Trial Outcomes & Findings for A Randomized, Controlled, Safety and Tolerability Study of VRDN-001 in Participants With Thyroid Eye Disease (TED) (NCT NCT06384547)

Last Updated: 2026-08-13

Results Overview

An adverse event (AE) was defined as any untoward medical occurrence that developed or worsened in severity during the conduct of a clinical study and did not necessarily have a causal relationship to study drug. TEAE was defined as an AE occurring on or after the day of the first dose of study medication through the end of the study. A summary of all Serious Adverse Events (SAEs) and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

231 participants

Primary outcome timeframe

Baseline through Week 15

Results posted on

2026-08-13

Participant Flow

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 safety analyses and secondary efficacy analysis were measured in the pre-specified study eye per individual participant.

Ocular assessments were performed in both eyes at baseline. Study eye was the most proptotic eye by exophthalmometer at baseline. If both eyes were equally proptotic, then the eye with the worse visual acuity (VA) was designated as the study eye. If proptosis and VA were equal in both eyes, then the right eye was designated as the study eye.

Participant milestones

Participant milestones
Measure	Veligrotug 10 mg/kg Participants received 5 intravenous (IV) infusions of veligrotug 10 milligrams (mg)/kilogram (kg) at 3-week intervals for 12 weeks.	Veligrotug 3 mg/kg Participants received 5 IV infusions of veligrotug 3 mg/kg at 3-week intervals for 12 weeks.
Overall Study STARTED	173	58
Overall Study Received at Least 1 Dose of Study Drug	173	58
Overall Study COMPLETED	141	46
Overall Study NOT COMPLETED	32	12

Reasons for withdrawal

Reasons for withdrawal
Measure	Veligrotug 10 mg/kg Participants received 5 intravenous (IV) infusions of veligrotug 10 milligrams (mg)/kilogram (kg) at 3-week intervals for 12 weeks.	Veligrotug 3 mg/kg Participants received 5 IV infusions of veligrotug 3 mg/kg at 3-week intervals for 12 weeks.
Overall Study Adverse Event	4	2
Overall Study Progressive disease	0	1
Overall Study Withdrawal by Subject	14	5
Overall Study Failure to adhere to protocol requirements	3	1
Overall Study Lost to Follow-up	11	2
Overall Study Other Than Specified	0	1

Baseline Characteristics

A Randomized, Controlled, Safety and Tolerability Study of VRDN-001 in Participants With Thyroid Eye Disease (TED)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Veligrotug 10 mg/kg n=173 Participants Participants received 5 IV infusions of veligrotug 10 mg/kg at 3-week intervals for 12 weeks.	Veligrotug 3 mg/kg n=58 Participants Participants received 5 IV infusions of veligrotug 3 mg/kg at 3-week intervals for 12 weeks.	Total n=231 Participants Total of all reporting groups
Age, Continuous	49.1 years STANDARD_DEVIATION 12.99 • n=1 Participants	50.3 years STANDARD_DEVIATION 12.89 • n=1 Participants	49.4 years STANDARD_DEVIATION 12.95 • n=1 Participants
Sex: Female, Male Female	132 Participants n=1 Participants	46 Participants n=1 Participants	178 Participants n=1 Participants
Sex: Female, Male Male	41 Participants n=1 Participants	12 Participants n=1 Participants	53 Participants n=1 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	31 Participants n=1 Participants	13 Participants n=1 Participants	44 Participants n=1 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	132 Participants n=1 Participants	38 Participants n=1 Participants	170 Participants n=1 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	10 Participants n=1 Participants	7 Participants n=1 Participants	17 Participants n=1 Participants
Race/Ethnicity, Customized Race · Asian	17 Participants n=1 Participants	4 Participants n=1 Participants	21 Participants n=1 Participants
Race/Ethnicity, Customized Race · Black or African American	16 Participants n=1 Participants	6 Participants n=1 Participants	22 Participants n=1 Participants
Race/Ethnicity, Customized Race · White	119 Participants n=1 Participants	39 Participants n=1 Participants	158 Participants n=1 Participants
Race/Ethnicity, Customized Race · American Indian or Alaska Native	1 Participants n=1 Participants	0 Participants n=1 Participants	1 Participants n=1 Participants
Race/Ethnicity, Customized Race · Unknown	3 Participants n=1 Participants	0 Participants n=1 Participants	3 Participants n=1 Participants
Race/Ethnicity, Customized Race · Other	5 Participants n=1 Participants	3 Participants n=1 Participants	8 Participants n=1 Participants
Race/Ethnicity, Customized Race · Not Reported	8 Participants n=1 Participants	6 Participants n=1 Participants	14 Participants n=1 Participants
Race/Ethnicity, Customized Race · Multiple	4 Participants n=1 Participants	0 Participants n=1 Participants	4 Participants n=1 Participants

PRIMARY outcome

Timeframe: Baseline through Week 15

Population: Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.

Outcome measures

Outcome measures
Measure	Veligrotug 10 mg/kg n=173 Participants Participants received 5 IV infusions of veligrotug 10 mg/kg at 3-week intervals for 12 weeks.	Veligrotug 3 mg/kg n=58 Participants Participants received 5 IV infusions of veligrotug 3 mg/kg at 3-week intervals for 12 weeks.
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Through Week 15	153 Participants	55 Participants

SECONDARY outcome

Timeframe: Baseline, Week 15

Population: Modified Intent-to-Treat (mITT) population included all participants who received at least 1 IV infusion of study drug. 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. 'Overall number of participants analyzed' = participants evaluable for this outcome measure.

Proptosis was defined as distance between the lateral orbital rim and the most anterior position of the cornea in millimeters (mm), measured using an exophthalmometer. Observed change from baseline in proptosis in study eye is reported. Missing data were not imputed.

Outcome measures

Outcome measures
Measure	Veligrotug 10 mg/kg n=156 Participants Participants received 5 IV infusions of veligrotug 10 mg/kg at 3-week intervals for 12 weeks.	Veligrotug 3 mg/kg n=49 Participants Participants received 5 IV infusions of veligrotug 3 mg/kg at 3-week intervals for 12 weeks.
Change From Baseline in Proptosis in the Study Eye as Measured by Exophthalmometer	-2.3 mm Standard Deviation 1.74	-2.1 mm Standard Deviation 1.42

SECONDARY outcome

Timeframe: Baseline through Week 52

Population: Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.

An AE was defined as any untoward medical occurrence that developed or worsened in severity during the conduct of a clinical study and did not necessarily have a causal relationship to study drug. TEAE was defined as an AE occurring on or after the day of the first dose of study medication through the end of the study. A summary of all SAEs and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.

Outcome measures

Outcome measures
Measure	Veligrotug 10 mg/kg n=173 Participants Participants received 5 IV infusions of veligrotug 10 mg/kg at 3-week intervals for 12 weeks.	Veligrotug 3 mg/kg n=58 Participants Participants received 5 IV infusions of veligrotug 3 mg/kg at 3-week intervals for 12 weeks.
Number of Participants With TEAEs Through Week 52	159 Participants	55 Participants

Adverse Events

Veligrotug 10 mg/kg

Serious events: 10 serious events

Other events: 142 other events

Deaths: 0 deaths

Veligrotug 3 mg/kg

Serious events: 3 serious events

Other events: 47 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Additional Information

Viridian Clinical Trials Desk

Viridian Therapeutics, Inc.

Phone: 617-272-4609

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Veligrotug 10 mg/kg n=173 participants at risk Participants received 5 IV infusions of veligrotug 10 mg/kg at 3-week intervals for 12 weeks.	Veligrotug 3 mg/kg n=58 participants at risk Participants received 5 IV infusions of veligrotug 3 mg/kg at 3-week intervals for 12 weeks.
Cardiac disorders Cardiac failure	0.58% 1/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	0.00% 0/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Endocrine disorders Graves' disease	0.58% 1/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	0.00% 0/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Eye disorders Endocrine ophthalmopathy	0.58% 1/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	0.00% 0/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Eye disorders Optic disc disorder	0.58% 1/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	0.00% 0/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Eye disorders Retinal haemorrhage	0.58% 1/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	0.00% 0/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Eye disorders Retinal tear	0.58% 1/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	0.00% 0/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Gastrointestinal disorders Mesenteric panniculitis	0.00% 0/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	1.7% 1/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Injury, poisoning and procedural complications Infusion related reaction	0.58% 1/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	0.00% 0/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Investigations Amylase increased	0.00% 0/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	1.7% 1/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Investigations Lipase increased	0.00% 0/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	1.7% 1/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Metabolism and nutrition disorders Diabetes mellitus	0.58% 1/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	0.00% 0/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Musculoskeletal and connective tissue disorders Intervertebral disc protrusion	0.58% 1/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	0.00% 0/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Joint neoplasm	0.00% 0/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	1.7% 1/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Nervous system disorders Carotid aneurysm rupture	0.58% 1/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	0.00% 0/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Psychiatric disorders Panic attack	0.58% 1/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	0.00% 0/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.

Measure	Veligrotug 10 mg/kg n=173 participants at risk Participants received 5 IV infusions of veligrotug 10 mg/kg at 3-week intervals for 12 weeks.	Veligrotug 3 mg/kg n=58 participants at risk Participants received 5 IV infusions of veligrotug 3 mg/kg at 3-week intervals for 12 weeks.
Ear and labyrinth disorders Ear discomfort	9.8% 17/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	3.4% 2/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Ear and labyrinth disorders Hypoacusis	2.9% 5/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	5.2% 3/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Ear and labyrinth disorders Tinnitus	12.1% 21/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	13.8% 8/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Eye disorders Eye pain	1.2% 2/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	5.2% 3/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Gastrointestinal disorders Diarrhoea	11.0% 19/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	15.5% 9/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Gastrointestinal disorders Nausea	8.7% 15/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	10.3% 6/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
General disorders Fatigue	13.3% 23/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	19.0% 11/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Infections and infestations Nasopharyngitis	8.1% 14/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	3.4% 2/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Infections and infestations Upper respiratory tract infection	3.5% 6/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	6.9% 4/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Injury, poisoning and procedural complications Infusion related reaction	8.1% 14/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	8.6% 5/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Investigations Blood creatine phosphokinase increased	10.4% 18/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	8.6% 5/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Investigations Blood glucose increased	5.8% 10/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	5.2% 3/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Investigations Gamma-glutamyltransferase increased	4.0% 7/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	5.2% 3/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Investigations Glycosylated haemoglobin increased	4.6% 8/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	6.9% 4/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Musculoskeletal and connective tissue disorders Muscle spasms	44.5% 77/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	29.3% 17/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Nervous system disorders Dizziness	4.0% 7/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	6.9% 4/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Nervous system disorders Headache	17.3% 30/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	8.6% 5/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Reproductive system and breast disorders Amenorrhoea	32.9% 23/70 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	19.2% 5/26 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Skin and subcutaneous tissue disorders Alopecia	17.3% 30/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	10.3% 6/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Skin and subcutaneous tissue disorders Dry skin	13.3% 23/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	8.6% 5/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Skin and subcutaneous tissue disorders Onychoclasis	12.1% 21/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	10.3% 6/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.
Vascular disorders Hypertension	7.5% 13/173 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.	3.4% 2/58 • Baseline through Week 52 Safety Population included all enrolled participants who received at least 1 IV infusion of study drug.