Clinical trial NCT06625411: An Efficacy, Safety, and Tolerability Study of VRDN-003 in Participants With Active Thyroid Eye Disease (TED)
Official title: REVEAL-1 - A Phase 3, Randomized, Double-masked, Placebo-controlled, Efficacy, Safety, and Tolerability Study of VRDN-003 in Participants With Active Thyroid Eye Disease (TED)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Thyroid Eye Disease
Keywords: TED
Thyroid Eye Disease
Graves
Thyroid-Associated Ophthalmopathy
Dysthyroid Ophthalmopathy
Graves Eye Disease
Graves Orbitopathy
Myopathic Ophthalmopathy
Congestive Ophthalmopathy
Edematous Ophthalmopathy
Infiltrative Ophthalmopathy
Lead sponsor: Viridian Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2024-10-03
Enrollment: 117
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Participants will be randomized to one of the three study arms.
Primary purpose: TREATMENT

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.

Eligibility criteria: Key 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.

Primary outcomes:
measure: Proptosis Responder Rate in the study eye
timeFrame: At Week 24
Secondary outcomes:
measure: Change from baseline in proptosis in the study eye
timeFrame: At Week 24
measure: Overall Responder Rate in the study eye
timeFrame: At Week 24
measure: Change from baseline in Clinical Activity Score (CAS) in the study eye
timeFrame: At Week 24
measure: Proportion of participants with a Clinical Activity Score (CAS) of 0 or 1 in the study eye
timeFrame: At Week 24
measure: Diplopia Responder Rate for participants with baseline Diplopia Score greater than 0
timeFrame: At Week 24
measure: Diplopia Resolution Rate for participants with baseline Diplopia Score greater than 0
timeFrame: At Week 24
measure: Clinical Activity Responder Rate in the study eye
timeFrame: At Week 24
Other outcomes:
Locations:
city: Scottsdale
country: United States
facility: Scottsdale Clinical Trials
state: Arizona
city: Canoga Park
country: United States
facility: Alliance Research Institute - Canoga Park
state: California
city: Huntington Beach
country: United States
facility: Marvel Clinical Research
state: California
city: Inglewood
country: United States
facility: United Medical Research Institute
state: California
city: Los Angeles
country: United States
facility: Advancing Research International, LLC
state: California
city: Los Angeles
country: United States
facility: Roski Eye Institute, Keck School of Medicine, USC
state: California
city: Lynwood
country: United States
facility: Alliance Research Institute - Lynwood
state: California
city: Newport Beach
country: United States
facility: A.P.J. Office
state: California
city: Palo Alto
country: United States
facility: Byers Eye Institute at Stanford University
state: California
city: Pasadena
country: United States
facility: Pasadena Clinical Trials
state: California
city: San Diego
country: United States
facility: Senta Clinic
state: California
city: Cape Coral
country: United States
facility: C&A Clinical Trials Corp
state: Florida
city: Kissimmee
country: United States
facility: Ilumina Medical Research
state: Florida
city: Miami
country: United States
facility: Med-Care Research
state: Florida
city: Miami
country: United States
facility: Continental Clinical Research
state: Florida
city: Miami
country: United States
facility: Hype Clinical Research LLC
state: Florida
city: Miami
country: United States
facility: Anmed Health Services LLC
state: Florida
city: Sarasota
country: United States
facility: Sarasota Retina Institute
state: Florida
city: Atlanta
country: United States
facility: Agile Clinical Research Trials, LLC
state: Georgia
city: Orland Park
country: United States
facility: Vision Medical Research
state: Illinois
city: East Weymouth
country: United States
facility: Opthalmic Consultants of Boston
state: Massachusetts
city: Fraser
country: United States
facility: Fraser Eye Care Center
state: Michigan
city: Livonia
country: United States
facility: Kahana Oculoplastic & Orbital Surgery
state: Michigan
city: Kansas City
country: United States
facility: University Health
state: Missouri
city: Las Vegas
country: United States
facility: S.L. Office
state: Nevada
city: Sparks
country: United States
facility: Vector Clinical Trials
state: Nevada
city: Newark
country: United States
facility: Rutgers New Jersey Medical School
state: New Jersey
city: Asheville
country: United States
facility: Asheville Clinical Research
state: North Carolina
city: Durham
country: United States
facility: Duke Eye Center
state: North Carolina
city: Houston
country: United States
facility: Baylor College of Medicine/Alkek Eye Center
state: Texas
city: Houston
country: United States
facility: Gulf Coast Clinical Trials
state: Texas
city: Houston
country: United States
facility: Neuro-Eye Clinical Trials
state: Texas
city: Seattle
country: United States
facility: University of Washington, Eye institute
state: Washington
city: Morgantown
country: United States
facility: West Virginia University Eye Institute
state: West Virginia
Study officials:
References: