Skip to content
Clinical Trials – home

Rare diseases

Investigational molecules

Locations

Our offerNewsroom

Not recruiting

Study of VRDN-001 for Patients with Thyroid Eye Disease Who Did Not Respond to Previous Treatment

Fast replyRegistered drugNo placebo
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition known as Thyroid Eye Disease. This condition can cause the eyes to bulge and become red, swollen, and painful. The study will use a treatment called VRDN-001, which is an experimental medication designed to block a specific protein that may be involved in the disease. The study will also use Sodium Chloride 0.9% Intravenous Infusion, commonly known as normal saline, which is a standard fluid used in medical treatments.

The purpose of this study is to provide access to VRDN-001 for participants who did not respond to previous treatments in earlier studies. The study will assess the safety and effectiveness of VRDN-001 in these participants. Participants will receive the treatment through an intravenous infusion, which means the medication will be given directly into a vein. The study will last for a period of 12 weeks, during which participants will receive several doses of the medication.

Throughout the study, participants will be monitored for any changes in their condition, as well as any side effects they may experience. The study aims to determine if VRDN-001 can help reduce the symptoms of Thyroid Eye Disease in those who have not responded to other treatments. Participants will be closely observed to ensure their safety and to gather information on how well the treatment works.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, ensure understanding of the procedures and risks involved. Written consent is required before any study-related activities begin.

    Eligibility includes having completed at least 5 intravenous infusions in previous studies and being identified as a non-responder.

  2. Step 2

    Initial assessment

    An initial assessment will be conducted to confirm eligibility and establish baseline measurements for the study.

  3. Step 3

    Treatment administration

    Receive VRDN-001, an insulin-like growth factor-1 receptor inhibitor, through intravenous infusion. This is a concentrate for solution for infusion.

    The treatment involves a series of infusions, with the frequency and duration determined by the study protocol.

  4. Step 4

    Monitoring and follow-up

    Regular monitoring will occur to assess the safety and effectiveness of the treatment. This includes tracking any adverse events.

    Assessments will focus on changes in eye protrusion and clinical activity in the most affected eye.

  5. Step 5

    Final assessment

    A final assessment will be conducted to evaluate the overall response to the treatment.

    The primary focus will be on the reduction of eye protrusion and any changes in clinical activity.

Who can join the trial?

7 criteria

  • Must be able to understand the study procedures and the risks involved and must provide written informed consent before the first study-related activity.
  • Must have completed at least 5 intravenous (IV) infusions and assessments required to determine **proptosis** responder status 3 weeks after the fifth IV infusion. **Proptosis** means the bulging of the eye.
  • Must have been a participant in either the VRDN-001-101 or VRDN-001-301 studies and found to be a non-responder, meaning the treatment did not work as expected.
  • Must not require immediate eye or eye socket surgery in the study eye for any reason.
  • If female, must have a negative urine pregnancy test at the start and before each dose of study medication, and agree to use an acceptable method of contraception, such as a condom and another highly effective method, during the study and for 100 days after the last dose.
  • If male and not surgically sterile, must agree to use an acceptable method of contraception, such as a condom and another highly effective method, during the study and for 100 days after the last dose.
  • Must be willing and able to follow all the study requirements for the entire duration of the study.

Who cannot join the trial?

8 criteria

  • Participants who have not previously been treated with the study medication or a placebo in the related studies.
  • Participants who have not shown a lack of response to the treatment after the specified period in the related studies.
  • Participants who have any other medical conditions that might interfere with the study or its results.
  • Participants who are unable to comply with the study requirements or follow-up procedures.
  • Participants who are pregnant or breastfeeding, as the effects on the baby are unknown.
  • Participants who are currently participating in another clinical trial that might interfere with this study.
  • Participants who have a history of allergic reactions to the study medication or similar drugs.
  • Participants who have any serious health conditions that could pose a risk during the study.
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

Not recruiting
Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

Investigated drugs

VRDN-001 is a medication being studied to help people who did not respond to previous treatments in earlier studies. It is given through an intravenous infusion, which means it is delivered directly into the bloodstream through a vein. The goal of this study is to see if VRDN-001 is safe and effective for these participants.

What is already known about the treatment

VRDN-001 – This medication is administered through intravenous infusion and is currently being studied in clinical trials for its effectiveness in treating thyroid eye disease. It is in the experimental phase, with ongoing research to determine its safety and efficacy. The main therapeutic indication for VRDN-001 is thyroid eye disease, a condition that affects the muscles and tissues around the eyes. The medication works by targeting specific molecular pathways involved in the inflammation and swelling associated with this condition. VRDN-001 is classified under investigational drugs, as it is still undergoing clinical evaluation to establish its role in medical treatment.

Investigated diseases

Thyroid eye disease – This condition is an autoimmune disorder that affects the muscles and other tissues around the eyes. It is often associated with an overactive thyroid gland, known as hyperthyroidism. The disease can cause the eyes to bulge or protrude, a condition known as proptosis. Patients may experience symptoms such as redness, swelling, and discomfort in the eyes. As the disease progresses, it can lead to double vision and difficulty moving the eyes. The severity of symptoms can vary, and they may fluctuate over time.
Trial detailsLast updated 4 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-507350-33-00Protocol codeVRDN-001-302Estimated enrolment147 patientsSponsorViridian Therapeutics Inc.

sourced from the EU Clinical Trials Register and site verification

Want to learn more about this trial or check if you can participate?

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.