Ended Phase II and Phase III (Integrated) Thyroid eye disease (TED)

A multiple ascending dose (MAD) safety, tolerability and efficacy study of VRDN-001, a humanized monoclonal antibody directed against the IGF-1 receptor, in normal healthy volunteers (NHVs) and subjects with thyroid eye disease (TED)

EU CTIS ID: 2024-512244-36-00

What this study is testing

To establish the safety, tolerability, and efficacy of VRDN-001, and the pharmacokinetic (PK) and pharmacodynamic (PD) profiles of VRDN-001 in NHV and TED patients over a dose range of 3.0 to 20.0 mg/kg.

  • Phase II and Phase III (Integrated)

A plain-language read of the study's public EU CTIS listing. The study team confirms the details.

Who can take part

You may be able to join if

  • Enrollment in the HV and Active TED MAD cohorts has been completed. The inclusion criteria corresponding to the HV and Active and Chronic TED MAD cohorts are now described in Appendices 4 and 5, respectively.
  • If female, have a negative serum pregnancy test at screening and further negative urine tests immediately before each dose of study medication following the last dose of study medication as described in Appendix 1C if the participant is a woman of childbearing potential (including those with <2 years since the onset of menopause, amenorrhea for <2 year, or not surgically sterile); such participants must agree to use an acceptable method of contraception such as a condom and a second highly effective method of contraception as described in Section 4.4 from Screening up to and including 100 days after the last dose of study medication. If the participant is initiating hormonal contraception at time of Screening or within one cycle of Day 1, participant agrees to use a double-barrier method of contraception until completing one-full cycle of hormonal contraception. An acceptable double-barrier combination method is a condom with either diaphragm or sponge with spermicide
  • Be surgically sterile males for at least 6 weeks, or agree to use an acceptable method of contraception such as a condom and a second highly effective method of contraception as described in Section 4.4 from Screening up to and including 100 days after the last dose of study medication
  • Be willing and able to comply with all the requirements of the protocol for the entire duration of the study.
  • Active TED Pivotal (THRIVE) participants Participants must: 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
  • Be an adult male or female participant, at least 18 years of age or older

You likely can't join if

  • Exclusion Criteria Enrollment in the HV and Active TED MAD cohorts has been completed. The exclusion criteria corresponding to the HV and Active and Chronic TED MAD cohorts are now described in Appendices 4 and 5, respectively.
  • Have a compressive optic neuropathy of TED that is expected to require surgical decompression in the immediate future.
  • Have corneal decompensation in the study eye unresponsive to medical management
  • Have a decrease in CAS of ≥2 points in the study eye between screening assessment and Day -1
  • Have a decrease in proptosis of ≥2 mm in the study eye between screening assessment and Day -1
  • Have had previous orbital irradiation or decompression surgery involving excision of fat for TED to the study eye's orbit
See the full eligibility criteria
Who can join
  • Enrollment in the HV and Active TED MAD cohorts has been completed. The inclusion criteria corresponding to the HV and Active and Chronic TED MAD cohorts are now described in Appendices 4 and 5, respectively.
  • If female, have a negative serum pregnancy test at screening and further negative urine tests immediately before each dose of study medication following the last dose of study medication as described in Appendix 1C if the participant is a woman of childbearing potential (including those with <2 years since the onset of menopause, amenorrhea for <2 year, or not surgically sterile); such participants must agree to use an acceptable method of contraception such as a condom and a second highly effective method of contraception as described in Section 4.4 from Screening up to and including 100 days after the last dose of study medication. If the participant is initiating hormonal contraception at time of Screening or within one cycle of Day 1, participant agrees to use a double-barrier method of contraception until completing one-full cycle of hormonal contraception. An acceptable double-barrier combination method is a condom with either diaphragm or sponge with spermicide
  • Be surgically sterile males for at least 6 weeks, or agree to use an acceptable method of contraception such as a condom and a second highly effective method of contraception as described in Section 4.4 from Screening up to and including 100 days after the last dose of study medication
  • Be willing and able to comply with all the requirements of the protocol for the entire duration of the study.
  • Active TED Pivotal (THRIVE) participants Participants must: 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
  • Be an adult male or female participant, at least 18 years of age or older
  • Have had a clinical diagnosis of TED with a CAS of ≥ 3 on the 7-item scale for the study eye
  • Have moderate to severe (i.e., has an appreciable impact on daily living) active TED associated with proptosis of ≥3 mm above normal values for race and gender in the opinion of the investigator and at least one of the following: lid retraction of ≥2 mm, moderate or severe soft tissue involvement, inconstant or constant diplopia, spontaneous retrobulbar pain or pain on eye movement, swelling of the conjunctiva, eyelids or plica, or redness of the eyelids or plica in the study eye
  • Have documented evidence of ocular symptoms or signs associated with active TED that began within 15 months prior to study screening
  • VRDN-001 can be started concomitantly with attempts to achieve euthyroid status. Underlying thyroid status is not an inclusion criterion.
  • Not require expected immediate surgical ophthalmological or orbital surgery in the study eye for any reason
  • VRDN-001 can be used with caution in patients with diabetes mellitus. Diabetic participants should be monitored by their endocrinologist or other appropriately trained personnel and have at study entry a glycated hemoglobin (HbA1c) of <8.5%
What rules you out
  • Exclusion Criteria Enrollment in the HV and Active TED MAD cohorts has been completed. The exclusion criteria corresponding to the HV and Active and Chronic TED MAD cohorts are now described in Appendices 4 and 5, respectively.
  • Have a compressive optic neuropathy of TED that is expected to require surgical decompression in the immediate future.
  • Have corneal decompensation in the study eye unresponsive to medical management
  • Have a decrease in CAS of ≥2 points in the study eye between screening assessment and Day -1
  • Have a decrease in proptosis of ≥2 mm in the study eye between screening assessment and Day -1
  • Have had previous orbital irradiation or decompression surgery involving excision of fat for TED to the study eye's orbit
  • Have history of or screening audiometry assessment of significant (as determined by the Investigator) ear pathology, relevant ear surgery or hearing loss
  • Have inflammatory bowel disease (e.g., biopsy proven or clinical evidence of inflammatory bowel disease)
  • Have a known hypersensitivity to any of the components of VRDN- 001 or placebo formulations, or prior hypersensitivity to monoclonal antibodies (mAbs)
  • Have any condition, which in the opinion of the Investigator, would preclude inclusion in the study
  • Have a positive test for human immunodeficiency virus (HIV-1 and HIV-2)
  • Active TED Pivotal (THRIVE) participants Participants must not: Have used systemic corticosteroids for any condition, including TED, or selenium within 2 weeks prior to the first dose of study medication (topical steroids or multivitamins that contain selenium are permitted)
  • Have a positive test for active hepatitis B or hepatitis C infection
  • Have previously participated in this study or any study of VRDN-001
  • French participating sites only: In accordance with the provisions of articles L.1121-5 et seq. of the Public Health Code, pregnant or breast- feeding women, persons deprived of their liberty by a judicial or administrative decision, persons under psychiatric care without their consent, minors and adults under a legal protection measure must not be included
  • Note: Prior thyroidectomy, radioactive iodine (RAI) treatment, or orbital decompression surgery limited to bone only are NOT exclusions.
  • Have received other immunosuppressive agents, including rituximab, or tocilizumab, for any condition, including TED, within 8 weeks prior to the first dose of study medication
  • Have received any other therapy for TED within 8 weeks prior to the first dose of study medication (artificial tears are permitted)
  • Have received an investigational agent for any condition within 8 weeks prior to the first dose of study medication
  • Have a pre-existing ophthalmic condition in the study eye which in the opinion of the Investigator, would confound interpretation of the study results
  • Be a pregnant or lactating woman
  • Be an active alcoholic or illicit drug user or considered at high risk of relapse by the Investigator
  • Active TED Pivotal (THRIVE) participants Participants must not: 1. Have received prior treatment with another anti-IGF-1R therapy or any investigational agent for TED

The study team makes the final eligibility decision.

Where it's taking place

  • Turkey
  • Canada
  • United States
  • Israel
  • China
  • Australia
  • United Kingdom

Questions & answers

Do participants get paid in this trial?

This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.

Is it free to join, and do I need insurance?

Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.

How long does this study last?

The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.

Who can join this trial?

This study is enrolling all sexes, 18-64 years, 65+ years. The study team makes the final eligibility decision.

Where is this trial taking place?

Study sites include Turkey; Canada; United States; Israel; China; Australia and 1 more location(s). Enter your location above to see the nearest site and check your eligibility.

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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.