Thyroid Eye Disease
Conditions
Keywords
Thyroid Eye Disease, Thyroid-Associated Ophthalmopathy, Dysthyroid Ophthalmopathy, Myopathic Ophthalmopathy, Congestive Ophthalmopathy, Edematous Ophthalmopathy, Infiltrative Ophthalmopathy, Thyroid-Associated Orbitopathy, Graves Disease, Graves Eye Disease, Graves Orbitopathy
Brief 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 TED. The primary objective of this clinical trial is to evaluate the safety and tolerability of VRDN-001 in participants with TED.
Interventions
DRUGVeligrotug
5 infusions of veligrotug
Sponsors
Viridian Therapeutics, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Intervention model description
A randomized, controlled, safety and tolerability study of VRDN-001, a humanized monoclonal antibody directed against the IGF-1 receptor, in participants with thyroid eye disease (TED).
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
* Have a clinical diagnosis of TED with or without proptosis and with any clinical activity score (CAS) (0 - 7) and in the opinion of the investigator may benefit from treatment * Must agree to use highly effective contraception method as specified in the protocol * Female TED participants must have a negative serum pregnancy test at screening * Not require immediate ophthalmological or orbital surgery in the study eye for any reason.
Exclusion criteria
* Must not have received prior treatment with another anti-IGF-1R therapy * Must not have used systemic corticosteroids, or selenium within 2 weeks prior to Day 1 * Must not have received rituximab, tocilizumab or other immunosuppressive agents within 8 weeks prior to Day 1 * Must not have received any other therapy for TED within 8 weeks prior to Day 1 * Must not have received an investigational agent for any condition within 8 weeks prior to the first dose of study medication * Must not have a pre-existing ophthalmic condition in the study eye that in the opinion of the Investigator would confound interpretation of the study results * Must not have had previous orbital irradiation or decompression surgery involving excision of fat for TED in the study eye's orbit * Must not have inflammatory bowel disease * Must not have abnormal hearing test before first dose. Must also not have a history of ear conditions considered significant by study doctor. * Female TED participants must not be pregnant or lactating.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) Through Week 15 | Baseline through Week 15 | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Proptosis in the Study Eye as Measured by Exophthalmometer | Baseline, Week 15 | 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. |
| Number of Participants With TEAEs Through Week 52 | Baseline through Week 52 | 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. |
Countries
Australia, France, Germany, Poland, Spain, United Kingdom, United States
Participant flow
Recruitment details
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.
Pre-assignment details
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.
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Continuous | 49.4 years STANDARD_DEVIATION 12.95 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 13 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 132 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 7 Participants |
| Race/Ethnicity, Customized Race American Indian or Alaska Native | 1 Participants |
| Race/Ethnicity, Customized Race Asian | 21 Participants |
| Race/Ethnicity, Customized Race Black or African American | 22 Participants |
| Race/Ethnicity, Customized Race Multiple | 4 Participants |
| Race/Ethnicity, Customized Race Not Reported | 6 Participants |
| Race/Ethnicity, Customized Race Other | 3 Participants |
| Race/Ethnicity, Customized Race Unknown | 3 Participants |
| Race/Ethnicity, Customized Race White | 39 Participants |
| Sex: Female, Male Female | 46 Participants |
| Sex: Female, Male Male | 41 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 173 | 0 / 58 |
| other Total, other adverse events | 142 / 173 | 47 / 58 |
| serious Total, serious adverse events | 10 / 173 | 3 / 58 |
Outcome results
None listed