A Randomized, Double-masked, Placebo-controlled Safety, Tolerability, and Efficacy Study of VRDN-001, a Humanized Monoclonal Antibody Directed Against the IGF-1 Receptor, in Participants With Chronic Thyroid Eye Disease (TED)

Sponsor

Viridian Therapeutics, Inc. (Industry)

Overall Status

Not yet recruiting

CT.gov ID

NCT06021054

Collaborator

(none)

159

Enrollment

Arms

Anticipated Duration (Months)

Study Details

Study Description

Brief Summary

A randomized, double-masked, placebo-controlled safety, tolerability and efficacy study of VRDN-001 in participants with chronic thyroid eye disease (TED)

Condition or Disease	Intervention/Treatment	Phase
Thyroid Eye Disease	Drug: VRDN-001 10 mg/kg Drug:	Phase 3

Detailed Description

A randomized, double-masked, placebo-controlled safety, tolerability and efficacy study of VRDN-001, a humanized monoclonal antibody directed against the IGF-1 receptor, in participants with chronic thyroid eye disease (TED)

Study Design

Study Type:

Interventional

Anticipated Enrollment :

159 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Double (Participant, Investigator)

Primary Purpose:

Treatment

Official Title:

A Randomized, Double-masked, Placebo-controlled Safety, Tolerability, and Efficacy Study of VRDN-001, a Humanized Monoclonal Antibody Directed Against the IGF-1 Receptor, in Participants With Chronic Thyroid Eye Disease (TED)

Anticipated Study Start Date :

Sep 1, 2023

Anticipated Primary Completion Date :

Dec 1, 2024

Anticipated Study Completion Date :

Sep 1, 2025

Arms and Interventions

Arm	Intervention/Treatment
Experimental: VRDN-001 10 mg/kg Drug: 5 Infusions of VRDN-001 10 mg/kg	Drug: VRDN-001 10 mg/kg Drug: Drug: 5 Infusions of VRDN-001 10mg/ Placebo: 5 Infusions of placebo
Experimental: Placebo Drug Placebo Drug: 5 Infusions of placebo	Drug: VRDN-001 10 mg/kg Drug: Drug: 5 Infusions of VRDN-001 10mg/ Placebo: 5 Infusions of placebo

Arm

Intervention/Treatment

Experimental: VRDN-001 10 mg/kg

Drug: 5 Infusions of VRDN-001 10 mg/kg

Drug: VRDN-001 10 mg/kg Drug:

Drug: 5 Infusions of VRDN-001 10mg/ Placebo: 5 Infusions of placebo

Experimental: Placebo Drug

Placebo Drug: 5 Infusions of placebo

Drug: VRDN-001 10 mg/kg Drug:

Drug: 5 Infusions of VRDN-001 10mg/ Placebo: 5 Infusions of placebo

Outcome Measures

Primary Outcome Measures

Proptosis Responder Rate in the study eye [3 weeks post the fifth infusion]
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)

Secondary Outcome Measures

Change from Baseline in proptosis in the study eye at Week 15 [Week 15]
Change from Baseline in proptosis in the study eye as measured by exophthalmometer at Week 15
Clinical Activity Responder Rate in the study eye [Week 15]
Clinical Activity Responder Rate in the study eye
Change from baseline in CAS in the study eye [Week 15]
Change from baseline in CAS in the study eye
Overall Response Rate in the study eye [Week 15]
Overall Response Rate in the study eye
Diplopia Resolution Rate [Week 15]
Diplopia Resolution Rate (i.e., reduction in Diplopia Score to 0 from baseline for participants with baseline Diplopia Score >0) at Week 15

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Yes

Inclusion Criteria:

Must have moderate to severe chronic TED with documented evidence of ocular symptoms or signs that began > 15 months prior to screening
Must have Clinical Activity Score (CAS) of (0-7) 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:

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