New Treatment for Graves' Eye Disease

(OLE Trial)

No longer recruiting at 62 trial locations

Overseen By John Mandeville, MD

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Viridian Therapeutics, Inc.

Must not be taking: Corticosteroids, Rituximab, Tocilizumab, others

Disqualifiers: Ear pathology, Hearing loss, 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: Unknown

Duration: 15 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new drug, VRDN-001, for individuals with Graves' eye disease, also known as thyroid eye disease (TED). The goal is to determine if this drug can reduce eye inflammation and swelling by blocking a specific receptor that contributes to these symptoms. Participants who did not respond to earlier treatments with VRDN-001 are eligible, especially if they have had TED and have not needed surgery. This trial might be suitable for those in this situation. As a Phase 3 trial, it represents the final step before FDA approval, providing a chance to access a potentially effective treatment before it becomes widely available.

Will I have to stop taking my current medications?

The trial requires that you stop taking systemic corticosteroids at least 2 weeks before starting, and you must not have taken certain immunosuppressive agents like rituximab or tocilizumab within 8 weeks before starting. Other medications are not specifically mentioned, so it's best to discuss with the trial team.

Is there any evidence suggesting that VRDN-001 is likely to be safe for humans?

Research has shown that VRDN-001, a treatment for thyroid eye disease, is generally well-tolerated by patients. Studies found that after five treatments, patients experienced improvements in symptoms such as eye bulging and double vision. Importantly, trials reported no serious side effects, suggesting that VRDN-001 is safe for most people. However, individual experiences may vary with any treatment. It is essential to consult a healthcare provider about potential risks before joining a clinical trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

VRDN-001 is unique because it offers a promising new approach for treating Graves' Eye Disease, with researchers particularly excited about its potential mechanism of action. Unlike current treatments like corticosteroids or surgery, which often focus on managing symptoms or physically altering the affected area, VRDN-001 is an experimental monoclonal antibody that targets the underlying inflammatory process of the disease. This targeted approach could lead to more effective and faster symptom relief. Researchers are hopeful that by directly addressing the inflammation at its root, VRDN-001 could offer a more efficient treatment option with potentially fewer side effects compared to traditional therapies.

What evidence suggests that VRDN-001 might be an effective treatment for Graves' eye disease?

In this trial, participants will receive VRDN-001, which has shown promise in previous studies for treating thyroid eye disease (TED) linked to Graves' disease. Research indicates that VRDN-001 can significantly reduce eye bulging, improve clinical activity scores, and fix double vision in patients with TED after just five treatments. These improvements suggest that VRDN-001 effectively reduces inflammation and swelling in the eyes. The studies reported no serious side effects, enhancing the treatment's appeal. Overall, the evidence supports VRDN-001 as a promising option for those with TED. ^{2 3 4 6 7}

Are You a Good Fit for This Trial?

This trial is for people with Graves' Eye Disease who didn't respond to previous treatments in the VRDN-001-101 or VRDN-001-301 studies. Participants must have completed at least 5 infusions, be non-pregnant if female, and agree to use effective contraception.

Inclusion Criteria

I am a woman with Thyroid Eye Disease and not pregnant.

I participated in the VRDN-001-101 or VRDN-001-301 study and did not respond to treatment.

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

Exclusion Criteria

Must not have received an investigational agent for any condition (other than VRDN-001 or placebo associated with the VRDN-001-101 or VRDN-001-301 pivotal studies) within 8 weeks prior to Day 1

I haven't taken rituximab, tocilizumab, or similar drugs in the last 8 weeks.

I have never been treated with anti-IGF-1R or experimental drugs for TED.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive 5 infusions of VRDN-001 10 mg/kg over a 15-week period

15 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

3 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Intervention/Treatment

Trial Overview The study tests a treatment on those who didn't improve after prior therapy for Graves' Ophthalmopathy. It's an open-label trial, meaning both researchers and participants know what treatment is being given.

How Is the Trial Designed?

Group I: VRDN-001 10 mg/kg Experimental Treatment 1 Intervention

5 infusions of VRDN-001 10 mg/kg

Who Is Running the Clinical Trial?

Viridian Therapeutics, Inc.

Lead Sponsor

Trials

7

Recruited

1,200+

Published Research Related to This Trial

Voretigene neparvovec-rzyl (VN) is the first FDA-approved gene therapy for a hereditary genetic disease, specifically targeting RPE65 mutation-associated inherited retinal disease, and has shown significant improvements in visual function through subretinal injection.

Clinical studies demonstrated that VN treatment led to enhanced performance on mobility tests, increased light sensitivity, and improved visual fields, indicating its efficacy in restoring vision in patients with this specific genetic condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Voretigene neparvovec-rzyl for treatment of RPE65-mediated inherited retinal diseases: a model for ocular gene therapy development. Ciulla, TA., Hussain, RM., Berrocal, AM., et al.[2021]

Current treatments for ocular diseases, like eye drops and injections, often face challenges such as low effectiveness and potential side effects, highlighting the need for better delivery methods.

Nanotechnology is emerging as a promising solution for ocular drug delivery, allowing for improved targeting and interaction with specific eye tissues, which could lead to more effective treatments for various eye conditions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Nanotechnology-based strategies for treatment of ocular disease. Weng, Y., Liu, J., Jin, S., et al.[2022]

The conference highlighted promising new treatments for glaucoma and ocular hypertension, including OT-440 and an extended-release brimonidine implant, which could improve patient management of these conditions.

Other innovative therapies discussed include AC-8 for ocular diseases related to HSV infection and fidarestat for corneal neovascularization, showcasing a broad range of research aimed at addressing various eye health issues.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Association for Research in Vision and Ophthalmology (ARVO)--2010 Annual Meeting. For Sight: The Future of Eye and Vision Research--part 2. Hookes, L.[2018]

Voretigene neparvovec (VN) is the first gene therapy approved for treating RPE65-mediated hereditary retinal dystrophy, marking a significant advancement in ophthalmology.

The therapy's European market approval comes with strict regulatory conditions, highlighting the importance of following specific hospital protocols for its safe and effective administration to patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Implementation of a new gene therapy in ophthalmology: Regulatory and organizational issues]. Daruich, A., Boinet, R., Falcou, C., et al.[2021]

The research focuses on understanding the molecular and cellular mechanisms behind ocular diseases, which helps identify potential treatment targets for conditions like ocular hypertension and glaucoma.

Preclinical studies are essential for filing investigational new drug applications, providing necessary safety and efficacy data, and enhancing the quality of research for developing new therapies and devices in ophthalmology.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

iDrugs and iDevices Discovery Research: Preclinical Assays, Techniques, and Animal Model Studies for Ocular Hypotensives and Neuroprotectants. Sharif, NA.[2019]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05176639

NCT05176639 | A Safety, Tolerability and Efficacy Study of ... A Safety, Tolerability and Efficacy Study of Veligrotug (VRDN 001) in Healthy Volunteers and Participants With Thyroid Eye Disease (TED) ( THRIVE ) (THRIVE).

2. investors.viridiantherapeutics.com investors.viridiantherapeutics.com/news/news-details/2024/Viridian-Therapeutics-Announces-Positive-Topline-Results-from-Veligrotug-VRDN-001-Phase-3-THRIVE-Clinical-Trial-in-Patients-with-Active-Thyroid-Eye-Disease/default.aspx

News Details The data showed substantial improvements in proptosis, clinical activity score, and diplopia in patients with TED after just five infusions, and ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT06021054

NCT06021054 | An Efficacy, Safety, and Tolerability Study ... This is a clinical trial assessing the efficacy, safety, and tolerability of an investigational drug, veligrotug (VRDN-001), in participants with chronic ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11182121/

Emerging therapies in the medical management of thyroid ... No patients treated with VRDN-001 achieve completed resolution of diplopia at week six. Moreover, there were no reported serious adverse events, including ...

5. delveinsight.com delveinsight.com/blog/graves-disease-drugs-in-late-stage-development

6 Graves' Disease Drugs in Late-stage Development Preliminary results showed that VRDN-001 treatment significantly reduced proptosis, improved CAS scores, and resolved diplopia. The therapy was ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT06384547

Study Details | NCT06384547 | A Randomized, Controlled, ... The primary objective of this clinical trial is to evaluate the safety and tolerability of VRDN-001 in patients with TED. Detailed Description. A randomized, ...

7. viridiantherapeutics.com viridiantherapeutics.com/pipeline/ted-programs/

Thyroid Eye Disease Programs - Viridian Therapeutics, Inc. veligrotug (VRDN-001) acts as a full antagonist of IGF-1R, and based on clinical data to date, has demonstrated robust clinical activity and was generally well ...