VRDN-003 for Graves' Eye Disease

(REVEAL-1 Trial)

Not currently recruiting at 35 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Viridian Therapeutics, Inc.

Must not be taking: Corticosteroids, Immunosuppressives

Disqualifiers: Ophthalmic condition, Ear conditions, IBD, others

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: 6

Duration: 24 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the effectiveness and safety of the investigational drug VRDN-003 for individuals with active Thyroid Eye Disease (TED). TED can cause bulging eyes, double vision, and discomfort. The trial includes three groups: one receiving VRDN-003 every 4 weeks, another every 8 weeks, and a third receiving a placebo. Suitable candidates have moderate to severe TED with eye symptoms that began within the last 15 months. As a Phase 3 trial, this study represents the final step before FDA approval, giving participants the opportunity to contribute to potentially bringing a new treatment to market.

Will I have to stop taking my current medications?

The trial requires that you stop taking certain medications before participating. You must not have taken systemic corticosteroids within 2 weeks or other immunosuppressive drugs within 8 weeks before the first dose.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research shows that VRDN-003 is being tested for safety and tolerability in treating Thyroid Eye Disease (TED). Earlier studies on similar drugs indicate they are usually well-tolerated. For VRDN-003, past studies have reported no serious side effects (unexpected medical problems). This suggests the treatment might be safe. However, these trials are still ongoing, and more information is needed to confirm its safety. Always consult a healthcare provider for personalized advice. ^{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 targets the insulin-like growth factor-1 receptor (IGF-1R), a novel approach for treating Graves' Eye Disease. Most current treatments, such as corticosteroids or orbital decompression surgery, focus on reducing inflammation or alleviating symptoms. However, VRDN-003 aims to address one of the underlying causes of the disease by blocking the action of IGF-1R, which plays a role in the abnormal tissue growth associated with the condition. This targeted mechanism of action offers the potential for more effective and longer-lasting relief with fewer side effects. Researchers are excited because this treatment could significantly improve patient outcomes by providing a more focused and efficient way to manage the disease.

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

Research shows that VRDN-003, a new treatment for Thyroid Eye Disease (TED), holds promise. Earlier studies on similar treatments, such as VRDN-001, have demonstrated significant reductions in symptoms like eye bulging. In this trial, participants will receive VRDN-003 in different dosing schedules: every 4 weeks, every 8 weeks, or a placebo. VRDN-003 is designed to be more effective and remain active in the body longer, potentially requiring fewer doses to achieve improvements. The aim is for VRDN-003 to reduce symptoms and enhance the quality of life for people with TED. ^{1 2 3 4 6}

Are You a Good Fit for This Trial?

This trial is for individuals with active Thyroid Eye Disease (TED), a condition associated with thyroid problems that affects the eyes. Specific eligibility criteria are not provided, but typically participants must meet certain health standards and may be excluded based on factors like other medical conditions or treatments that could interfere with the study.

Inclusion Criteria

Must have a Clinical Activity Score (CAS) of ≥ 4 on the 7-item scale for the study eye

Must agree to use highly effective contraception as specified in the protocol

My eye condition started less than 15 months ago and is moderate to severe.

Exclusion Criteria

Must not have a pre-existing ophthalmic condition in the study eye which in the study doctor's opinion, would interfere with interpretation of study results

Must not have abnormal hearing test before first dose. Must also not have a history of ear conditions considered significant by study doctor

I have not had radioactive iodine treatment in the last 8 weeks.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive subcutaneous injections of VRDN-003 or placebo every 4 or 8 weeks for up to 24 weeks

24 weeks

6 visits (in-person) for every 4 weeks group, 3 visits (in-person) for every 8 weeks group

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Extension

Participants who do not have a meaningful response at Week 24 may receive additional subcutaneous injections of VRDN-003

What Are the Treatments Tested in This Trial?

Interventions

• VRDN-003

Trial Overview The trial is testing VRDN-003, an investigational drug, against a placebo to see if it's effective in treating TED. Participants will either receive VRDN-003 or a placebo without knowing which one they're getting to fairly measure the drug's effects.

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

Group III: Placebo every 4 weeks Placebo Group 1 Intervention

6 subcutaneous administrations of placebo

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/PMC11182121/

Emerging therapies in the medical management of thyroid ... In this study, two infusions led to substantial reductions in proptosis and clinical activity scores. No patients treated with VRDN-001 achieve completed ...

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

A Safety and Tolerability Study of VRDN-003 in ... The key objectives of this study are to determine if VRDN-003 is safe and tolerable when administered as a series of subcutaneous (SC) injections every 4 weeks ...

5. healio.com healio.com/news/endocrinology/20251010/treatment-for-thyroid-eye-disease-in-an-exciting-era-of-growth

Treatment for thyroid eye disease in an exciting era of growth Viridian is also working on a subcutaneous agent called VRDN-003, and it initiated two phase 3 trials in 2024. Bhan said the availability of a ...

6. clinicaltrials.eu clinicaltrials.eu/trial/study-on-the-effects-of-vrdn-003-for-patients-with-active-thyroid-eye-disease/

Study on the Effects of VRDN-003 for Patients with Active ... This study investigates the efficacy and safety of VRDN-003, an injectable treatment under trial for reducing symptoms in patients with active Thyroid Eye ...