Thyroid Eye DiseasePhase 3OLENCT06179875
An Open-label Study (OLE) for Non-responders of VRDN-001-101 and VRDN-001-301
A Phase 3 study testing Veligrotug in people with Thyroid Eye Disease, run by Viridian Therapeutics, Inc. at 45 sites in 8 countries.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Thyroid Eye Disease
- What is being tested?
- Veligrotug
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 139 participants
- Where
- 45 sitesin 8 countries
- Study dates
- 31 Jan 2024to 23 Jun 2025
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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 join
- Participants must not:
- 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
Trial locations and contacts
45 sites in 8 countries.
45 sites in 8 countries
United States18 sites
University of Vermont Medical Center
Burlington, Vermont, 05401
Ophthalmic Consultants of Boston
East Weymouth, Massachusetts, 02189
Baylor College of Medicine
Houston, Texas, 77030
Neuro-Eye Clinical Trials
Houston, Texas, 77074
Ophthalmic Plastic, Reconstructive, Orbital and Cosmetic Surgery
Las Vegas, Nevada, 89144
Kahana Oculoplastic & Orbital Surgery
Livonia, Michigan, 48152
Advancing Research International, LLC
Los Angeles, California, 90023
USC Eye Institute
Los Angeles, California, 90033
University of Miami Miller School of Medicine, Bascom Palmer Eye Institute
Miami, Florida, 33136
Rutgers New Jersey Medical School
Newark, New Jersey, 07103
Advancing Research International, LLC
Newport Beach, California, 90023
Vision Medical Research
Oak Lawn, Illinois, 60453
Stanford Byers Eye Institute
Palo Alto, California, 94303
Hospital of the University of Pennsylvania Perleman Center
Philadelphia, Pennsylvania, 19104
Cockerham Eye Consultants, PC
San Diego, California, 92108
Sarasota Retina Institute
Sarasota, Florida, 34239
University of Washington Medical Center
Seattle, Washington, 98104-2430
The Center for Eye and Facial Plastic Surgery
Somerset, New Jersey, 08873
Germany5 sites
Charite - Universitatsmedizin Berlin KoR, Campus Virchow Klinikum, Klinik tor Augenheilkunde
Berlin
Universitatsklinikum Carl Gustav Carus
Dresden
Universitatsklinikum Essen AoR - Klinik fur Augenheilkunde
Essen
University Medical Center Freiburg
Freiburg im Breisgau
Universitatmedizin Gottingen
Göttingen, Lower Saxony, 37075
Spain5 sites
Hospital Arruzafa. Servicio de Oftalmologia
Córdoba, 14012
Hospital Universitario Ramon y Cajal
Madrid, 28034
Hospital Clinico San Carlos
Madrid
Hospital Universitario Virgen de la Macarena
Seville, 41009
Hospital Universitario Miguel Servel
Zaragoza, 50009
United Kingdom5 sites
University Hospital Bristol and Weston NHS Foundation Trust- Bristol Eye Hospital
Bristol
Imperial College Healthcare NHS Trust Western Eye Hospital
London, NW15QH
Guy's and St. Thomas Trust
London, SE17EH
Newcastle Eye Centre
Newcastle
Norfolk and Norwich University Hospital NHS Foundation Trust
Norwich, London
Poland4 sites
Uniwersytecki Szpital Kliniczny w Bialymstoku
Bialystok, 15-276
Optimum Profesorskie Centrum Okulistyki
Gdansk, 80809
Centrum Medyczne Pulawska
Piaseczno, 05-500
Centrum Medyczne Piasta
Wałbrzych, Piasta, 58-304
Turkey (Türkiye)4 sites
Hacettepe Universitesi Tip Fakultesi
Ankara, 06230
Gazi University Medical Faculty Hospital
Ankara, 06560
Akdeniz University Medical Faculty Hospital
Antalya, 7070
Marmara University Faculty of Medicine
Istanbul, 34899
France3 sites
CHU Angers
Angers, 49933
Centre Hospitalier Universitaire de Montpellier Hopital Lapeyronie
Montpellier, Hérault, 34295
Centre Hospitalier Universitaire De Nantes G.R. Laenne
Saint-Herblain, Loire-Atlantique, 44800
About this study
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
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.
- Conditions
- Thyroid Eye Disease
- Being tested
- Veligrotug
- Keywords
- Graves Disease, Thyroid-Associated Ophthalmopathy, Thyroid Eye Disease, Dysthyroid Ophthalmopathy, Graves Eye Disease, Graves Orbitopathy, Myopathic Ophthalmopathy, Congestive Ophthalmopathy, Edematous Ophthalmopathy, Infiltrative Ophthalmopathy
- Medical subject headings
- Graves Ophthalmopathy, Graves Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VeligrotugDrug
Also called VRDN-001
Veligrotug 10 mg/kg (5 infusions of Veligrotug 10 mg/kg)
| Study group | Role | What they receive |
|---|---|---|
| Veligrotug/Veligrotug | Experimental | 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. Receives: Drug: Veligrotug |
| Placebo/Veligrotug | Experimental | 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. Receives: Drug: Veligrotug |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proptosis Responder Rate (PRR) in the Most Proptotic Eye as Measured by Exophthalmometer
Proptosis response in the most proptotic eye was defined as a reduction of proptosis of ≥2 millimeters (mm) from baseline in the most proptotic eye (without a corresponding increase of ≥2 mm in the other eye) as measured by exophthalmometer. Missing data were not imputed.
Measured: Baseline to Week 15
Secondary outcomes (7)
Change From Baseline in Proptosis in the Most Proptotic Eye as Measured by Exophthalmometer
Proptosis was defined as distance between the lateral orbital rim and the most anterior position of the cornea in mm, measured using an exophthalmometer. Missing data were not imputed.
Measured: Baseline, Week 15
PRR in the Most Proptotic Eye as Measured by Magnetic Resonance Imaging (MRI)/Computed Tomography (CT)
Proptosis responder in the most proptotic eye by MRI/CT was defined as a reduction of proptosis of ≥2 mm from baseline in the most proptotic eye by MRI/CT (without a corresponding increase of ≥2 mm in the other eye) as measured by MRI/CT. Missing data were not imputed.
Measured: Baseline to Week 15
Change From Baseline in Proptosis in the Most Proptotic Eye as Measured by MRI/CT
Measurement of proptosis was conducted by the central imaging reading center using MRI/CT of the orbits acquired without contrast. Measurements were conducted by 2 independent readers with adjudication if the difference between the 2 primary readers in proptosis measurement exceeded 5% (calculated as the difference divided by the larger measurement). The average of 2 (or 3 if adjudicated) measurements were used for analyses. Missing data were not imputed.
Measured: Baseline, Week 15
Clinical Activity Responder Rate in the Most Proptotic Eye as Measured by Exophthalmometer
Clinical activity responder in the most proptotic eye was defined as no worsening in clinical activity score (CAS) from baseline in the most proptotic eye without a corresponding increase of ≥2 points in the other eye. Missing data were not imputed.
Measured: Week 15
ORR Comprising PRR and Clinical Activity Responder Rate in the Most Proptotic Eye as Measured by Exophthalmometer
ORR was comprised of PRR in the most proptotic eye (reduction of proptosis of ≥2 mm from baseline in the most proptotic eye [without a corresponding increase of ≥2 mm in the other eye]) as measured by exophthalmometer at Week 15 and clinical activity responder rate in the most proptotic eye (no worsening in CAS from baseline in the most proptotic eye without a corresponding increase of ≥2 points in the other eye) as measured by exophthalmometer at Week 15. Missing data were not imputed.
Measured: Baseline to Week 15
Diplopia Responder Rate
A diplopia responder was defined as having a decrease of ≥1 from baseline for participants with a baseline Gorman subjective diplopia score >0. Gorman subjective diplopia score (range, 0 to 3) includes 4 categories: no diplopia (absent, scored as 0), diplopia in the primary position of gaze when the participant is tired or awakening (intermittent, scored as 1), diplopia at extremes of gaze (inconstant, scored as 2), and continuous diplopia in the primary or reading position (constant, scored as 3). Missing data were not imputed.
Measured: Week 15
Diplopia Resolution Rate
Diplopia resolution was defined as reduction in Gorman subjective diplopia score to 0 from baseline for participants with baseline Gorman subjective diplopia score >0. Gorman subjective diplopia score (range, 0 to 3) includes 4 categories: no diplopia (absent, scored as 0), diplopia in the primary position of gaze when the participant is tired or awakening (intermittent, scored as 1), diplopia at extremes of gaze (inconstant, scored as 2), and continuous diplopia in the primary or reading position (constant, scored as 3). Missing data were not imputed.
Measured: Week 15
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Dec 2023
- Study start31 Jan 2024
- Primary completion25 Apr 2025
- Study completion23 Jun 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Thyroid Eye DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 139 participants (actual)
Sponsor and investigators
- Lead sponsor
- Viridian Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06179875
- Sponsor's protocol ID
- VRDN-001-302
- Acronym
- OLE
- Status last verified
- Jul 2026
- First submitted
- 12 Dec 2023
- Last updated
- 14 Aug 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06179875
- Acronym / sponsor ID
- OLE / VRDN-001-302
- Conditions
- Thyroid Eye Disease
- Keywords
- Graves Disease, Thyroid-Associated Ophthalmopathy, Thyroid Eye Disease, Dysthyroid Ophthalmopathy, Graves Eye Disease, Graves Orbitopathy, Myopathic Ophthalmopathy, Congestive Ophthalmopathy
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 139 participants (actual)
- Lead sponsor
- Viridian Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 31 Jan 2024 (actual)
- Primary completion
- 25 Apr 2025 (actual)
- Study completion
- 23 Jun 2025 (actual)
- First posted
- 22 Dec 2023 (actual)
- Results
- Posted 14 Aug 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Veligrotug/Veligrotug | Experimental | Drug: VeligrotugNon-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. |
| Placebo/Veligrotug | Experimental | Drug: VeligrotugNon-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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VeligrotugAlso: VRDN-001Veligrotug 10 mg/kg (5 infusions of Veligrotug 10 mg/kg) | Drug | Placebo/Veligrotug, Veligrotug/Veligrotug |
Primary outcome
Proptosis Responder Rate (PRR) in the Most Proptotic Eye as Measured by Exophthalmometer
Time frame: Baseline to Week 15
Proptosis response in the most proptotic eye was defined as a reduction of proptosis of ≥2 millimeters (mm) from baseline in the most proptotic eye (without a corresponding increase of ≥2 mm in the other eye) as measured by exophthalmometer. Missing data were not imputed.
7 secondary outcomes
Change From Baseline in Proptosis in the Most Proptotic Eye as Measured by Exophthalmometer
Time frame: Baseline, Week 15
PRR in the Most Proptotic Eye as Measured by Magnetic Resonance Imaging (MRI)/Computed Tomography (CT)
Time frame: Baseline to Week 15
Change From Baseline in Proptosis in the Most Proptotic Eye as Measured by MRI/CT
Time frame: Baseline, Week 15
Clinical Activity Responder Rate in the Most Proptotic Eye as Measured by Exophthalmometer
Time frame: Week 15
ORR Comprising PRR and Clinical Activity Responder Rate in the Most Proptotic Eye as Measured by Exophthalmometer
Time frame: Baseline to Week 15
Diplopia Responder Rate
Time frame: Week 15
Diplopia Resolution Rate
Time frame: Week 15
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Aug 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
45 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Veligrotug in people with Thyroid Eye Disease, run by Viridian Therapeutics, Inc. at 45 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 45 sites in United States, Germany, Spain, United Kingdom, Poland and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 14 Aug 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proptosis Responder Rate (PRR) in the Most Proptotic Eye as Measured by Exophthalmometer (time frame: Baseline to Week 15). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Aug 2026.