An Open-label Study (OLE) for Non-responders of VRDN-001-101 and VRDN-001-301
Key facts
- Who runs it
- Viridian Therapeutics, Inc.
- Where
- Burlington, East Weymouth, Houston, Las Vegas, Livonia, Los Angeles, Miami, Newark, Newport Beach, Oak Lawn, Palo Alto, Philadelphia, San Diego, Sarasota, Seattle, and Somerset, United StatesSee the 18 sitesAlso in 7 other countries, 27 sites
- Trial code
- Status
- Completed
- Phase
- Phase 3
- People taking part
- 139 people enrolled
- Started
- 31 Jan 2024
- Ended
- 23 Jun 2025
- Type of study
- Interventional, 2 groups
- Treatments tested
- Veligrotug
- Record updated
- 14 August 2026
Who can take part
- Age
- 18 years and older
- Sex
- Women and men
- Healthy volunteers
- Cannot take part
The registry criteria, word for word
Show all 14 criteriaShow less
Who can take part
- Be able to understand the study procedures and the risks involved and be willing to provide written informed consent before the first study-related activity
- Have completed at least 5 IV infusions and assessments required to determine proptosis responder status 3 weeks post the fifth IV infusion (i.e., Week 15) as defined in either the VRDN-001-101 or VRDN-001-301 pivotal studies
- Been a participant in either the VRDN-001-101 or VRDN-001-301 studies and found to be a non-responder as defined within the VRDN-001-101 or VRDN-001-301 study
- Not require immediate surgical ophthalmological or orbital surgery in the study eye for any reason
- Must agree to use highly effective contraception as specified in the protocol
- Female TED participants must have a negative urine pregnancy test at screening
- Be willing and able to comply with all the requirements of the protocol for the entire duration of the study
Who cannot take part
- Have received prior treatment with another anti-IGF-1R agent
- Have received systemic corticosteroids for any condition, including TED, or selenium within 2 weeks prior to first dose
- Have received other immunosuppressive drugs or another investigational agent for any condition, including TED (other than VRDN-001 or placebo associated with the VRDN-001-101 or VRDN-001-301 pivotal studies), or any other therapy for TED, within 8 weeks prior to first dose
- Have received radioactive iodine (RAI) treatment within 8 weeks prior to first dose
- Have had previous orbital irradiation or decompression surgery involving excision of fat for TED to the study eye's orbit
- Have abnormal hearing test before first dose. Have a history of ear conditions considered significant by study doctor
- Participants must not:
Where in United States
Also in 7 other countries, 27 sites
- University of Vermont Medical CenterBurlington, United StatesNo longer taking part
- Ophthalmic Consultants of BostonEast Weymouth, United StatesNo longer taking part
- Baylor College of MedicineHouston, United StatesNo longer taking part
- Neuro-Eye Clinical TrialsHouston, United StatesNo longer taking part
- Ophthalmic Plastic, Reconstructive, Orbital and Cosmetic SurgeryLas Vegas, United StatesNo longer taking part
- Kahana Oculoplastic & Orbital SurgeryLivonia, United StatesNo longer taking part
- Advancing Research International, LLCLos Angeles, United StatesNo longer taking part
- USC Eye InstituteLos Angeles, United StatesNo longer taking part
- University of Miami, Miller School of Medicine, Bascom Palmer Eye InstituteMiami, United StatesNo longer taking part
- Rutgers New Jersey Medical SchoolNewark, United StatesNo longer taking part
- Advancing Research International, LLCNewport Beach, United StatesNo longer taking part
- Vision Medical ResearchOak Lawn, United StatesNo longer taking part
- Stanford Byers Eye InstitutePalo Alto, United StatesNo longer taking part
- Hospital of the University of Pennsylvania Perleman CenterPhiladelphia, United StatesNo longer taking part
- Cockerham Eye Consultants, PCSan Diego, United StatesNo longer taking part
- Sarasota Retina InstituteSarasota, United StatesNo longer taking part
- University of Washington Medical CenterSeattle, United StatesNo longer taking part
- The Center for Eye and Facial Plastic SurgerySomerset, United StatesNo longer taking part
About the trial
What the trial wants to find out
The investigational drug, VRDN-001, is a monoclonal antibody that inhibits the activity of a cell surface receptor called insulin-like growth factor-1 receptor (IGF-1R). Inhibition of IGF-1R may help to reduce the inflammation and associated tissue swelling that occurs in participants with thyroid eye disease (TED). The primary objectives of this clinical trial are to provide open-label access to VRDN-001 for participants who were previously non-responders at 3 weeks post the fifth IV infusion (i.e., 15 weeks) in the VRDN-001-101 (THRIVE) and VRDN-001-301 (THRIVE-2) pivotal studies and assess the safety and efficacy of VRDN-001 in participants who were previously treated with VRDN-001 or placebo.
The 2 groups in the trial
- Veligrotug/VeligrotugTreatment being tested
Non-responder participants who received veligrotug in the parent Study VRDN-001-101 or VRDN-001-301 will receive veligrotug 10 milligrams (mg)/kilogram (kg) every 3 weeks (Q3W) for 12 weeks.
Treatment: Veligrotug
- Placebo/VeligrotugTreatment being tested
Non-responder participants who received placebo in the parent Study VRDN-001-101 or VRDN-001-301 will receive veligrotug 10 mg/kg Q3W for 12 weeks.
Treatment: Veligrotug
Data and sources
- Registry record
- Listed on ClinicalTrials.gov.
- Official 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
- Record updated
- 14 August 2026