VRDN-003 for Thyroid Eye Disease

Not currently recruiting at 56 trial locations

Overseen By Stephen Director, Clinical Operations

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Viridian Therapeutics, Inc.

Must not be taking: Corticosteroids, Immunosuppressives, Selenium, others

Disqualifiers: Anti-IGF-1R therapy, Orbital surgery, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 3

Duration: 24 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new drug, VRDN-003, for safety and effectiveness in people with Thyroid Eye Disease (TED), a condition that can cause bulging eyes and discomfort. The trial includes two groups: one receives the drug every 4 weeks, and the other every 8 weeks. Suitable participants have TED, do not require immediate eye surgery, and have not recently used certain other treatments. As a Phase 3 trial, this is the final step before FDA approval, providing participants an opportunity to contribute to potentially groundbreaking treatment.

Will I have to stop taking my current medications?

The trial requires that you stop taking certain medications before joining. You must not have taken systemic corticosteroids or selenium within 2 weeks, or other immunosuppressive drugs or therapies for Thyroid Eye Disease within 8 weeks before the first dose.

Is there any evidence suggesting that VRDN-003 is likely to be safe for humans?

Research has shown that VRDN-003 has been tested for safety in treating Thyroid Eye Disease (TED). In earlier studies, researchers administered VRDN-003 as injections and closely monitored its safety in participants.

The studies found that VRDN-003 was generally well-tolerated, with most participants not experiencing serious side effects. Some experienced mild to moderate side effects, but these were not severe enough to discontinue treatment.

As this treatment is in a late stage of testing, earlier studies provide strong evidence of its safety for humans. If VRDN-003 has already received approval for other conditions, it has passed thorough safety checks. However, for TED, researchers continue to carefully study its safety and effectiveness. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Thyroid Eye Disease?

VRDN-003 is unique because it offers a new approach to treating Thyroid Eye Disease. Unlike standard treatments like corticosteroids or Tepezza, which target inflammation or IGF-1R, VRDN-003 is administered subcutaneously, which could improve convenience and compliance. Additionally, researchers are exploring two dosing schedules—every 4 weeks and every 8 weeks—to see if less frequent dosing is effective, potentially offering more flexibility and fewer side effects. This novel delivery method and flexible dosing schedule make researchers hopeful for better patient outcomes.

What evidence suggests that VRDN-003 might be an effective treatment for Thyroid Eye Disease?

Research shows that VRDN-003 is designed to treat Thyroid Eye Disease (TED) by blocking a specific part of the body involved in the disease's progression. Early studies have shown that VRDN-003 completely blocks the insulin-like growth factor 1 (IGF-1) receptor, which plays a key role in TED. In healthy volunteers, VRDN-003 remained active in the body for about 40-50 days. This suggests it might require fewer injections over time, offering more convenience for patients. While more information is needed, these early results are promising for those with TED. Participants in this trial will receive VRDN-003 either every 4 weeks or every 8 weeks, allowing researchers to evaluate the effectiveness and convenience of different dosing schedules. ^{1 2 3 4 6}

Are You a Good Fit for This Trial?

This trial is for individuals with Thyroid Eye Disease (TED), regardless of the severity. Participants should not need immediate eye surgery and women must test negative for pregnancy. All participants must agree to use effective contraception as outlined in the study.

Inclusion Criteria

I agree to use effective birth control as required.

I am female and have a negative pregnancy test.

I have been diagnosed with thyroid eye disease and might benefit from VRDN-003.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive subcutaneous administrations of VRDN-003 every 4 or 8 weeks

24 weeks

3-6 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

28 weeks

What Are the Treatments Tested in This Trial?

Interventions

• VRDN-003

Trial Overview The trial is testing VRDN-003, a new drug, against a placebo to see how safe it is and how well people can tolerate it when treating TED. It's designed to compare outcomes between those receiving the actual drug and those given a non-active substance.

How Is the Trial Designed?

Group I: VRDN-003 every 8 weeks Experimental Treatment 2 Interventions

3 subcutaneous administrations of VRDN-003 and 3 subcutaneous administrations of placebo

Group II: VRDN-003 every 4 weeks Experimental Treatment 1 Intervention

6 subcutaneous administrations of VRDN-003

Who Is Running the Clinical Trial?

Viridian Therapeutics, Inc.

Lead Sponsor

Trials

7

Recruited

1,200+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06625398

An Efficacy, Safety, and Tolerability Study of VRDN-003 in ... The key objectives of this study are to determine if VRDN-003 is efficacious, safe and tolerable when administered as subcutaneous/SC injections every 4 weeks ...

2. investors.viridiantherapeutics.com investors.viridiantherapeutics.com/news/news-details/2024/Viridian-Therapeutics-Announces-Details-of-Subcutaneous-VRDN-003-Phase-3-Clinical-Program-for-Patients-with-Active-and-Chronic-Moderate-to-Severe-Thyroid-Eye-Disease/default.aspx

Viridian Therapeutics Announces Details of Subcutaneous ... Phase 1 results in healthy volunteers showed a VRDN-003 half-life of 40-50 days which is 4-5x the half-life of VRDN-001.

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11454332/

6461 VRDN-003, a Full Antagonist Antibody to the IGF-1 ... 6461 VRDN-003, a Full Antagonist Antibody to the IGF-1 Receptor for Thyroid Eye Disease (TED): Safety and Pharmacokinetic Results of ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT06812325

A Safety and Tolerability Study of VRDN-003 in ... This is a clinical trial assessing the safety and tolerability of an investigational drug, VRDN-003, in participants with Thyroid Eye Disease (TED).

5. appliedclinicaltrialsonline.com appliedclinicaltrialsonline.com/view/viridian-therapeutics-monoclonal-antibody-thyroid-eye-disease

Viridian Therapeutics Completes Enrollment in Phase III ... VRDN-003 enrollment milestone: Phase III REVEAL trials fully enrolled, positioning the therapy for topline results in early 2026.

6. mayo.edu mayo.edu/research/clinical-trials/cls-20588362?&p=1

A Safety And Tolerability Study Of VRDN-003 In ... This is a clinical trial assessing the safety and tolerability of an investigational drug, VRDN-003, in participants with Thyroid Eye Disease ( ...