A Safety, Tolerability and Efficacy Study of VRDN 001 in Healthy Volunteers and Persons With Thyroid Eye Disease (TED)

• Status: Recruiting
• Trial end date: 2023-12-01
• Target enrollment: 0
• Participant gender: All

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 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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: Accepts Healthy Volunteers

Details

• Lead Sponsor: Viridian Therapeutics, Inc.

Criteria

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 hearing impairment

Key Inclusion Criteria for Participants with TED:

• Must have moderate to severe active TED with documented evidence of ocular symptoms or signs that began within 1 year prior to screening

• Must have Clinical Activity Score (CAS) of ≥ 4 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:

• 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, or 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