Thyroid Eye Disease
Conditions
Keywords
TED, Thyroid Eye Disease, Graves, Thyroid-Associated Ophthalmopathy, Dysthyroid Ophthalmopathy, Graves Eye Disease, Graves Orbitopathy, Myopathic Ophthalmopathy, Edematous Ophthalmopathy, Infiltrative Ophthalmopathy
Brief summary
This is a clinical trial assessing the efficacy, safety, and tolerability of an investigational drug, VRDN-003, in participants with chronic Thyroid Eye Disease (TED).
Detailed description
This is a randomized (meaning participants will be assigned to study arms by chance), double-masked (meaning study doctor and participant will not know which study arm participant is assigned to), placebo-controlled study that will include participants with chronic TED. The key objectives of this study are to determine if VRDN-003 is efficacious, safe and tolerable when administered as subcutaneous/SC injections every 4 weeks or every 8 weeks compared to placebo in participants with chronic TED. Participants who do not have a meaningful response at Week 24 (irrespective of the initial treatment arm) may be eligible to receive additional subcutaneous injections of VRDN-003.
Interventions
DRUGVRDN-003
VRDN-003 is an investigational, subcutaneously administered, humanized monoclonal antibody directed against the Insulin-like Growth Factor-1 receptor (IGF-1R).
DRUGPlacebo
All participants will receive the same number of injections to maintain masking. Masking will be maintained by the use of placebo injections that appear identical to VRDN-003 injections.
Sponsors
Viridian Therapeutics, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Investigator, Outcomes Assessor)
Masking description
Participants will be randomized to one of the three study arms.
Intervention model description
Participants will be randomized to one of the three study arms.
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Must have moderate to severe chronic TED, with any CAS (0-7) on the 7-item scale, and with documented evidence of ocular symptoms or signs that began greater than 15 months prior to screening * Must agree to use highly effective contraception as specified in the protocol * Female TED participants must have a negative serum pregnancy test at screening Key
Exclusion criteria
* Must not have received prior treatment with another anti-IGF-1R therapy * Must not have received systemic corticosteroids or steroid eye drops for any condition, including TED, or selenium within 2 weeks prior to first dose. * Must not have received other immunosuppressive drugs for any condition, including TED, or any other therapy for TED within 12 weeks prior to first dose * Must not have received an investigational agent for any condition, including TED, within 8 weeks or longer duration (depending on the type of investigational agent) prior to first dose * Must not have received radioactive iodine (RAI) treatment within 8 weeks prior to first dose * Must not have had previous orbital irradiation or decompression surgery for TED to the study eye's orbit * Must not have a pre-existing ophthalmic condition in the study eye which in the study doctor's opinion, would interfere with interpretation of study results * Must not have abnormal hearing test before first dose. Must also not have a history of ear conditions considered significant by study doctor * Must not have a history of inflammatory bowel disease * Female TED participants must not be pregnant or breastfeeding NOTE: There are additional eligibility criteria for participants who do not have a meaningful response at Week 24 (irrespective of initial treatment arm) who may receive additional injections of VRDN-003. These are described in the protocol.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Title: Proptosis Responder Rate in the study eye | At Week 24 | Description: Proportion of participants with a ≥2 mm reduction from baseline in proptosis in the study eye \[without a corresponding increase of ≥2 mm in the fellow eye\] |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from baseline in proptosis in the study eye | At Week 24 | Change from baseline in proptosis in the study eye |
| Overall Responder Rate in the study eye | At Week 24 | Proportion of participants with a ≥2 mm reduction from baseline in proptosis in the study eye \[without a corresponding increase of ≥2 mm in the fellow eye\] AND no worsening in CAS from baseline in the study eye \[without a corresponding increase of ≥2 points in the fellow eye\] |
| Diplopia Responder Rate for participants with baseline Diplopia Score greater than 0 | At Week 24 | Proportion of participants with a reduction in Diplopia Score of ≥1 from baseline (for participants with baseline Diplopia Score greater than 0) |
| Diplopia Resolution Rate for participants with baseline Diplopia Score greater than 0 | At Week 24 | Proportion of participants with a reduction in Diplopia Score to 0 from baseline (for participants with baseline Diplopia Score greater than 0) |
Countries
United States
Outcome results
None listed