An Efficacy, Safety, and Tolerability Study of Veligrotug (VRDN-001), in Participants With Chronic Thyroid Eye Disease (TED)

NCT06021054 | Completed Phase 3 Results DMC FDA Drug

• 1 other identifier: VRDN-001-301
• Study Type: interventional
• Target: 188
• Locations: 9 countries
• Sites: 57

Brief Summary

This is a clinical trial assessing the efficacy, safety, and tolerability of an investigational drug, veligrotug (VRDN-001), in participants with chronic thyroid eye disease (TED).

Conditions

Thyroid Eye Disease

Keywords

Thyroid Eye Disease; Thyroid-Associated Ophthalmopathy; Dysthyroid Ophthalmopathy; Graves Eye Disease; Graves Orbitopathy; Myopathic Ophthalmopathy; Congestive Ophthalmopathy; Edematous Ophthalmopathy; Infiltrative Ophthalmopathy; TED; Thyroid-Associated Orbitopathy; Graves Disease

Outcome Measures

Primary Outcomes (1)

• Proptosis Responder Rate (PRR) in the Most Proptotic Eye as Measured by Exophthalmometer

  Proptosis response in the most proptotic eye was defined as a reduction of proptosis of ≥2 millimeters (mm) from baseline in the most proptotic eye (without a corresponding increase of ≥2 mm in the other eye) as measured by exophthalmometer. Missing data were imputed with the Multiple Imputation (MI) method.

  Baseline to Week 15

Secondary Outcomes (7)

• Change From Baseline in Proptosis in the Most Proptotic Eye as Measured by Exophthalmometer

  Baseline, Week 15

• PRR in the Most Proptotic Eye, as Measured by Magnetic Resonance Imaging (MRI)/Computed Tomography (CT)

  Baseline to Week 15

• Change From Baseline in Proptosis in the Most Proptotic Eye as Measured by MRI/CT

  Baseline, Week 15

• Clinical Activity Responder Rate in the Most Proptotic Eye as Measured by Exophthalmometer

  Week 15

• Overall Responder Rate (ORR) Comprising PRR and Clinical Activity Responder Rate in the Most Proptotic Eye as Measured by Exophthalmometer

  Baseline to Week 15

Study Arms (2)

Veligrotug

EXPERIMENTAL

Participants will receive veligrotug 10 mg/kg as an intravenous (IV) infusion every 3 weeks (Q3W) for 12 weeks.

Drug: Veligrotug

Placebo

EXPERIMENTAL

Participants will receive placebo matched to veligrotug as an IV infusion Q3W for 12 weeks.

Drug: Placebo

Interventions

Placebo DRUG

5 IV Infusions of veligrotug matched placebo

Placebo

Veligrotug DRUG

5 IV Infusions of veligrotug 10 mg/kg

Also known as: VRDN-001

Veligrotug

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Must have moderate to severe chronic TED with documented evidence of ocular symptoms or signs that began greater than 15 months prior to screening
• Must have had a clinical diagnosis of TED, with any CAS (0-7)
• Must agree to use highly effective contraception as specified in the protocol
• Female TED participants must have a negative serum pregnancy test at screening

You may not qualify if:

• Must not have received prior treatment with another anti-IGF-1R therapy
• Must not have received systemic corticosteroids for any condition, including TED, or selenium within 2 weeks prior to first dose
• Must not have received other immunosuppressive drugs or another investigational agent for any condition, including TED, or any other therapy for TED, within 8 weeks prior to first dose
• Must not have received radioactive iodine (RAI) treatment within 8 weeks prior to first dose
• Must not have a pre-existing ophthalmic condition in the study eye that in the opinion of the study doctor would confound interpretation of the study results
• Must not have had previous orbital irradiation or decompression surgery for TED to the study eye's orbit
• Must not have inflammatory bowel disease
• Must not have abnormal baseline audiometry Pure Tone Average (PTA) assessment or history of significant (as determined by the Investigator) ear pathology, relevant ear surgery or hearing loss.
• Female TED participants must not be pregnant or lactating

Sponsors & Collaborators

• Viridian Therapeutics, Inc.: lead

Study Sites (57)

Advancing Research International, LLC

Los Angeles, California, 90023, United States

Location

USC Roski Eye Institute

Los Angeles, California, 90089, United States

Location

Amy Patel Jain, MD

Newport Beach, California, 92660, United States

Location

Stanford Byers Eye Institute

Palo Alto, California, 94303, United States

Location

Cockerham Eye Consultants, PC

San Diego, California, 92108, United States

Location

Bascom Palmer Eye Institute - Ophthalmology - Miami

Miami, Florida, 33136, United States

Location

Sarasota Retina Institute

Sarasota, Florida, 34239, United States

Location

Northwestern University

Chicago, Illinois, 60611, United States

Location

Family Eye Physicians, Ltd

Oak Lawn, Illinois, 60453, United States

Location

Massachusetts Eye and Ear

Boston, Massachusetts, 02114, United States

Location

Ophthalmic Consultants of Boston

Boston, Massachusetts, 02189, United States

Location

Michigan State University, Department of Neurology

East Lansing, Michigan, 48824, United States

Location

Kahana Oculoplastic and Orbital Surgery

Livonia, Michigan, 48152, United States

Location

Washington University School of Medicine in St Louis - Ophthalmology

St Louis, Missouri, 63110, United States

Location

Steven Leibowitz, MD.

Las Vegas, Nevada, 89144, United States

Location

Rutgers - New Jersey Medical School

Newark, New Jersey, 07103, United States

Location

The Center for Eye and Facial Plastic Surgery

Somerset, New Jersey, 08873, United States

Location

Department of Ophthalmology, University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

Wills Eye Hospital

Philadelphia, Pennsylvania, 19107, United States

Location

Baylor College of Medicine (BCM) - Ophthalmology

Houston, Texas, 77030, United States

Location

Neuro Eye Clinical Trials

Houston, Texas, 77074, United States

Location

University of Vermont Medical Center

Burlington, Vermont, 05401, United States

Location

Harborview Medical Center

Seattle, Washington, 98104, United States

Location

North Shore Private Hospital

Saint Leonards, New South Wales, 2065, Australia

Location

CHU Angers

Angers, 49033, France

Location

CHU de Bordeaux - Hôpital Saint-André

Bordeaux, 33000, France

Location

Centre Hospitalier Universitaire De Nantes - G. R. Laennec

Nantes, 44093, France

Location

CH Nice

Nice, 06000, France

Location

Charité - Universitätsmedizin Berlin KöR

Berlin, 13353, Germany

Location

Universitätsklinikum Essen AöR

Essen, 45122, Germany

Location

University Medical Center Freiburg

Freiburg im Breisgau, 79106, Germany

Location

Universitätsmedizin Göttingen

Göttingen, 37075, Germany

Location

Budapest Retina Intezet

Budapest, H-1133, Hungary

Location

Ganglion Orvosi Kozpont

Pécs, H-7621, Hungary

Location

Pecsi Tudomanyegyetem Klinikai Kozpont Szemeszeti Klinika

Pécs, H-7623, Hungary

Location

Uniwersytecki Szpital Kliniczny w Bialymstoku

Bialystok, 15-276, Poland

Location

Oculomedica Sp. z o.o.

Bialystok, 85-157, Poland

Location

Optimum Profesorskie Centrum Okulistyki Sp. z o.o.

Gdansk, 80-809, Poland

Location

Centrum Medyczne Pulawska Sp. z o.o.

Piaseczno, 05-500, Poland

Location

Centrum Medyczne Piasta 47 sp. z o.o.

Wałbrzych, 58-304, Poland

Location

Complexo Hospitalario Universitario de Santiago-Hospital Médico-Cirúrxico de Conxo

A Coruña, 15706, Spain

Location

Centro de Oftalmologia Barraquer (Barraquer Ophthalmology Centre)

Barcelona, 08021, Spain

Location

Hospital La Arruzafa

Córdoba, 14012, Spain

Location

Hospital Universitario Ramón y Cajal

Madrid, 28034, Spain

Location

Hospital Universitario Virgen De La Macarena

Seville, 41009, Spain

Location

Hospital Universitario Virgen del Rocio

Seville, 41013, Spain

Location

Hospital Universitario Y Politécnico La Fe

Valencia, 46026, Spain

Location

Hospital Unviersitario Miguel Servet

Zaragoza, 50009, Spain

Location

Hacettepe Universitesi Tip Fakultesi

Ankara, 06230, Turkey (Türkiye)

Location

Gazi University Medical Faculty Hospital

Ankara, 06500, Turkey (Türkiye)

Location

Akdeniz University Medical Faculty Hospital

Antalya, 07059, Turkey (Türkiye)

Location

Marmara University Faculty of Medicine

Istanbul, 34854, Turkey (Türkiye)

Location

Moorfields Eye Hospital

London, EC1V 2PD, United Kingdom

Location

Imperial College Healthcare NHS Trust - Western eye Hospital

London, NW1 5QH, United Kingdom

Location

St. Thomas' Hospital

London, SE1 7EH, United Kingdom

Location

The Newcastle Upon Tyne Hospitals NHS Foundation Trust

Newcastle, NEI 4LP, United Kingdom

Location

University Hospital Southampton NHS Foundation Trust

Southampton, SO16 6YD, United Kingdom

Location

MeSH Terms

Conditions

Graves Ophthalmopathy; Graves Disease

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Exophthalmos; Orbital Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Goiter; Thyroid Diseases; Endocrine System Diseases; Hyperthyroidism; Autoimmune Diseases; Immune System Diseases

Results Point of Contact

• Title: Viridian Clinical Trials Desk
• Organization: Viridian Therapeutics, Inc.

viridian-clinical-trials@viridiantherapeutics.com

617-272-4609

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 25, 2023
• First Posted: September 1, 2023
• Study Start: November 1, 2023
• Primary Completion: November 4, 2024
• Study Completion: July 25, 2025
• Last Updated: August 13, 2026
• Results First Posted: August 13, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will not share

Locations

TU (4); AU (1); DE (4); GB (5); HU (3); PL (5); ES (8); FR (4); US (23)