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You can help make a difference for yourself
and others living with VEDS

AM I ELIGIBLE?

The goal of the DiSCOVER Trial is to help advance the science of treating VEDS and improve the lives of people living with this condition. And we need your help to make that happen.

We are assessing the medication celiprolol to determine its effectiveness at reducing the occurrence of VEDS-related events. We’re looking for people 15 years of age or older with a confirmed diagnosis of Vascular Ehlers-Danlos Syndrome (VEDS) caused by a COL3A1 mutation to enroll in the DiSCOVER Trial.

Those who join the study can participate from home and can receive compensation for completed telemedicine and in-home visits. Plus, you’ll receive a full vascular screening and confirmation of genetic test results at no cost to you.

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Participate
from Home

The trial is designed to make it as convenient and easy as possible to participate, with telemedicine and in-home visits

Medical Monitoring

Medical
Monitoring

Your health and safety will be closely monitored during the trial—and you will continue to see your regular doctor

Continue Most Current Medications

Continue Most
Current Medications

You can stay on any current, non-beta-blocker medications, including Losartan

Early Access Program

Early Access
Program

At the end of the treatment testing phase, you will have access to the medication before it’s approved in the U.S.

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About the Trial

The DiSCOVER Trial is an opportunity to be part of a scientific process designed to evaluate the safety and effectiveness of celiprolol in an effort to bring a much-needed treatment option for people living with VEDS in the U.S.

What is the medication used in the trial?

The DiSCOVER Trial is assessing the effectiveness of celiprolol, a unique type of beta blocker that is commonly used off-label to treat VEDS in Europe (“off-label” refers to a medication that is prescribed for a purpose different than its officially approved use). Celiprolol has been in clinical use in several countries to treat other conditions for many years. It reduces mechanical stress on, and potentially stabilizes, arterial walls and hollow organs which is why we are investigating its effectiveness at reducing VEDS-related events.

How will my participation make a difference?

By participating in the trial, you will help advance the science of treatment for VEDS, now and in the future. Advancements in treatment are only made possible through trial participants like you. Participation in this study does not guarantee results, but information from this study may benefit other patients with VEDS.

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What will my participation involve?

During your participation in the DiSCOVER Trial, you will continue to see your regular doctor and, after completing a vascular system scan, participate from home with monthly telemedicine visits plus occasional in-home visits from a trial nurse. And you can stay on any current medications not classified as beta blockers, including Losartan. You can withdraw from the study at any time.

More details about the trial

Along with helping to advance the science of treating VEDS, there are other good reasons to participate in the trial:

  • Two-thirds of participants will have access to celiprolol—a medication commonly used off-label to treat VEDS in Europe and not available in the U.S.—at no cost to you.
  • You only have a one-third chance of receiving the placebo. The trial is double-blind, meaning neither you nor your doctor will know if you are receiving the study drug or placebo. If you experience an event while on the placebo, you will be able to receive celiprolol for the remainder of the trial.
  • At the end of the treatment testing phase, you will have access to the medication before it’s approved in the U.S. as part of the open label extension.
  • Participants also get a full vascular system scan at no cost, which can be scheduled at a convenient time and location. Study-related costs will be covered for participants.
  • Participants will receive monetary compensation for the screening and monthly visits while participating in the trial.
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Who can join the DiSCOVER Trial?

Pre-qualification requirements include:

  • 15 years of age or older, living in the U.S.
  • Receive a magnetic resonance angiogram (MRA) and genetic test screening (saliva sample) at no cost to you
  • Discontinuation of current beta-blocker treatment, if applicable
  • Have not had an arterial rupture, dissection, uterine rupture, and/or intestinal rupture within the last six months

The DiSCOVER Trial is designed to make it as convenient and easy as possible to participate.  Start by finding out if you meet the requirements to enroll in the trial. Just complete the short, online questionnaire.

AM I ELIGIBLE?

For complete details about trial participation requirements, please contact us at 984-253-4376 or vedstudy@science37.com

What’s next if I’m interested in participating?

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Step 01

Fill out the pre-screener questionnaire to see if you may be eligible to participate.
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Step 02

We will contact you right away to let you know if you pre-qualify for the study.
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Step 03

If you pre-qualify, we will guide you through the next steps and answer any questions you may have.

We encourage you to share information about the research study with your primary doctor or healthcare provider. And always feel free to contact us with any questions at 984-253-4376 or vedstudy@science37.com

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FAQs

At Zevra Therapeutics, we understand that the success of clinical trials depends on the participation and commitment of people like you. We know you have questions about the DiSCOVER Trial, and we have answers to help you learn more.

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.

Study participants 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 should 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 financial 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.

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.

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 continuing participation 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.
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 Losartan).
No, you cannot be enrolled in any other clinical trial to be eligible to participate in the DiSCOVER Trial.
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.
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.

About Us

At Zevra Therapeutics, our goal is to bring life-changing therapeutics to people living with rare diseases. We are a rare disease company leading with science to create transformational therapies for diseases with limited or no treatment options. By making life-changing therapeutics available to individuals with significant unmet needs, our goal is to give every rare disease patient a fighting chance for a better life.

Contact Us

We invite you to reach out to our excellent team of doctors, research and patient experience coordinators who are eager to help guide you through the process.

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

To see if you may be qualified to take part in the study, click the “Am I Eligible?” button.

AM I ELIGIBLE?

THANK YOU for your interest in the DiSCOVER Trial. Together, we share the goal of seeking to improve the lives of people living with VEDS and we appreciate your time and consideration.

The DiSCOVER Trial for VEDS

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

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