VRDN-003 for Graves' Disease

Recruiting at 3 trial locations

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: 6

Duration: 24 weeks

75 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial investigates a new treatment called VRDN-003 for individuals with Thyroid Eye Disease (TED), which can cause symptoms like bulging eyes. Researchers aim to assess the safety and effectiveness of VRDN-003 by testing different doses and administration methods. Suitable participants have been diagnosed with TED and do not require immediate eye surgery. The trial excludes those who have recently taken certain medications or treatments for TED. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants the opportunity to contribute to a potentially groundbreaking treatment for TED.

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, other immunosuppressive drugs within 12 weeks, or any investigational agents within 8 weeks before the first dose.

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

Research has shown that VRDN-003 was safely tested in earlier studies. Patients tolerated a single dose well, whether administered through an IV or injected under the skin. The treatment demonstrated promising pharmacokinetics, with a half-life of 40-50 days, indicating that the drug remains in the body longer, which could benefit long-term treatment.

In previous trials, some patients with diabetes experienced changes in blood sugar levels, though these were not directly linked to VRDN-003. Overall, the treatment appears well-tolerated, with few side effects reported. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Graves' Disease?

Researchers are excited about VRDN-003 for Graves' Disease because it offers a novel delivery method that sets it apart from current treatments, which typically involve antithyroid medications, radioactive iodine, or surgery. VRDN-003 is administrated subcutaneously using an autoinjector or a vial and syringe, allowing for a more convenient and potentially more consistent dosing schedule. This treatment is also being tested in different dosing intervals, every 4 or 8 weeks, which could provide greater flexibility and improve patient adherence. By targeting the underlying mechanisms of Graves' Disease with a new approach, VRDN-003 could offer significant benefits over existing therapies.

What evidence suggests that VRDN-003 could be an effective treatment for Graves' Disease?

Research has shown that VRDN-003 might help treat Thyroid Eye Disease (TED). Earlier studies found that just two doses of a similar treatment greatly reduced eye bulging and other symptoms. VRDN-003 blocks a growth factor that causes inflammation and tissue growth in TED. It remains active in the body for about 40-50 days, allowing for fewer doses. In this trial, participants will receive VRDN-003 on different dosing schedules, either every 4 weeks or every 8 weeks, using an autoinjector or vial and syringe. Overall, these early results suggest VRDN-003 could offer significant relief for people with TED. ^{1 2 3 4 6}

Are You a Good Fit for This Trial?

This trial is for individuals with Thyroid Eye Disease (TED), a condition often associated with Graves' disease. 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 agree to use highly effective contraception as specified in the protocol

I have thyroid eye disease and my doctor thinks VRDN-003 could help me.

I don't need eye surgery right now.

Exclusion Criteria

Must not have received an investigational agent/device for any condition, including TED, within 8 weeks or longer (depending on the type of agent/device) prior to first dose

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 or history of ear conditions considered significant by study doctor

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, depending on the assigned group

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

What Are the Treatments Tested in This Trial?

Interventions

• VRDN-003

Trial Overview The trial is testing VRDN-003, an investigational drug delivered via autoinjector, to evaluate its safety, how well it's tolerated by patients, and how the body processes it (pharmacokinetics).

How Is the Trial Designed?

Group I: VRDN-003 every 8 weeks using vial and syringe Experimental Treatment 1 Intervention

3 subcutaneous administrations of VRDN-003 \[1 loading dose of 600mg and 2 doses of 300mg\]

Group II: VRDN-003 every 8 weeks using autoinjector Experimental Treatment 2 Interventions

3 subcutaneous administrations of VRDN-003 \[1 loading dose of 600mg and 2 doses of 300mg\]

Group III: VRDN-003 every 4 weeks using autoinjector Experimental Treatment 2 Interventions

6 subcutaneous administrations of VRDN-003 \[1 loading dose of 600mg and 5 doses of 300mg\]

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. academic.oup.com academic.oup.com/jes/article/8/Supplement_1/bvae163.2209/7812923

6461 VRDN-003, a Full Antagonist Antibody to the IGF-1 ... A single dose of VRDN-003 administered IV or SC to HVs was well tolerated and showed favorable PK for SC administration including a half-life of 40-50 days.

4. 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 ...

5. 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 ...

6. s203.q4cdn.com s203.q4cdn.com/807813999/files/doc_presentations/2024/Jun/VRDN-Corporate-Deck-June-2024-FINAL.pdf

Corporate Presentation VRDN-003 Development Plan Update Both patients with hyperglycemia were diabetic at baseline; in 1 case glucose variability was determined by masked PI to be unrelated to drug. Sources: Viridian ...