knowledge, getting more involved in their own health care, learning about their condition, joining a community, and getting support. You’ll learn about all the potential benefits and risks of joining the DiSCOVER Trial to evaluate celiprolol to treat Vascular Ehlers-Danlos Syndrome, during the informed consent process.

You can always share information about the research study with your primary doctor or healthcare provider. You should continue to see your doctor throughout the VEDS research study for routine visits and tests. Our study team is available to talk to both you and your doctor if there are any concerns or questions. Working together helps to ensure that other medications or treatments you receive will not be a problem with the research study protocol. However, because these studies are investigational, the result of the study will not be available to individual patients.

There is no cost to participate, and many research studies offer reimbursement for your time and effort. Compensation depends on the research study and is discussed during the informed consent process.

Research studies are designed by a team of scientists, physicians, and statisticians. The duration of a research study varies depending on the protocol that this team develops. When you learn about a research study, you will be informed about the time commitment needed before you agree to participate in the study.

After the first portion of the study is complete, there will be an option to continue in another similar study in which all patients will receive celiprolol. This portion will be similar to the previous part of the study

Patients are enrolled either until 1) they have a primary event (as assessed by the adjudications committee) and they can go into open label and receive celiprolol, 2) we reach statistical significance at their interim analysis after 28 events at ~2 years after the study is fully enrolled, or 3) at the end of the study after 46 events ~ 4 years after the study is fully enrolled.

Science 37 research studies are designed to make participation easier for you. Where permitted, study supplies are shipped directly to your home. If you have any problems with receiving the supplies, you will be able to contact the study team at any time. The Science 37 app on your smartphone will keep you connected to the study team and ensure that you know what to do, and when to do it. The app displays your personalized study calendar and sends you reminders and notifications.

Besides talking to the study staff, you should minimize sharing your experience in the research study with others. We understand that talking about your experience is an important part of feeling supported, but we ask that you not share information about the research study on social media or with other research study participants. This helps to ensure the most valid and highest-quality scientific results. Our study team is always available to address your questions and concerns.

You can leave the research study at any time and for any reason. You are under no obligation to complete a research study or give a reason why you’re leaving one. Although we do our best to make our research studies as easy as possible for you to take part in, we understand that there are many reasons why finishing may not be possible.

After you complete the research study, the study team will talk with you to answer your questions and get your feedback on your overall experience during the trial. As a Science 37 research study participant, you will have access to news and updates about medical discoveries, educational content, and future research opportunities. You will continue to see your local primary doctor or healthcare provider for routine medical care. All participants will be allowed to continue treatment on celiprolol at the end of the study or after experiencing a qualifying event.

The symptoms of Type 4 Vascular Ehlers-Danlos Syndrome can vary from person to person, and some people may have milder symptoms than others. However, there are several common symptoms that are associated with this condition:

  • Aneurysms
  • Ruptured arteries
  • Ruptured intestines
  • Ruptured uterus during pregnancy
  • Collapsed lung
  • Dislocated hip in newborns
  • Family history of Vascular Ehlers-Danlos Syndrome 

Visual markers can be seen across VEDS patients.  These visual VEDS symptoms include:

  • Large eyes that protrude 
  • Eye swelling and abnormal vision
  • Thin nose
  • Receding gums
  • Small jaw
  • Easy bruising
  • Thin pale skin
  • Hands that look older than they are
  • Small joints that are unusually mobile
  • Early onset varicose veins
  • Chronic joint dislocations
  • Club foot

You only need to stop taking beta blockers, but only under the guidance of the prescribing physician. All other meds can be continued while in the study including other blood pressure medications not classified as beta blockers (such as Losarten).

No, you cannot be enrolled in any other clinical trial to be eligible to participate in the DiSCOVER trial.

The study is expected to last 40 months after the study is fully enrolled. After that it may take up to two years to submit the data to the FDA so they can review it and determine if it will be approved for treatment.

You should seek medical treatment as you normally would if you were not enrolled in the study. You can and should inform the ER that you are participating in a clinical trial. If an event occurs, Science 37 will work with an independent review committee to determine if the reason for the ER visit is study related.

Neither you or your doctor will know if you are taking the drug or placebo as the study is double blind.

The trial is designed to enroll 150 patients.

The trial is designed to randomize patients 2 to 1. That means if there are 3 patients in the study, 2 of them will get active drug and only 1 will get placebo. In this trial, 100 patients will receive celiprolol and 50 patients will receive placebo.

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CONTACT FOR THE DiSCOVER TRIAL

If you are interested in learning more and would like to be considered for this study, please click on the orange “Learn More”

For questions about this trial please call/ text 984-253-4376 or email vedstudy@science37.com