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: VRDN-003; 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 may be eligible to receive additional subcutaneous injections of VRDN-003.

Study Timeline

• Study Start: 2024-08-27
• Study First Submitted: 2024-10-01
• Study First Submitted QC: 2024-10-02
• Study First Posted: 2024-10-03
• Status Verified: 2025-01
• Last Update Submitted: 2025-01-06
• Last Update Posted: 2025-01-08
• Primary Completion: 2026-03-01
• Study Completion: 2026-11-15

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 84

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 ≥ 4 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

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Exclusion Criteria

• Must not have received prior treatment with another anti-IGF-1R therapy
• Must not have received systemic corticosteroids for any condition, including TED, 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 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 non-responders who may receive additional injections of VRDN-003. These are described in the protocol.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
VRDN-003 every 4 weeks	VRDN-003	6 subcutaneous administrations of VRDN-003
VRDN-003 every 8 weeks	VRDN-003	3 subcutaneous administrations of VRDN-003 and 3 subcutaneous administrations of placebo
VRDN-003 every 8 weeks	Placebo	3 subcutaneous administrations of VRDN-003 and 3 subcutaneous administrations of placebo
Placebo every 4 weeks	Placebo	6 subcutaneous administrations of placebo

Primary Outcome Measures

Name	Time	Method
Proptosis Responder Rate in the study eye	At 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

Name	Time	Method
Change from baseline in proptosis in the study eye	At Week 24	Change from baseline in proptosis in the study eye
Clinical Activity Responder Rate in the study eye	At Week 24	Clinical Activity Responder Rate in the study eye (i.e., ≥ 2 points reduction in CAS from baseline in the study eye \[without a corresponding increase of ≥2 points in the fellow eye\])
Diplopia Responder Rate for participants with baseline Diplopia Score greater than 0	At Week 24	Proportion of participants with a reduction in Diplopia Score of ≥1 from baseline (for participants with baseline Diplopia Score greater than 0
Diplopia Resolution Rate for participants with baseline Diplopia Score greater than 0	At Week 24	Proportion of participants with a reduction in Diplopia Score to 0 from baseline (for participants with baseline Diplopia Score greater than 0)
Overall Responder Rate in the study eye	At 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 eye	At 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 eye	At Week 24	Proportion of participants with a Clinical Activity Score (CAS) of 0 or 1 in the study eye

Trial Locations

Locations (17)

Marvel Clinical Research; 🇺🇸 Huntington Beach, California, United States

United Medical Research Institute; 🇺🇸 Inglewood, California, United States

Pasadena Clinical Trials; 🇺🇸 Pasadena, California, United States

Ilumina Medical Research; 🇺🇸 Kissimmee, Florida, United States

Anmed Health Services LLC; 🇺🇸 Miami, Florida, United States

Agile Clinical Research Trials, LLC; 🇺🇸 Atlanta, Georgia, United States

Gulf Coast Clinical Trials; 🇺🇸 Houston, Texas, United States

Advancing Research International, LLC; 🇺🇸 Los Angeles, California, United States

A.P.J. Office; 🇺🇸 Newport Beach, California, United States

Senta Clinic; 🇺🇸 San Diego, California, 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

Neuro-Eye Clinical Trials; 🇺🇸 Houston, Texas, United States