An Open-label Study for Non-responders of VRDN-001-101 and VRDN-001-301 (OLE)

Phase 3

Active, not recruiting

• Conditions: Thyroid Eye Disease
• Interventions: Drug: Intervention/Treatment

• Registration Number: NCT06179875
• Lead Sponsor: Viridian Therapeutics, Inc.

Brief Summary

An open-label study for participants who received VRDN-001 or placebo and were non-responders at end of the treatment period assessment (i.e., 15 weeks) in the VRDN-001-101 (THRIVE) and VRDN-001-301 (THRIVE-2) pivotal studies

Detailed Description

Not available

Study Timeline

• Study Start: 2023-02-01
• Study First Submitted: 2023-12-12
• Study First Submitted QC: 2023-12-21
• Study First Posted: 2023-12-22
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-27
• Last Update Posted: 2025-04-02
• Primary Completion: 2025-05
• Study Completion: 2025-07

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 143

Inclusion Criteria

1. Be able to understand the study procedures and the risks involved and be willing to provide written informed consent before the first study-related activity

2. Have completed at least 5 IV infusions and assessments required to determine proptosis responder status 3 weeks post the fifth IV infusion (i.e., Week 15) as defined in either the VRDN-001-101 or VRDN-001-301 pivotal studies

3. Been a participant in either the VRDN-001-101 or VRDN-001-301 studies and found to be a non-responder as defined within the VRDN-001-101 or VRDN-001-301 study

4. Not require immediate surgical ophthalmological or orbital surgery in the study eye for any reason

5. Must agree to use highly effective contraception as specified in the protocol

   ⦁ Female TED participants must have a negative urine pregnancy test at screening

6. Be willing and able to comply with all the requirements of the protocol for the entire duration of the study

Key

Exclusion Criteria

Participants must not:

1. Have received prior treatment with another anti-IGF-1R agent
2. Have received systemic corticosteroids for any condition, including TED, or selenium within 2 weeks prior to first dose
3. Have received other immunosuppressive drugs or another investigational agent for any condition, including TED (other than VRDN-001 or placebo associated with the VRDN-001-101 or VRDN-001-301 pivotal studies), or any other therapy for TED, within 8 weeks prior to first dose
4. Have received radioactive iodine (RAI) treatment within 8 weeks prior to first dose
5. Have had previous orbital irradiation or decompression surgery involving excision of fat for TED to the study eye's orbit
6. Must not have abnormal hearing test before first dose. Must also not have a history of ear conditions considered significant by study doctor

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
VRDN-001 10 mg/kg	Intervention/Treatment	5 infusions of VRDN-001 10 mg/kg

Primary Outcome Measures

Name	Time	Method
Proptosis Responder Rate in the most proptotic eye	Week 15	Proptosis Responder Rate in the most proptotic eye (i.e., reduction of proptosis of ≥ 2 mm from baseline \[without a corresponding increase of ≥ 2 mm in the other eye\]) at 3 weeks post the fifth IV infusion (i.e., Week 15)

Secondary Outcome Measures

Name	Time	Method
Change from Baseline in proptosis in the most proptotic eye	Week 15	Change from Baseline in proptosis in the most proptotic eye at Week 15
Clinical Activity Responder Rate in the most proptotic eye	Week 15	Clinical Activity Responder Rate in the most proptotic eye at Week 15
Overall Responder Rate in the most proptotic eye	Week 15	Overall Responder Rate in the most proptotic eye at Week 15
Diplopia Responder Rate	Week 15	Diplopia Responder Rate (i.e., reduction in Gorman Subjective Diplopia Score of ≥1 from baseline for participants with baseline Gorman Subjective Diplopia Score \>0) at Week 15.
Diplopia Resolution Rate	Week 15	Diplopia Resolution Rate (i.e., reduction in Gorman Subjective Diplopia Score to 0 from baseline for participants with baseline Gorman Subjective Diplopia Score \>0) at Week 15

Trial Locations

Locations (44)

USC Eye Institute; 🇺🇸 Los Angeles, California, United States

Stanford Byers Eye Institute; 🇺🇸 Palo Alto, California, United States

University of Miami Miller School of Medicine, Bascom Palmer Eye Institute; 🇺🇸 Miami, Florida, United States

Ophthalmic Plastic, Reconstructive, Orbital and Cosmetic Surgery; 🇺🇸 Las Vegas, Nevada, United States

Hospital of the University of Pennsylvania Perleman Center; 🇺🇸 Philadelphia, Pennsylvania, United States

Baylor College of Medicine; 🇺🇸 Houston, Texas, United States

University of Washington Medical Center; 🇺🇸 Seattle, Washington, United States

Centre Hospitalier Universitaire de Montperllier Hopital Lapeyronie; 🇫🇷 Montpellier Cedex 5, Hérault, France

Centre Hospitalier Universitaire De Nantes G.R. Laenne; 🇫🇷 Saint-Herblain, Loire-Atlantique, France

CHU Angers; 🇫🇷 Angers Cedex, France

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