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)

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

Study Overview

• Status: Recruiting
• Conditions: Thyroid Eye Disease
• Intervention / Treatment: Drug: VRDN-001 10 mg/kg Drug:

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 Type: Interventional
• Enrollment (Estimated): 159
• Phase: Phase 3

Contacts and Locations

Study Locations

• Turkey
  • Ankara, Turkey
    • Recruiting
    • Gazi Oniversitesi Tip FakOltesi Hastanesi
    • Contact: Onur Konuk; Phone Number: 00 90 242 249 69 54
  • Ankara, Turkey
    • Recruiting
    • Hacettepe Universitesi Tip Fak0ltesi
    • Contact: Hayyam Kirati; Phone Number: 90 312 305 10 80
  • Konya, Turkey, 07070
    • Recruiting
    • Akdeniz Oniversitesi T,p FakOltesi. Goz Hastal,k.an
    • Contact: Deniz Ilhan; Phone Number: 00 90 242 249 69 54
• United States
  • California
    • Los Angeles, California, United States, 90048
      • Recruiting
      • MACRO Trials, Inc
      • Contact: Steven Leibowitz, MD; Phone Number: 702-242-5555
    • Newport Beach, California, United States, 92660
      • Recruiting
      • Amy Patel Jain, MD Inc
      • Contact: Amy Patel Jain, MD; Phone Number: 949-423-9734
    • Palo Alto, California, United States, 94303
      • Recruiting
      • Byers Eye Institute
      • Contact: Andrea Kossler, MD; Phone Number: 650-723-6995
    • San Diego, California, United States, 92108
      • Recruiting
      • Senta Clinic
      • Contact: Kimberly Cockerham; Phone Number: 858-925-3363
  • Florida
    • Sarasota, Florida, United States, 34239
      • Recruiting
      • Sarasota Retina Institute
      • Contact: Coordinator Coordinator; Phone Number: 232 941-921-5335
  • Illinois
    • Oak Lawn, Illinois, United States, 60453
      • Recruiting
      • Vision Medical Research, INC
      • Contact: Mohammad Al-Khudari, MD; Phone Number: 708-636-9393
  • Massachusetts
    • Boston, Massachusetts, United States, 02189
      • Recruiting
      • Ophthalmic Consultants of Boston
      • Contact: John Mandeville, MD; Phone Number: 800-635-0489
  • Michigan
    • East Lansing, Michigan, United States, 48824
      • Recruiting
      • Michigan State University Department of Neurology and Ophthalmology
      • Contact: David Kaufman, DO; Phone Number: 517-353-8122
    • Livonia, Michigan, United States, 48152
      • Recruiting
      • Kahana Oculoplastic and Orbital Surgery
      • Contact: Coordinator Coordinator; Phone Number: 248-800-1177
  • New Jersey
    • Newark, New Jersey, United States, 07103
      • Recruiting
      • Rutgers New Jersey Medical
      • Contact: Roger Turbin; Phone Number: 973-972-2209
    • Somerset, New Jersey, United States, 08873
      • Recruiting
      • The Center for Eye and Facial Plastic Surgery
      • Contact: Sathyadeepak Ramesh, MD; Phone Number: 609-608-0142
  • Texas
    • Bellaire, Texas, United States, 77401
      • Recruiting
      • Eye Wellness Center- Neuro-Eye Clinical Trials, Inc.
      • Contact: Coordinator; Phone Number: 346-429-4135

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: Yes

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / 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

What is the study measuring?

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	Week 15
Change from baseline in CAS in the study eye	Change from baseline in CAS in the study eye	Week 15
Overall Response Rate in the study eye	Overall Response Rate in the study eye	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

Collaborators and Investigators

• Sponsor: Viridian Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): November 14, 2023
• Primary Completion (Estimated): December 1, 2024
• Study Completion (Estimated): September 1, 2025

Study Registration Dates

• First Submitted: August 25, 2023
• First Submitted That Met QC Criteria: August 25, 2023
• First Posted (Actual): September 1, 2023

Study Record Updates

• Last Update Posted (Actual): April 22, 2024
• Last Update Submitted That Met QC Criteria: April 19, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Keywords: Thyroid Eye Disease; Thyroid-Associated Ophthalmopathy; Dysthyroid Ophthalmopathy; Graves Eye Disease; Graves Orbitopathy; Myopathic Ophthalmopathy; Congestive Ophthalmopathy; Edematous Ophthalmopathy; Infiltrative Ophthalmopathy; Graves
• Additional Relevant MeSH Terms: Immune System Diseases; Autoimmune Diseases; Endocrine System Diseases; Genetic Diseases, Inborn; Eye Diseases, Hereditary; Graves Disease; Exophthalmos; Orbital Diseases; Goiter; Hyperthyroidism; Eye Diseases; Graves Ophthalmopathy; Thyroid Diseases
• Other Study ID Numbers: VRDN-001-301

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No