A Phase 3 interventional study of Veligrotug in Thyroid Eye Disease, sponsored by Viridian Therapeutics, Inc.. Completed at 45 sites in 8 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-08-14.
Sponsored by Viridian Therapeutics, Inc. · Phase 3, Interventional, and Treatment
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.
191 studies on the registry are indexed under Graves Ophthalmopathy; 58 are open to participants now.
This study's enrollment of 139 is above the median of 64 across 142 interventional studies indexed under Graves Ophthalmopathy.
Browse Graves Ophthalmopathy studies →Viridian Therapeutics, Inc. is the lead sponsor of 9 studies on the registry; 1 is open to participants now.
Of its 5 completed or terminated interventional studies of FDA-regulated products, 4 (80%) have results posted.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Key Exclusion Criteria:
Participants must not:
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.
Drug: Veligrotug
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.
Drug: Veligrotug
Veligrotug 10 mg/kg (5 infusions of Veligrotug 10 mg/kg)
Also known as: VRDN-001
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.
Time frame: Baseline to Week 15
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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: Week 15
As prespecified, data were collected and reported for the participants. Therefore, the 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. The primary and secondary efficacy analyses and safety analysis were measured in the pre-specified study eye per individual participant.
| Milestone | Veligrotug/Veligrotug | Placebo/Veligrotug |
|---|---|---|
| Started | 57 | 82 |
| Received at least 1 dose of study drug | 57 | 82 |
| Completed | 54 | 70 |
| Not completed | 3 | 12 |
| Withdrew: Adverse event | 1 | 2 |
| Withdrew: General or study specific changes | 0 | 1 |
| Withdrew: Lost to follow-up | 0 | 5 |
| Withdrew: Non-compliance | 0 | 2 |
| Withdrew: Withdrawal by subject | 2 | 2 |
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.
| percentage of participants | Veligrotug/Veligrotug | Placebo/Veligrotug |
|---|---|---|
| Proptosis Responder Rate (PRR) in the Most Proptotic Eye as Measured by Exophthalmometer | 21.1 | 51.2 |
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.
| mm | Veligrotug/Veligrotug | Placebo/Veligrotug |
|---|---|---|
| Change From Baseline in Proptosis in the Most Proptotic Eye as Measured by Exophthalmometer | -1.235 ± 1.2113 | -2.418 ± 1.6804 |
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.
| percentage of participants | Veligrotug/Veligrotug | Placebo/Veligrotug |
|---|---|---|
| PRR in the Most Proptotic Eye as Measured by Magnetic Resonance Imaging (MRI)/Computed Tomography (CT) | 3.5 | 41.5 |
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.
| mm | Veligrotug/Veligrotug | Placebo/Veligrotug |
|---|---|---|
| Change From Baseline in Proptosis in the Most Proptotic Eye as Measured by MRI/CT | -0.458 ± 0.7174 | -2.360 ± 1.7035 |
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.
| percentage of participants | Veligrotug/Veligrotug | Placebo/Veligrotug |
|---|---|---|
| Clinical Activity Responder Rate in the Most Proptotic Eye as Measured by Exophthalmometer | 89.5 | 84.1 |
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.
| percentage of participants | Veligrotug/Veligrotug | Placebo/Veligrotug |
|---|---|---|
| ORR Comprising PRR and Clinical Activity Responder Rate in the Most Proptotic Eye as Measured by Exophthalmometer | 19.3 | 48.8 |
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.
| percentage of participants | Veligrotug/Veligrotug | Placebo/Veligrotug |
|---|---|---|
| Diplopia Responder Rate | 36.8 | 34.8 |
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.
| percentage of participants | Veligrotug/Veligrotug | Placebo/Veligrotug |
|---|---|---|
| Diplopia Resolution Rate | 15.8 | 15.2 |
Collected over Baseline up to Week 24. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Veligrotug/Veligrotug | 0/57 (0%) | 0/57 (0%) | 47/57 (82.5%) |
| Placebo/Veligrotug | 0/82 (0%) | 1/82 (1.2%) | 67/82 (81.7%) |
| Event | Veligrotug/Veligrotug | Placebo/Veligrotug |
|---|---|---|
| Vestibular neuronitisInfections and infestations | 0/57 | 1/82 |
| Event | Veligrotug/Veligrotug | Placebo/Veligrotug |
|---|---|---|
| Muscle spasmsMusculoskeletal and connective tissue disorders | 14/57 | 25/82 |
| AmenorrhoeaReproductive system and breast disorders | 2/25 | 6/27 |
| OnychoclasisSkin and subcutaneous tissue disorders | 9/57 | 2/82 |
| Infusion related reactionInjury, poisoning and procedural complications | 1/57 | 9/82 |
| AlopeciaSkin and subcutaneous tissue disorders | 6/57 | 7/82 |
| Ear discomfortEar and labyrinth disorders | 4/57 | 8/82 |
| DiarrhoeaGastrointestinal disorders | 5/57 | 7/82 |
| TinnitusEar and labyrinth disorders | 4/57 | 7/82 |
| FatigueGeneral disorders | 3/57 | 7/82 |
| HeadacheNervous system disorders | 4/57 | 7/82 |
Modified Intent-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug. As prespecified, data were collected and reported for the participants.
| Age, Continuous(years) | Veligrotug/Veligrotug | Placebo/Veligrotug | Total |
|---|---|---|---|
| Mean | 46.2 ± 13.17 | 50.2 ± 12.49 | 48.5 ± 12.88 |
| Sex: Female, Male(Participants) | Veligrotug/Veligrotug | Placebo/Veligrotug | Total |
|---|---|---|---|
| Female | 42 | 61 | 103 |
| Male | 15 | 21 | 36 |
| Ethnicity (NIH/OMB)(Participants) | Veligrotug/Veligrotug | Placebo/Veligrotug | Total |
|---|---|---|---|
| Hispanic or Latino | 11 | 16 | 27 |
| Not Hispanic or Latino | 45 | 62 | 107 |
| Unknown or Not Reported | 1 | 4 | 5 |
| Race/Ethnicity, Customized(Participants) | Veligrotug/Veligrotug | Placebo/Veligrotug | Total |
|---|---|---|---|
| Race — Asian | 2 | 7 | 9 |
| Race — Black or African American | 7 | 4 | 11 |
| Race — White | 42 | 63 | 105 |
| Race — Unknown | 0 | 1 | 1 |
| Race — Not Reported | 3 | 4 | 7 |
| Race — Other | 3 | 3 | 6 |
Documents are hosted by the registry — open the source record to download them.
Plan to share: No
This study is completed, as verified in Jul 2026. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Viridian Therapeutics, Inc.