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A Safety, Tolerability and Efficacy Study of VRDN 001 in Healthy Volunteers and Persons With Thyroid Eye Disease (TED)
Recruiting
Trial ID: NCT05176639
Purpose
Please note that Phase 1/2 (HV & MAD) cohort - recruitment is completed and Phase 3 Component
(THRIVE) - is actively recruiting.
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
patients with thyroid eye disease (TED). This clinical trial will evaluate the safety,
tolerability and pharmacokinetics (the concentration of drug in the blood over time) of
VRDN-001 in healthy volunteers and in patients with TED. Study participants with TED will
also be evaluated over time for changes in their signs and symptoms of TED compared to their
baseline measurements.
Official Title
A Multiple Ascending Dose (MAD) Safety, Tolerability and Efficacy Study of VRDN 001, a Humanized Monoclonal Antibody Directed Against the IGF-1 Receptor, in Normal Healthy Volunteers (HV(s) and Participants With Thyroid Eye Disease (TED)
Stanford Investigator(s)
Andrea Lora Kossler, MD, FACS
Assoc Professor of Ophthalmology
Eligibility
Key Inclusion Criteria for Healthy Volunteers:
- Must be free of clinically significant disease or medical conditions as determined by
the Investigator
- Female volunteers must not be of child-bearing potential
Key Exclusion Criteria for Healthy Volunteers:
∙ Must not have a history of or any evidence of diabetes mellitus, recently diagnosed renal
impairment or inflammatory bowel disease, or clinically significant ear pathology or earing
impairment
Key Inclusion Criteria for Participants with TED in Phase 1/2 MAD cohorts:
- Must have moderate to severe active TED with documented evidence of ocular symptoms or
signs that began within 1 year prior to screening OR moderate to severe chronic TED
with documented evidence of ocular symptoms or signs that began over 1 year prior to
screening
- Must have active TED and a Clinical Activity Score (CAS) of ≥ 4 on the 7-item scale
for the study (more proptotic) eye OR chronic TED with no CAS requirement
- Must agree to use highly effective contraception as specified in the protocol
- Female TED participants must have a negative serum pregnancy test
Key Exclusion Criteria for Participants with TED in Phase 1/2 MAD cohorts:
- Must not have received prior treatment with another anti-IGF-1R monoclonal antibody
- Must not have used oral corticosteroids within 4 weeks prior to Day 1
- Must not have received rituximab, tocilizumab or other immunosuppressive agents within
90 days prior to Day 1
- Must not have evidence of optic nerve involvement within the previous 6 months
- Must not have corneal decompensation in the study eye unresponsive to medical
management
- Must not have had previous orbital irradiation or surgery for TED in the study eye
- Must not have a history inflammatory bowel disease Must not have clinically
significant ear pathology or hearing impairment
- Must not have received an investigational agent for any condition within 60 days
- Female TED participants must not be pregnant or lactating
Key Inclusion Criteria for Participants with TED in Phase 3 study:
- Must have moderate to severe active TED with documented evidence of ocular symptoms or
signs that began within 15 months prior to screening
- Must have Clinical Activity Score (CAS) of ≥ 3 on the 7-item scale for the study (more
proptotic) eye
- Must agree to use highly effective contraception as specified in the protocol
- Female TED participants must have a negative serum pregnancy test
Key Exclusion Criteria for Participants with TED in Phase 3 study:
- Must not have received prior treatment with another anti-IGF-1R monoclonal antibody
- Must not have used oral corticosteroids 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 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 surgery for TED in the study eye
- Must not have a history inflammatory bowel disease
- Must not have a history or screening audiometry assessment of clinically significant
(as determined by investigator) ear pathology, relevant ear surgery, or hearing l loss
- Must not have received an investigational agent for any condition
- Female TED participants must not be pregnant or lactating
Intervention(s):
drug: VRDN-001 Phase 1/2 MAD (HV and TED)
drug: VRDN-001 Phase 3 Cohort (THRIVE)
drug: VRDN-001 Placebo
Recruiting
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305