An Open-label Study (OLE) for Non-responders of VRDN-001-101 (NCT05176639) and VRDN-001-301 (NCT06021054)

NCT06179875 | Completed Phase 3 DMC FDA Drug

• 1 other identifier: VRDN-001-302
• Study Type: interventional
• Target: 143
• Locations: 8 countries
• Sites: 45

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 objectives of this clinical trial are to provide open-label access to VRDN-001 for participants who were previously non-responders at 3 weeks post the fifth IV infusion (i.e., 15 weeks) in the VRDN-001-101 (THRIVE) and VRDN-001-301 (THRIVE-2) pivotal studies and assess the safety and efficacy of VRDN-001 in participants who were previously treated with VRDN-001 or placebo.

Conditions

Thyroid Eye Disease

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Proptosis Responder Rate in the most proptotic eye

  Proptosis Responder Rate in the most proptotic eye (i.e., reduction of proptosis of ≥ 2 mm from baseline \[without a corresponding increase of ≥ 2 mm in the other eye\]) as measured by exophthalmometer

  Week 15

Secondary Outcomes (7)

• Change from Baseline in proptosis in the most proptotic eye

  Week 15

• Proptosis Responder Rate in the most proptotic eye

  Week 15

• Change from baseline in proptosis in the most proptotic eye

  Week 15

• Clinical Activity Responder Rate in the most proptotic eye

  Week 15

• Overall Responder Rate in the most proptotic eye

  Week 15

Study Arms (1)

Veligrotug 10 mg/kg

EXPERIMENTAL

5 infusions of Veligrotug 10 mg/kg

Drug: Veligrotug

Interventions

Veligrotug DRUG

Veligrotug 10 mg/kg (5 infusions of Veligrotug 10 mg/kg)

Also known as: VRDN-001

Veligrotug 10 mg/kg

Eligibility Criteria

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

You may qualify if:

• Be able to understand the study procedures and the risks involved and be willing to provide written informed consent before the first study-related activity
• Have completed at least 5 IV infusions and assessments required to determine proptosis responder status 3 weeks post the fifth IV infusion (i.e., Week 15) as defined in either the VRDN-001-101 or VRDN-001-301 pivotal studies
• Been a participant in either the VRDN-001-101 or VRDN-001-301 studies and found to be a non-responder as defined within the VRDN-001-101 or VRDN-001-301 study
• Not require immediate surgical ophthalmological or orbital surgery in the study eye for any reason
• Must agree to use highly effective contraception as specified in the protocol
• Female TED participants must have a negative urine pregnancy test at screening
• Be willing and able to comply with all the requirements of the protocol for the entire duration of the study

You may not qualify if:

• Participants must not:
• Have received prior treatment with another anti-IGF-1R agent
• Have received systemic corticosteroids for any condition, including TED, or selenium within 2 weeks prior to first dose
• Have received other immunosuppressive drugs or another investigational agent for any condition, including TED (other than VRDN-001 or placebo associated with the VRDN-001-101 or VRDN-001-301 pivotal studies), or any other therapy for TED, within 8 weeks prior to first dose
• Have received radioactive iodine (RAI) treatment within 8 weeks prior to first dose
• Have had previous orbital irradiation or decompression surgery involving excision of fat for TED to the study eye's orbit
• Have abnormal hearing test before first dose. Have a history of ear conditions considered significant by study doctor

Sponsors & Collaborators

• Viridian Therapeutics, Inc.: lead

Study Sites (45)

Advancing Research International, LLC

Los Angeles, California, 90023, United States

Location

USC Eye Institute

Los Angeles, California, 90033, United States

Location

Advancing Research International, LLC

Newport Beach, 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

University of Miami Miller School of Medicine, Bascom Palmer Eye Institute

Miami, Florida, 33136, United States

Location

Sarasota Retina Institute

Sarasota, Florida, 34239, United States

Location

Vision Medical Research

Oak Lawn, Illinois, 60453, United States

Location

Ophthalmic Consultants of Boston

East Weymouth, Massachusetts, 02189, United States

Location

Kahana Oculoplastic & Orbital Surgery

Livonia, Michigan, 48152, United States

Location

Ophthalmic Plastic, Reconstructive, Orbital and Cosmetic Surgery

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

Hospital of the University of Pennsylvania Perleman Center

Philadelphia, Pennsylvania, 19104, United States

Location

Baylor College of Medicine

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

University of Washington Medical Center

Seattle, Washington, 98104-2430, United States

Location

Centre Hospitalier Universitaire de Montpellier Hopital Lapeyronie

Montpellier, Hérault, 34295, France

Location

Centre Hospitalier Universitaire De Nantes G.R. Laenne

Saint-Herblain, Loire-Atlantique, 44800, France

Location

CHU Angers

Angers, 49933, France

Location

Universitatmedizin Gottingen

Göttingen, Lower Saxony, 37075, Germany

Location

Charite - Universitatsmedizin Berlin KoR, Campus Virchow Klinikum, Klinik tor Augenheilkunde

Berlin, Germany

Location

Universitatsklinikum Carl Gustav Carus

Dresden, Germany

Location

Universitatsklinikum Essen AoR - Klinik fur Augenheilkunde

Essen, Germany

Location

University Medical Center Freiburg

Freiburg im Breisgau, Germany

Location

Amsterdam UMC

Amsterdam, Netherlands

Location

Centrum Medyczne Piasta

Wałbrzych, Piasta, 58-304, Poland

Location

Uniwersytecki Szpital Kliniczny w Bialymstoku

Bialystok, 15-276, Poland

Location

Optimum Profesorskie Centrum Okulistyki

Gdansk, 80809, Poland

Location

Centrum Medyczne Pulawska

Piaseczno, 05-500, Poland

Location

Hospital Arruzafa. Servicio de Oftalmologia

Córdoba, 14012, Spain

Location

Hospital Universitario Ramon y Cajal

Madrid, 28034, Spain

Location

Hospital Clinico San Carlos

Madrid, Spain

Location

Hospital Universitario Virgen de la Macarena

Seville, 41009, Spain

Location

Hospital Universitario Miguel Servel

Zaragoza, 50009, Spain

Location

Hacettepe Universitesi Tip Fakultesi

Ankara, 06230, Turkey (Türkiye)

Location

Gazi University Medical Faculty Hospital

Ankara, 06560, Turkey (Türkiye)

Location

Akdeniz University Medical Faculty Hospital

Antalya, 7070, Turkey (Türkiye)

Location

Marmara University Faculty of Medicine

Istanbul, 34899, Turkey (Türkiye)

Location

Norfolk and Norwich University Hospital NHS Foundation Trust

Norwich, London, United Kingdom

Location

University Hospital Bristol and Weston NHS Foundation Trust- Bristol Eye Hospital

Bristol, United Kingdom

Location

Imperial College Healthcare NHS Trust Western Eye Hospital

London, NW15QH, United Kingdom

Location

Guy's and St. Thomas Trust

London, SE17EH, United Kingdom

Location

Newcastle Eye Centre

Newcastle, United Kingdom

Location

MeSH Terms

Conditions

Graves Ophthalmopathy

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Graves Disease; 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: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Single treatment assignment

Study Record Dates

• First Submitted: December 12, 2023
• First Posted: December 22, 2023
• Study Start: January 31, 2024
• Primary Completion: April 25, 2025
• Study Completion: June 23, 2025
• Last Updated: April 30, 2026

Record last verified: 2026-04

Locations

FR (3); NL (1); US (18); GB (5); PL (4); DE (5); ES (5); TU (4)