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Completed

NCT Number: NCT06021054

An Efficacy, Safety, and Tolerability Study of Veligrotug (VRDN-001), in Participants With Chronic Thyroid Eye Disease (TED)

This is a clinical trial assessing the efficacy, safety, and tolerability of an investigational drug, veligrotug (VRDN-001), in participants with chronic thyroid eye disease (TED).

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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–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

North Shore Private Hospital, Saint Leonards, New South Wales, Australia

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About this study

This is a randomized (meaning participants will be assigned to study arms by chance), double-masked (meaning study doctor and participant will not know which study arm participant is assigned to), placebo-controlled study that will include participants with chronic TED. The key objectives of this study are to determine if veligrotug (VRDN-001) is efficacious, safe, and tolerable when administered as 5 IV infusions given every 3 weeks for a total of 12 weeks in participants with chronic TED.

Who can participate

Healthy volunteers accepted: No

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

Key Inclusion Criteria:

  • Must have moderate to severe chronic TED with documented evidence of ocular symptoms or signs that began greater than 15 months prior to screening
  • Must have had a clinical diagnosis of TED, with any CAS (0-7)
  • Must agree to use highly effective contraception as specified in the protocol
  • Female TED participants must have a negative serum pregnancy test at screening

Key Exclusion Criteria:

  • Must not have received prior treatment with another anti-IGF-1R therapy
  • Must not have received systemic corticosteroids for any condition, including TED, or selenium within 2 weeks prior to first dose
  • Must not have received other immunosuppressive drugs or another investigational agent for any condition, including TED, or any other therapy for TED, within 8 weeks prior to first dose
  • Must not have received radioactive iodine (RAI) treatment within 8 weeks prior to first dose
  • Must not have a pre-existing ophthalmic condition in the study eye that in the opinion of the study doctor would confound interpretation of the study results
  • Must not have had previous orbital irradiation or decompression surgery for TED to the study eye's orbit
  • Must not have inflammatory bowel disease
  • Must not have abnormal baseline audiometry Pure Tone Average (PTA) assessment or history of significant (as determined by the Investigator) ear pathology, relevant ear surgery or hearing loss.
  • Female TED participants must not be pregnant or lactating

Treatment and study plan

Veligrotug

Drug

5 IV Infusions of veligrotug 10 mg/kg

Other names: VRDN-001

Placebo

Drug

5 IV Infusions of veligrotug matched placebo

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 imputed with the Multiple Imputation (MI) method.

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 imputed with the MI method.

  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 imputed using the Exophthalmometer Imputation (EXI) method, as applicable.

  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 imputed using the EXI method, as applicable.

  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 imputed with the MI method.

  5. Overall Responder Rate (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 imputed with the MI method.

  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 imputed with the MI method.

  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 imputed with the MI method.

Sponsors and collaborators

Lead sponsor

Viridian Therapeutics, Inc.

Industry

Registry information

Official study 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)

Acronym: THRIVE-2

Important dates

Study start
2023
Primary completion
2024
Study completion
2025
First posted
Sep 1, 2023
Registry last updated
Aug 13, 2026

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

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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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