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An Efficacy, Safety, and Tolerability Study of VRDN-003 in Participants with Active Thyroid Eye Disease (TED)

Phase 3
Recruiting
Conditions
Thyroid Eye Disease
Interventions
Drug: Placebo
Registration Number
NCT06625411
Lead Sponsor
Viridian Therapeutics, Inc.
Brief Summary

This is a clinical trial assessing the efficacy, safety, and tolerability of an investigational drug, VRDN-003, in participants with active Thyroid Eye Disease (TED).

Detailed Description

This is a randomized (meaning participants will be assigned to study arms by chance), double-masked (meaning study doctor and participant will not know which study arm participant is assigned to), placebo-controlled study that will include participants with active TED. The key objectives of this study are to determine if VRDN-003 is efficacious, safe and tolerable when administered as a series of subcutaneous/SC injections given every 4 weeks or every 8 weeks compared to placebo in participants with active TED.

Participants who do not have a meaningful response at Week 24 (irrespective of the initial treatment arm) may be eligible to receive additional subcutaneous injections of VRDN-003.

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
117
Inclusion Criteria
  • Must have moderate to severe active TED with documented evidence of ocular symptoms or signs that began within 15 months prior to screening
  • Must have a Clinical Activity Score (CAS) of ≥ 3 on the 7-item scale for the study eye
  • Must agree to use highly effective contraception as specified in the protocol
  • Female TED participants must have a negative serum pregnancy test at screening

Key

Exclusion Criteria
  • Must not have received prior treatment with another anti-IGF-1R therapy
  • Must not have received systemic corticosteroids or steroid eye drops for any condition, including TED, or selenium within 2 weeks prior to first dose
  • Must not have received other immunosuppressive drugs for any condition, including TED, or any other therapy for TED within 12 weeks prior to first dose.
  • Must not have received an investigational agent for any condition, including TED, within 8 weeks or longer duration (depending on the type of investigational agent) prior to first dose
  • Must not have received radioactive iodine (RAI) treatment within 8 weeks prior to first dose
  • Must not have had previous orbital irradiation or decompression surgery for TED to the study eye's orbit
  • Must not have a pre-existing ophthalmic condition in the study eye which in the study doctor's opinion, would interfere with interpretation of study results
  • 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 a history of inflammatory bowel disease
  • Female TED participants who must not be pregnant or breastfeeding

NOTE: There are additional eligibility criteria for participants who do not have a meaningful response at Week 24 (irrespective of initial treatment arm) who may receive additional injections of VRDN-003. These are described in the protocol.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
VRDN-003 every 4 weeksVRDN-0036 subcutaneous administrations of VRDN-003
VRDN-003 every 8 weeksVRDN-0033 subcutaneous administrations of VRDN-003 and 3 subcutaneous administrations of placebo
VRDN-003 every 8 weeksPlacebo3 subcutaneous administrations of VRDN-003 and 3 subcutaneous administrations of placebo
Placebo every 4 weeksPlacebo6 subcutaneous administrations of placebo
Primary Outcome Measures
NameTimeMethod
Proptosis Responder Rate in the study eyeAt Week 24

Proportion of participants with a ≥2 mm reduction from baseline in proptosis in the study eye \[without a corresponding increase of ≥2 mm in the fellow eye\]

Secondary Outcome Measures
NameTimeMethod
Change from baseline in proptosis in the study eyeAt Week 24

Change from baseline in proptosis in the study eye

Diplopia Responder Rate for participants with baseline Diplopia Score greater than 0At Week 24

Proportion of participants with a reduction in Diplopia Score of ≥1 from baseline (for participants with baseline Diplopia Score greater than 0

Clinical Activity Responder Rate in the study eyeAt Week 24

Clinical Activity Responder Rate in the study eye (i.e., ≥ 2 points reduction in CAS from baseline in the study eye \[without a corresponding increase of ≥2 points in the fellow eye\])

Overall Responder Rate in the study eyeAt Week 24

Proportion of participants with a ≥2 mm reduction from baseline in proptosis in the study eye \[without a corresponding increase of ≥2 mm in the fellow eye\]) AND ≥ 2 points reduction in CAS from baseline in the study eye \[without a corresponding increase of ≥2 points in the fellow eye\]

Change from baseline in Clinical Activity Score (CAS) in the study eyeAt Week 24

Change from baseline in Clinical Activity Score (CAS) in the study eye \[range, 0 to 7, with higher scores indicating greater level of inflammation\]

Proportion of participants with a Clinical Activity Score (CAS) of 0 or 1 in the study eyeAt Week 24

Proportion of participants with a Clinical Activity Score (CAS) of 0 or 1 in the study eye

Diplopia Resolution Rate for participants with baseline Diplopia Score greater than 0At Week 24

Proportion of participants with a reduction in Diplopia Score to 0 from baseline (for participants with baseline Diplopia Score greater than 0)

Trial Locations

Locations (31)

Scottsdale Clinical Trials

🇺🇸

Scottsdale, Arizona, United States

Alliance Research Institute - Canoga Park

🇺🇸

Canoga Park, California, United States

Marvel Clinical Research

🇺🇸

Huntington Beach, California, United States

United Medical Research Institute

🇺🇸

Inglewood, California, United States

Advancing Research International, LLC

🇺🇸

Los Angeles, California, United States

Roski Eye Institute, Keck School of Medicine, USC

🇺🇸

Los Angeles, California, United States

Alliance Research Institute - Lynwood

🇺🇸

Lynwood, California, United States

A.P.J. Office

🇺🇸

Newport Beach, California, United States

Byers Eye Institute at Stanford University

🇺🇸

Palo Alto, California, United States

Pasadena Clinical Trials

🇺🇸

Pasadena, California, United States

Senta Clinic

🇺🇸

San Diego, California, United States

Ilumina Medical Research

🇺🇸

Kissimmee, Florida, United States

Med-Care Research

🇺🇸

Miami, Florida, United States

Hype Clinical Research LLC

🇺🇸

Miami, Florida, United States

Anmed Health Services LLC

🇺🇸

Miami, Florida, United States

Sarasota Retina Institute

🇺🇸

Sarasota, Florida, United States

Agile Clinical Research Trials, LLC

🇺🇸

Atlanta, Georgia, United States

Vision Medical Research

🇺🇸

Orland Park, Illinois, United States

Opthalmic Consultants of Boston

🇺🇸

East Weymouth, Massachusetts, United States

Fraser Eye Care Center

🇺🇸

Fraser, Michigan, United States

Kahana Oculoplastic & Orbital Surgery

🇺🇸

Livonia, Michigan, United States

University Health

🇺🇸

Kansas City, Missouri, United States

S.L. Office

🇺🇸

Las Vegas, Nevada, United States

Vector Clinical Trials

🇺🇸

Sparks, Nevada, United States

Rutgers New Jersey Medical School

🇺🇸

Newark, New Jersey, United States

Asheville Clinical Research

🇺🇸

Asheville, North Carolina, United States

Baylor College of Medicine/Alkek Eye Center

🇺🇸

Houston, Texas, United States

Gulf Coast Clinical Trials

🇺🇸

Houston, Texas, United States

Neuro-Eye Clinical Trials

🇺🇸

Houston, Texas, United States

University of Washington, Eye institute

🇺🇸

Seattle, Washington, United States

West Virginia University Eye Institute

🇺🇸

Morgantown, West Virginia, United States

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