A Randomized, Controlled, Safety and Tolerability Study of VRDN-001 in Participants With Thyroid Eye Disease (TED)

NCT06384547 | Completed Phase 3 DMC FDA Drug

• 1 other identifier: VRDN-001-303
• Study Type: interventional
• Target: 231
• Locations: 7 countries
• Sites: 50

Brief Summary

The investigational drug, VRDN-001, is a monoclonal antibody that inhibits the activity of a cell surface receptor called insulin-like growth factor-1 receptor (IGF-1R). Inhibition of IGF-1R may help to reduce the inflammation and associated tissue swelling that occurs in patients with thyroid eye disease (TED). The primary objective of this clinical trial is to evaluate the safety and tolerability of VRDN-001 in patients with TED.

Conditions

Thyroid Eye Disease

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Treatment Emergent Adverse Event (TEAE) incidence rate

  Week 15

Secondary Outcomes (2)

• Change from baseline in proptosis in the study eye as measured by exophthalmometer

  Week 15

• Treatment Emergent Adverse Event (TEAE) incidence rate

  Week 52

Study Arms (2)

VRDN-001 10 mg/kg

EXPERIMENTAL

5 infusions of VRDN-001 10 mg/kg

Drug: VRDN-001 10 mg/kg

5 infusions of VRDN-001 3 mg/kg

EXPERIMENTAL

5 infusions of VRDN-001 3 mg/kg

Drug: VRDN-001 3 mg/kg

Interventions

VRDN-001 10 mg/kg DRUG

5 infusions of VRDN-001 10 mg/kg

VRDN-001 10 mg/kg

VRDN-001 3 mg/kg DRUG

5 infusions of VRDN-001 3 mg/kg

5 infusions of VRDN-001 3 mg/kg

Eligibility Criteria

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

You may qualify if:

• Have a clinical diagnosis of TED with or without proptosis and with any CAS (0 - 7) and in the opinion of the investigator may benefit from treatment
• Must agree to use highly effective contraception method as specified in the protocol
• Female TED participants must have a negative serum pregnancy test
• Not require immediate ophthalmological or orbital surgery in the study eye for any reason

You may not qualify if:

• Must not have received prior treatment with another anti-IGF-1R therapy
• Must not have used systemic corticosteroids, or selenium within 2 weeks prior to Day 1
• Must not have received rituximab, tocilizumab or other immunosuppressive agents within 8 weeks prior to Day 1
• Must not have received any other therapy for TED within 8 weeks prior to Day 1
• Must not have received an investigational agent for any condition within 8 weeks prior to the first dose of study medication
• Must not have a pre-existing ophthalmic condition in the study eye that in the opinion of the Investigator would confound interpretation of the study results
• Must not have had previous orbital irradiation or decompression surgery involving excision of fat for TED in the study eye's orbit
• Must not have inflammatory bowel disease
• Must not have abnormal hearing test before first dose. Must also not have a history of ear conditions considered significant by study doctor.
• Must not have received an investigational agent for any condition within 8 weeks prior to Day 1
• Female TED participants must not be pregnant or lactating

Sponsors & Collaborators

• Viridian Therapeutics, Inc.: lead

Study Sites (50)

United Medical Research Institute

Inglewood, California, 90301, United States

Location

Advancing Research International, LLC

Los Angeles, California, 90023, 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

FOMAT Medical Research

Santa Maria, California, 93454, United States

Location

University of Colorado - Dept of Ophthalmology

Aurora, Colorado, 80045, United States

Location

Sina Medical Center LLC

Homestead, Florida, 33034, United States

Location

Med-Care Research Inc.

Miami, Florida, 33125, United States

Location

Edward Jenner Research Group Center LLC

Plantation, Florida, 33317, United States

Location

Sarasota Retina Institute

Sarasota, Florida, 34231, United States

Location

Vision Medical Research Inc.

Orland Park, Illinois, 60462, United States

Location

University Health Diabetes, Endocrinology & Nephrology Center

Kansas City, Kansas, 64108, United States

Location

KU Medical Center, University of Kansas

Kansas City, Kansas, 66103, United States

Location

Massachusetts Eye and Ear

Boston, Massachusetts, 02114, United States

Location

Ophthalmic Consultants of Boston

Weymouth, Massachusetts, 02189, United States

Location

Fraser Eye Care Center

Fraser, Michigan, 48026, United States

Location

Kahana Oculoplastic & Orbital Surgery

Livonia, Michigan, 48152, United States

Location

University of Minnesota, Department of Ophthalmology and Visual Neurosciences

Minneapolis, Minnesota, 55455, United States

Location

Advancing Research International, LLC

Las Vegas, Nevada, 89144, United States

Location

Rutgers- New Jersey Medical School- Newark

Newark, New Jersey, 07103, United States

Location

New York Eye Ear Infirmary of Mount Sinai

New York, New York, 10003, 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

Neuro-Ophthalmology of Texas, PLLC

Houston, Texas, 77074, United States

Location

University of Vermont Medical Center

Burlington, Vermont, 05401, United States

Location

West Virginia University Eye Institute

Morgantown, West Virginia, 26506, United States

Location

Sydney Eye Hospital

Sydney, 2000, Australia

Location

North Shore Eye Surgery

Sydney, 2065, Australia

Location

CHU d'Angers

Angers, 49100, France

Location

Centre Hospitalier National D'ophtalmologie

Des Quinze Vingts Paris, 75012, France

Location

CH Nice

Nice, 06000, France

Location

AP-HP - Hôpital de la Pitié Salpétrière

Paris, 75013, France

Location

Universitätsmedizin Göttingen

Göttingen, 37075, Germany

Location

Johannes Gutenberg-University Medical Center

Mainz, 55131, Germany

Location

NZOZ E-Vita

Bialystok, 15-879, Poland

Location

Specjalistyczny Osrodek Okulistyczny Oculomedica

Bydgoszcz, 85-870, Poland

Location

Santa Familia PTG Lodz

Lódz, 90-302, Poland

Location

Panstwowy Instytut Medyczny MSWiA

Warsaw, 02-507, Poland

Location

4 Wojskowy Szpital Kliniczny z Poliklinika SP ZOZ

Wroclaw, 53-114, Poland

Location

Clinica Bonanova de Cirugia Ocular

Barcelona, 08022, Spain

Location

Hospital Arruzafa

Córdoba, 14012, Spain

Location

Clínica Universidad de Navarra

Madrid, 28027, Spain

Location

Hospital Universitario Ramón y Cajal

Madrid, 28034, Spain

Location

Clinica Universidad de Navarra

Pamplona, 31008, Spain

Location

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

Santiago de Compostela, A Coruña, 15706, Spain

Location

Hospital Universitario Virgen De La Macarena, Servicio de Oftalmología

Seville, 41071, Spain

Location

Miguel Servet University Hospital, Clinical Trials Unit

Zaragoza, 50009, Spain

Location

Northwick Park Hospital

London, HA1 3UJ, United Kingdom

Location

Western Eye Hospital, Imperial College NHS Trust

London, NW1 5QH, United Kingdom

Location

Guy's and St. Thomas NHS Trust

London, SE1 7EH, 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

Study Design

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

Model Details: A randomized, controlled, safety and tolerability study of VRDN-001, a humanized monoclonal antibody directed against the IGF-1 receptor, in participants with thyroid eye disease (TED).

Study Record Dates

• First Submitted: April 22, 2024
• First Posted: April 25, 2024
• Study Start: May 23, 2024
• Primary Completion: May 1, 2025
• Study Completion: January 19, 2026
• Last Updated: April 30, 2026

Record last verified: 2026-04

Locations

ES (8); GB (3); FR (4); US (26); AU (2); DE (2); PL (5)