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OpenTrials
Completed

NCT Number: NCT06179875

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

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 participants with thyroid eye disease (TED). The primary objectives of this clinical trial are to provide open-label access to VRDN-001 for participants who were previously non-responders at 3 weeks post the fifth IV infusion (i.e., 15 weeks) in the VRDN-001-101 (THRIVE) and VRDN-001-301 (THRIVE-2) pivotal studies and assess the safety and efficacy of VRDN-001 in participants who were previously treated with VRDN-001 or placebo.

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Key information

Conditions

Thyroid Eye Disease Autoimmune Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Endocrine System Diseases Exophthalmos Eye Diseases Eye Diseases, Hereditary Genetic Diseases, Inborn Goiter Graves Disease Graves Ophthalmopathy Hyperthyroidism Immune System Diseases Orbital Diseases Thyroid Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Centre Hospitalier Universitaire de Montpellier Hopital Lapeyronie, Montpellier, Hérault, France

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Key Inclusion Criteria:

  • 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
  • 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
  • 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
  • Not require immediate surgical ophthalmological or orbital surgery in the study eye for any reason
  • Must agree to use highly effective contraception as specified in the protocol
  • Female TED participants must have a negative urine pregnancy test at screening
  • 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:

  • Have received prior treatment with another anti-IGF-1R agent
  • Have received systemic corticosteroids for any condition, including TED, or selenium within 2 weeks prior to first dose
  • 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
  • Have received radioactive iodine (RAI) treatment within 8 weeks prior to first dose
  • Have had previous orbital irradiation or decompression surgery involving excision of fat for TED to the study eye's orbit
  • Have abnormal hearing test before first dose. Have a history of ear conditions considered significant by study doctor

Treatment and study plan

Veligrotug

Drug

Veligrotug 10 mg/kg (5 infusions of Veligrotug 10 mg/kg)

Other names: VRDN-001

Primary outcomes

  1. Proptosis Responder Rate (PRR) in the Most Proptotic Eye as Measured by Exophthalmometer

    Time frame: Baseline to Week 15

    Proptosis response in the most proptotic eye was defined as a reduction of proptosis of ≥2 millimeters (mm) from baseline in the most proptotic eye (without a corresponding increase of ≥2 mm in the other eye) as measured by exophthalmometer. Missing data were not imputed.

Secondary outcomes

  1. Change From Baseline in Proptosis in the Most Proptotic Eye as Measured by Exophthalmometer

    Time frame: Baseline, Week 15

    Proptosis was defined as distance between the lateral orbital rim and the most anterior position of the cornea in mm, measured using an exophthalmometer. Missing data were not imputed.

  2. PRR in the Most Proptotic Eye as Measured by Magnetic Resonance Imaging (MRI)/Computed Tomography (CT)

    Time frame: Baseline to Week 15

    Proptosis responder in the most proptotic eye by MRI/CT was defined as a reduction of proptosis of ≥2 mm from baseline in the most proptotic eye by MRI/CT (without a corresponding increase of ≥2 mm in the other eye) as measured by MRI/CT. Missing data were not imputed.

  3. Change From Baseline in Proptosis in the Most Proptotic Eye as Measured by MRI/CT

    Time frame: Baseline, Week 15

    Measurement of proptosis was conducted by the central imaging reading center using MRI/CT of the orbits acquired without contrast. Measurements were conducted by 2 independent readers with adjudication if the difference between the 2 primary readers in proptosis measurement exceeded 5% (calculated as the difference divided by the larger measurement). The average of 2 (or 3 if adjudicated) measurements were used for analyses. Missing data were not imputed.

  4. Clinical Activity Responder Rate in the Most Proptotic Eye as Measured by Exophthalmometer

    Time frame: Week 15

    Clinical activity responder in the most proptotic eye was defined as no worsening in clinical activity score (CAS) from baseline in the most proptotic eye without a corresponding increase of ≥2 points in the other eye. Missing data were not imputed.

  5. ORR Comprising PRR and Clinical Activity Responder Rate in the Most Proptotic Eye as Measured by Exophthalmometer

    Time frame: Baseline to Week 15

    ORR was comprised of PRR in the most proptotic eye (reduction of proptosis of ≥2 mm from baseline in the most proptotic eye [without a corresponding increase of ≥2 mm in the other eye]) as measured by exophthalmometer at Week 15 and clinical activity responder rate in the most proptotic eye (no worsening in CAS from baseline in the most proptotic eye without a corresponding increase of ≥2 points in the other eye) as measured by exophthalmometer at Week 15. Missing data were not imputed.

  6. Diplopia Responder Rate

    Time frame: Week 15

    A diplopia responder was defined as having a decrease of ≥1 from baseline for participants with a baseline Gorman subjective diplopia score >0. Gorman subjective diplopia score (range, 0 to 3) includes 4 categories: no diplopia (absent, scored as 0), diplopia in the primary position of gaze when the participant is tired or awakening (intermittent, scored as 1), diplopia at extremes of gaze (inconstant, scored as 2), and continuous diplopia in the primary or reading position (constant, scored as 3). Missing data were not imputed.

  7. Diplopia Resolution Rate

    Time frame: Week 15

    Diplopia resolution was defined as reduction in Gorman subjective diplopia score to 0 from baseline for participants with baseline Gorman subjective diplopia score >0. Gorman subjective diplopia score (range, 0 to 3) includes 4 categories: no diplopia (absent, scored as 0), diplopia in the primary position of gaze when the participant is tired or awakening (intermittent, scored as 1), diplopia at extremes of gaze (inconstant, scored as 2), and continuous diplopia in the primary or reading position (constant, scored as 3). Missing data were not imputed.

Sponsors and collaborators

Lead sponsor

Viridian Therapeutics, Inc.

Industry

Registry information

Official study title

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

Acronym: OLE

Important dates

Study start
2024
Primary completion
2025
Study completion
2025
First posted
Dec 22, 2023
Registry last updated
Aug 14, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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