Open-label study for non-responder participants from VRDN-001-101 and VRDN-001-301 pivotal studies
Research type
Research Study
Full title
An open-label study for participants who are non-responders at the end of treatment assessment on the VRDN-001-101 and VRDN-001-301 pivotal studies
IRAS ID
1008136
Contact name
Barrett Katz
Contact email
Sponsor organisation
Viridian Therapeutics, Inc.
Eudract number
2023-507350-33
Research summary
Thyroid eye disease (TED) is an autoimmune condition commonly associated with overproduction of thyroid hormones. TED causes pain, redness and swelling in the tissues in and around the eyes, leading to bulging of eyes (proptosis), double vision (diplopia), reduced movement of one or both eyes (restricted duction and version), and increased pressure on the nerves in the eyes, eventually followed by loss of vision.
Treatment has historically included vitamins, corticosteroids, topical agents, and antibiotics to address various symptoms of TED. As symptoms become more severe, the only alternatives have historically been surgery and radiation therapy. Biologic therapies have been used to treat TED, but there is little supportive clinical data, and none are licensed for the treatment of TED. Teprotumumab, was recently approved for the treatment of TED in the USA and active TED in Brazil. In clinical trials teprotumumab was more effective than placebo in reducing TED symptoms. While this therapy has been a transformational treatment for patients, it requires eight infusions over 24 weeks, and has been associated with adverse events.
This study, sponsored by Viridian Therapeutics, Inc., will test safety and efficacy of VRDN-001 given as five open-label infusions (all participants receive active drug) over a 12-week treatment period in participants who were found to be non-responders in either the VRDN-001-101 or VRDN-001-301 studies. It is estimated that 143 participants with active or chronic TED will be included across approximately 45 sites in Australia, Canada, China, EU, Israel, Turkey, UK and USA.
Similar to teprotumumab, VRDN-001 is a fully humanized monoclonal antibody (laboratory-produced protein that acts like a human antibody) which targets IGF-1R, a protein present on the surface of eye cells. By blocking IGF-1R, it is thought that VRDN-001 will disrupt inflammation which contributes to TED, thus relieving active and chronic TED.REC name
South Central - Berkshire B Research Ethics Committee
REC reference
24/SC/0003
Date of REC Opinion
1 Feb 2024
REC opinion
Further Information Favourable Opinion