VRDN-003-301: A phase 3, randomized, double-masked, placebo-controlled, efficacy, safety, and tolerability study of VRDN-003 in participants with active thyroid eye disease (TED)
Overview
- Phase
- Phase 3
- Status
- Recruiting
- Enrollment
- 45
- Locations
- 19
- Primary Endpoint
- The primary study endpoint will assess the percentage of participants who have a response called Overall Responder Rate at Week 24. An Overall Responder is a participant who achieves a ≥ 2 mm reduction in eye bulging AND shows a reduction in Clinical Activity Score or CAS (that is an estimate of disease severity/symptoms) ≥ 2 points at Week 24 versus measurements before the first dose.
Overview
Brief Summary
To determine if VRDN-003 is efficacious (meaning if VRDN-003 affects the signs and symptoms of TED), safe and tolerable (meaning if the body reacts acceptably to VRDN-003) when administered as a series of subcutaneous/SC (injection into layer of fat between skin and muscle) injections given every 4 weeks or every 8 weeks compared to placebo (a substance with no active drug ingredients) in participants with active TED
Eligibility Criteria
- Ages
- 18 years to 65+ years (65+ Years, 18-64 Years)
- Accepts Healthy Volunteers
- Yes
Inclusion Criteria
- •Adult males or females, ≥18 to ≤75 years of age who have a clinical diagnosis of TED with a value ≥ 3 on the CAS scale
- •Must have moderate to severe active TED with eye bulging value in the study eye before the first dose as defined in the protocol and at least one additional sign/symptom as described in the protocol
- •Have experienced eye-related signs or symptoms that began within 15 months before the first study visit
- •Must agree to use highly effective contraception as specified in the protocol
- •Female participants must have a negative pregnancy test at first study visit
Exclusion Criteria
- •Received prior treatment with another anti-IGF-1R therapy
- •Received corticosteroids (drugs that reduce inflammation in the body) for any condition, including TED, within 2 weeks prior to first dose
- •Received other immunosuppressive drugs (drugs that dampen the body’s immune response) or any other therapy for TED within 12 weeks prior to first dose or another investigational agent for any condition, including TED, within 8 weeks or longer duration (depending on the type of investigational agent) prior to first dose
- •Have had previous orbital irradiation or decompression surgery for TED or a pre-existing eye condition which in the study doctor’s opinion, would interfere with interpretation of study results
- •Have abnormal hearing test before first dose. History of ear conditions considered significant by study doctor
- •Have inflammatory bowel disease (conditions that involve inflammation of the gastrointestinal [GI] tract)
- •Female participants who are pregnant or breastfeeding
Outcomes
Primary Outcomes
The primary study endpoint will assess the percentage of participants who have a response called Overall Responder Rate at Week 24. An Overall Responder is a participant who achieves a ≥ 2 mm reduction in eye bulging AND shows a reduction in Clinical Activity Score or CAS (that is an estimate of disease severity/symptoms) ≥ 2 points at Week 24 versus measurements before the first dose.
The primary study endpoint will assess the percentage of participants who have a response called Overall Responder Rate at Week 24. An Overall Responder is a participant who achieves a ≥ 2 mm reduction in eye bulging AND shows a reduction in Clinical Activity Score or CAS (that is an estimate of disease severity/symptoms) ≥ 2 points at Week 24 versus measurements before the first dose.
Secondary Outcomes
- The secondary endpoints will assess changes in eye bulging, disease severity and symptoms versus measurements before the first dose, and percent of participants who achieve the required reduction in eye bulging, disease severity and symptoms, and reduction and resolution of double vision at Week 24.
- Safety Endpoint: the study will also evaluate safety events (side effects), the effects of VRDN-003 on the body and vice-versa throughout the study.
Investigators
Chief Medical Officer
Scientific
Viridian Therapeutics S.à.r.l.