In short
Clinical trials are studying Vrdn-001 in people with thyroid eye disease, also called TED. These studies look at safety, tolerability, and how well the treatment works, including in chronic TED and in people who did not respond in earlier trials. The trials include both patients with TED and some healthy volunteers.
Key points
- Vrdn-001 clinical trials are studying thyroid eye disease, including chronic TED and TED of any duration. The data include Phase 3 and Phase 4 interventional studies. Most trials focus on safety, tolerability, and efficacy, while also measuring eye bulging and disease activity. Some studies compare Vrdn-001 with placebo, and one open-label study gave access to people who did not respond in earlier trials. The trials also included normal healthy volunteers in one study.
Trial overview
The available studies of Vrdn-001 are all about thyroid eye disease (TED), a condition that affects the eyes and can cause bulging and inflammation. The trial data show that researchers studied Vrdn-001 in people with TED of any duration, people with chronic TED, and in one study, normal healthy volunteers as well as TED patients.
All four trials in the source data were interventional studies, which means the researchers gave a treatment and then compared outcomes. The studies were completed, and they focused on safety, tolerability, and how well Vrdn-001 worked for TED.
Who participated
Most participants were people with TED, including those with chronic thyroid eye disease and those with TED of any duration. One study also included normal healthy volunteers, meaning people without the disease who took part to help researchers learn more about the treatment in a controlled way.
One open-label study was designed for people who were non-responders in earlier pivotal studies, meaning they did not meet the study response goal after the earlier treatment period. That study gave these participants another chance to receive Vrdn-001 and let researchers assess safety and efficacy again.
Trial phases and study design
The source data include Phase 3 and Phase 4 trials. Phase 3 studies are usually larger studies that help confirm whether a treatment works and is safe enough for wider use. Phase 4 studies are done later and can add more information about real-world use and safety.
Several studies compared Vrdn-001 with placebo, which is a treatment without the active study drug. The open-label study was different because participants knew they were receiving the study treatment, which is what open-label means.
The trial schedules in the data often used five intravenous infusions, meaning the treatment was given through a vein several times over the study period. One study also described a dose range of 3.0 to 20.0 mg/kg, while other studies used 10 mg/kg.
What the trials measured
The main safety measures were adverse events (AEs) and serious adverse events (SAEs), which are unwanted medical problems that happen during a study. The studies also tracked any clinically important changes in other safety checks.
Several efficacy outcomes focused on proptosis, which means eye bulging. A common response goal was a reduction of at least 2 mm in the study eye without a similar worsening in the other eye, measured with an exophthalmometer, a tool used to measure how far the eye sticks out.
Some studies also measured Clinical Activity Score (CAS), which shows how active the disease is by looking at signs of inflammation. In the source data, response was often checked 3 weeks after the fifth infusion, which was Week 15.
Key studies in the data
NCT06384547 was a Phase 3 study in 297 people with TED of any duration. It was designed to confirm the safety and tolerability of Vrdn-001 given as five IV infusions of 10 mg/kg every three weeks, and its main outcome was the rate of treatment-emergent adverse events through Week 15.
NCT06021054 was a Phase 3 study in 290 people with chronic TED. It compared Vrdn-001 with placebo and measured an overall response rate based on both proptosis response and clinical activity response at 3 weeks after the fifth infusion, while also tracking safety events.
NCT05176639 was a Phase 4 study in 157 participants, including normal healthy volunteers and people with TED. This study looked at safety, tolerability, efficacy, and also pharmacokinetic and pharmacodynamic profiles, which describe how the body handles the treatment and how the treatment affects the body.
2023-507350-33-00 was an open-label Phase 3 study in 147 people who had not responded in earlier pivotal studies. It provided access to Vrdn-001 again and measured overall response rate and safety outcomes at Week 15.
What these results may mean for patients
These studies show that Vrdn-001 was tested in several groups of people with TED, including those with long-lasting disease and those who had not improved in earlier trials. The research focused not only on whether the treatment could help eye bulging and disease activity, but also on whether it could be given safely.
Because the trials used clear response measures, such as changes in proptosis and CAS, they were designed to show whether Vrdn-001 made a meaningful difference for people with TED. The completed studies give a structured picture of how the treatment was tested in later-stage clinical research.