A Safety, Tolerability and Efficacy Study of VRDN 001 in Healthy Volunteers and Persons With Thyroid Eye Disease (TED)

Phase 3

Active, not recruiting

• Conditions: Thyroid Eye Disease
• Interventions: Drug: Veligrotug (VRDN-001) Phase 1/2 MAD (HV and TED); Drug: VRDN-001 Phase 3 Cohort (THRIVE); Drug: VRDN-001 Placebo

• Registration Number: NCT05176639
• Lead Sponsor: Viridian Therapeutics, Inc.

Brief Summary

Please note that Phase 1/2 (HV \& MAD) cohort - recruitment is completed and Phase 3 Component (THRIVE) - is actively recruiting.

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). This clinical trial will evaluate the safety, tolerability and pharmacokinetics (the concentration of drug in the blood over time) of VRDN-001 in healthy volunteers and in patients with TED. Study participants with TED will also be evaluated over time for changes in their signs and symptoms of TED compared to their baseline measurements.

Detailed Description

Not available

Study Timeline

• Study Start: 2021-12-03
• Study First Submitted: 2021-12-15
• Study First Submitted QC: 2021-12-15
• Study First Posted: 2022-01-04
• Status Verified: 2024-06
• Last Update Submitted: 2024-06-26
• Last Update Posted: 2024-06-28
• Primary Completion: 2024-07
• Study Completion: 2025-03

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 154

Inclusion Criteria

Not provided

Read More

Exclusion Criteria

Not provided

Read More

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Phase 1/2 MAD (HV and TED)	Veligrotug (VRDN-001) Phase 1/2 MAD (HV and TED)	Healthy volunteers and participants with TED will be randomized to receive two intravenous infusions of veligrotug (VRDN-001) or placebo with an interval of 3
Phase 3 Cohort (THRIVE)	VRDN-001 Phase 3 Cohort (THRIVE)	Participants with TED will be randomized to either veligrotug (VRDN-001) 10mg/kg or placebo.
Phase 3 Cohort (THRIVE)	VRDN-001 Placebo	Participants with TED will be randomized to either veligrotug (VRDN-001) 10mg/kg or placebo.

Primary Outcome Measures

Name	Time	Method
Proptosis Responder Rate in the study eye	Week 6 for TED MAD participants, and Week 15 for Phase 3 participants	Proptosis Responder Rate in the study eye (i.e., reduction of proptosis of ≥ 2 mm from baseline without a corresponding increase of ≥ 2 mm in the fellow eye\] as measured by exophthalmometer)
Incidence of treatment-emergent adverse events	Up to Day 50 for HV MAD

Secondary Outcome Measures

Name	Time	Method
Change from baseline in proptosis in the study eye as measured by exophthalmometer	Week 6 for TED MAD participants, and Week 15 for Phase 3 participants
Proptosis Responder Rate in the most proptotic eye as measured by MRI/CT	Week 15 for Phase 3 participants
Change from baseline in proptosis in the most proptotic eye as measured by MRI/CT	Week 15 for Phase 3 participants
Change from baseline in Clinical Activity Score (CAS)	Week 6 for TED MAD participants, and Week 15 for Phase 3 participants	Each of 7 clinical signs and symptoms of ocular inflammation is scored as present or absent (score of 1 or 0, respectively). The CAS is the sum of the individual scores (range from 0 to 7) where a higher score indicates a greater level of inflammation.
Overall Responder Rate comprising Proptosis Responder Rate as measured by exophthalmometer and Clinical Activity Responder Rate in the study eye	Week 6 for TED MAD participants, and Week 15 for Phase 3 participants
Clinical Activity Responder Rate in the study eye	Week 6 for TED MAD participants, and Week 15 for Phase 3 participants
Diplopia Responder Rate	Week 6 for TED MAD participants, and Week 15 for Phase 3 participants	Diplopia Responder Rate (i.e., reduction in Gorman Subjective Diplopia Score of ≥1 from baseline for participants with baseline Gorman Subjective Diplopia Score \>0)
Diplopia Resolution Rate	Week 6 for TED MAD participants, and Week 15 for Phase 3 participants	Diplopia Resolution Rate (i.e. reduction in Gorman Subjective Diplopia Score to 0 from baseline for participants with baseline Gorman Subjective Diplopia Score \> 0)
Proportion of participants with a CAS of zero or one in the study eye	Week 6 for TED MAD participants, and Week 15 for Phase 3 participants

Trial Locations

Locations (50)

Hospital Lapeyronie; 🇫🇷 Montpellier, France

Hospital Universitario Ramon y Cajal. Ctra. de Colmenar,; 🇪🇸 Madrid, Spain

The Private Office of Raymond Douglas; 🇺🇸 Beverly Hills, California, United States

USC Roski Eye Institute; 🇺🇸 Los Angeles, California, United States

MACRO Trials, Inc.; 🇺🇸 Los Angeles, California, United States

Amy Patel Jain, MD; 🇺🇸 Newport Beach, California, United States

Byers Eye Institute/Stanford University; 🇺🇸 Palo Alto, California, United States

Senta Clinic; 🇺🇸 San Diego, California, United States

University of California, San Francisco; 🇺🇸 San Francisco, California, United States

University of Colorado - Department of Ophthalmology; 🇺🇸 Aurora, Colorado, United States

University of Florida Bascom Palmer Eye Center; 🇺🇸 Miami, Florida, United States

Sarasota Retina Institute; 🇺🇸 Sarasota, Florida, United States

Emory University Eye Center; 🇺🇸 Atlanta, Georgia, United States

Massachusetts Eye and Ear; 🇺🇸 Boston, Massachusetts, United States

Ophthalmic Consultants of Boston; 🇺🇸 East Weymouth, Massachusetts, United States

Michigan State University - Department of Neurology and Ophthalmology; 🇺🇸 East Lansing, Michigan, United States

University of Minnesota Eye Clinic; 🇺🇸 Minneapolis, Minnesota, United States

Washington University St. Louis; 🇺🇸 Saint Louis, Missouri, United States

Rutgers New Jersey Medical School - Department of Ophthalmology & Visual Science; 🇺🇸 Newark, New Jersey, United States

Duke Eye Center; 🇺🇸 Durham, North Carolina, United States

Scheie Eye Institute Dept of OPH University of Pennsylvania; 🇺🇸 Philadelphia, Pennsylvania, United States

Department of Neuro-Ophthalmology Wills Eye Hospital; 🇺🇸 Philadelphia, Pennsylvania, United States

UPMC Eye Center; 🇺🇸 Pittsburgh, Pennsylvania, United States

Eye Wellness Center- Neuro-Eye Clinical Trials, Inc.; 🇺🇸 Bellaire, Texas, United States

Baylor College of Medicine - Alkek Eye Center; 🇺🇸 Houston, Texas, United States

Moran Eye Center; 🇺🇸 Salt Lake City, Utah, United States

University of Washington - Department of Ophthalmology; 🇺🇸 Seattle, Washington, United States

North Shore Private Hospital; 🇦🇺 Saint Leonards, Australia

Oshawa Clinic; 🇨🇦 Oshawa, Ontario, Canada

Ottawa Hospital Research Institute (OHRI); 🇨🇦 Ottawa, Ontario, Canada

Institution: CHU d'Angers; 🇫🇷 Angers, France

Hôpital René et Guillaume Laennec; 🇫🇷 Saint-Herblain, France

Charité Universitaetsmedizin Berlin; 🇩🇪 Berlin, Germany

Klinik und Poliklinik für Augenheilkunde; 🇩🇪 Dresden, Germany

University Medical Center Göttingen; 🇩🇪 Göttingen, Germany

Istituto Auxologico Italiano; 🇮🇹 Milan, Italy

Amsterdam University Medical Center; 🇳🇱 Amsterdam, Netherlands

Erasmus MC; 🇳🇱 Rotterdam, Netherlands

Hospital La Arruzafa Avenida de la Arruzafa; 🇪🇸 Córdoba, Spain

Hospital La Arruzafa; 🇪🇸 Córdoba, Spain

Hospital Clinico San Carlos; 🇪🇸 Madrid, Spain

Hospital Universitario; 🇪🇸 Sevilla, Spain

Hospital Universitario y Politécnico La Fe Servicio Oftalmología; 🇪🇸 Valence, Spain

Hospital Universitario Miguel Servet, Servicio de Paseo Isabel La Catdlica1; 🇪🇸 Zaragoza, Spain

Marmara University Pendik Education and Research Hospital; 🇹🇷 Pendik, Turkey

Gazi Oniversitesi Tip Fakultesi Hastanesi,; 🇹🇷 Yenimahalle, Turkey

Pharmacy trials unit, c/o Pharmacy Stores Level 3 Bristol Royal Infirmary; 🇬🇧 Bristol, United Kingdom

Eye Research Department South Wing St Thomas' Hospital; 🇬🇧 London, United Kingdom

Western Eye Hospital and Charing Cross Hospital; 🇬🇧 London, United Kingdom

Imperial College London; 🇬🇧 London, United Kingdom