TEPEZZA (teprotumumab-trbw) Post-Marketing Requirement

  • Research type

    Research Study

  • Full title

    A Phase 3b/4, Double-masked, Randomized, International, Parallel-assignment, Multicenter Trial in Patients with Thyroid Eye Disease to Evaluate the Safety and Tolerability of Different Dosing Durations of Teprotumumab

  • IRAS ID

    1004199

  • Contact name

    Michael Karon

  • Contact email

    mkaron@horizontherapeutics.com

  • Sponsor organisation

    Horizon Therapeutics U.S.A., Inc.

  • Eudract number

    2020-005999-36

  • Clinicaltrials.gov Identifier

    NCT05002998

  • Research summary

    Thyroid eye disease (TED) is a serious, debilitating and painful autoimmune disease that can, in severe cases, lead to blindness. TED is commonly associated with Graves’ disease, but also occurs in some patients with other autoimmune thyroid diseases, including Hashimoto’s thyroiditis. The eye problems seen with TED include swelling, redness and discomfort of the eyelids and eye surface, increased tearing, thickening and pulling back of the eyelids, bulging of the eyes, squints, and double vision.

    The purpose of this study is to obtain information on the safety, efficacy, tolerability, and need for re-treatment of 3 different treatment durations with a drug called teprotumumab (HZN-001) in adults with thyroid eye disease (TED). This study is being performed to fulfil a post-marketing requirement from the US FDA.

    Participants who meet all eligibility criteria will be randomised to receive one of the following treatment lengths:
    • 4 infusions of teprotumumab (Group 1), followed by either 4 infusions of teprotumumab or 4 infusions of placebo.
    or
    • 8 infusions of teprotumumab (Group 2)
    or
    • 16 infusions of teprotumumab (Group 3)

    The study consists of screening assessments, a 24 or 48 week initial treatment period (9 or 17 study visits) and a 52 week follow-up period (3 study visits). There is also a re-treatment period if TED returns during the follow-up period (36 weeks). Assessments include physical examinations, vital signs, blood and urine sample collection for testing and completion of quality of life questionnaire. The total study duration will be a maximum of 136 weeks depending on which group (also known as a cohort) participants are assigned to and if re-treatment is required.

    Approximately 300 participants will participate worldwide with approximately 70 patients from 7 hospitals in the UK. The Sponsor of this study is Horizon Therapeutics USA , Inc..

  • REC name

    South Central - Hampshire A Research Ethics Committee

  • REC reference

    22/SC/0012

  • Date of REC Opinion

    4 Feb 2022

  • REC opinion

    Further Information Favourable Opinion