New Treatment for Graves' Eye Disease

(OLE Trial)

Recruiting at 51 trial locations

Overseen By John Mandeville, MD

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Viridian Therapeutics, Inc.

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

Disqualifiers: Ear pathology, Hearing loss, others

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?

An open-label study for participants who received VRDN-001 or placebo and were non-responders at end of the treatment period assessment (i.e., 15 weeks) in the VRDN-001-101 (THRIVE) and VRDN-001-301 (THRIVE-2) pivotal studies

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.

How does the new treatment for Graves' Eye Disease differ from existing treatments?

The new treatment for Graves' Eye Disease is unique because it may involve novel approaches like gene therapy or nanotechnology-based strategies, which are emerging fields in ocular treatment. These methods aim to improve drug delivery and effectiveness by overcoming the eye's natural barriers, potentially offering more targeted and efficient treatment compared to traditional methods like eye drops or injections. ^{1 2 3 4 5}

Eligibility Criteria

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

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

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

Trial Timeline

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

Treatment Details

Interventions

• Intervention/Treatment (Other)

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.

Participant Groups

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+

Findings from Research

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]

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]

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]

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]

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]

References

1. France pubmed.ncbi.nlm.nih.gov

[Implementation of a new gene therapy in ophthalmology: Regulatory and organizational issues]. [2021]

2. Netherlands pubmed.ncbi.nlm.nih.gov

Nanotechnology-based strategies for treatment of ocular disease. [2022]

3. England pubmed.ncbi.nlm.nih.gov

Voretigene neparvovec-rzyl for treatment of RPE65-mediated inherited retinal diseases: a model for ocular gene therapy development. [2021]

4. England 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. [2018]

5. United States pubmed.ncbi.nlm.nih.gov

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