An Open-label Study for Participants Who Are Non-responders at the End of Treatment Assessment on the VRDN-001-101 and VRDN-001-301 Pivotal Studies

Study Overview
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Brief Summary
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An open-label study for participants who are non-responders at end of the treatment period assessment (i.e., 15 weeks) in the VRND-001-101 (THRIVE) and VRDN-001-301 (THRIVE-2) pivotal studies

Official Title
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An Open-label Study for Participants Who Are Non-responders at the End of Treatment Assessment on the VRDN-001-101 and VRDN-001-301 Pivotal Studies

Conditions
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Thyroid Eye Disease

Intervention / Treatment
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* Drug: Intervention/Treatment


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Have completed at least 5 infusions and assessments required to determine proptosis responder status as measured by exophthalmometer 3 weeks post the fifth infusion (i.e., Week 15) as defined in either the VRDN-001-101 or VRDN-001-301 pivotal studies Been a participant in either the VRDN-001-101 or VRDN-001-301 pivotal studies and found to be non-responder Must agree to use highly effective contraception as specified in the protocol Female Thyroid Eye Disease (TED) participants must have a negative urine pregnancy test Exclusion Criteria: Must not have received prior treatment with another anti-IGF-1R monoclonal antibody or any other investigational agent for TED Must not have used systemic 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 history or screening audiometry assessment of clinically significant (as determined by investigator) ear pathology, relevant ear surgery, or hearing loss Must not have received an investigational agent for any condition (other than VRDN-001 or placebo associated with the VRDN-001-101 or VRDN-001-301 pivotal studies) within 8 weeks prior to Day 1

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: Single treatment assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: VRDN-001 10 mg/kg<br>5 infusions of VRDN-001 10 mg/kg | Drug: Intervention/Treatment<br>* VRDN-001 10 mg/kg (5 infusions of VRDN-001 10 mg/kg)<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Proptosis Responder Rate in the study eye | Proptosis Responder Rate in the study eye (i.e., reduction of proptosis of ≥2 mm from baseline [without a corresponding increase of ≥2 mm in the fellow eye] as measured by exophthalmometer) at 3 weeks post the fifth infusion (i.e., Week 15) | 3 weeks post the fifth infusion | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from Baseline in proptosis in the study eye at Week 15 | Change from Baseline in proptosis in the study eye as measured by exophthalmometer at Week 15 | Week 15 | 
| Clinical Activity Responder Rate in the study eye | Clinical Activity Responder Rate in the study eye at Week 15 | Week 15 | 
| Change from baseline in Clinical Activity Score in the study eye | Change from baseline in Clinical Activity Score in the study eye at Week 15 | Week 15 | 
| Overall Responder Rate in the study eye | Overall Responder Rate in the study eye at Week 15 | Week 15 | 
| Diplopia Resolution Rate | Diplopia Resolution Rate (i.e., reduction in Diplopia Score to 0 from baseline for participants with baseline Diplopia Score >0) at Week 15 | Week 15 |