Treatment of Graves' Orbitopathy to Reduce Proptosis With Teprotumumab Infusions in an Open-Label Clinical Extension Study
Condition: Thyroid Eye Disease
MeSH terms: Eye Diseases; Graves Ophthalmopathy
Study type: Interventional
Phase: Phase 3

Summary: 
      The overall objective is to evaluate the safety and efficacy of teprotumumab in the treatment
      of thyroid eye disease (TED) in participants who participated in the lead-in study
      HZNP-TEP-301 (NCT03298867; OPTIC) and who were either proptosis non-responders at Week 24 of
      HZNP-TEP-301 or were proptosis responders at Week 24 but met the criteria for re-treatment
      due to relapse during the Follow-Up Period of HZNP-TEP-301.
    

Detailed description: 
      This is a multi-center, open-label extension study of HZNP-TEP-301 (NCT03298867) examining
      the safety and efficacy of teprotumumab in the treatment of TED in adult participants.
      Participants who complete the 24-week double-masked Treatment Period in Study HZNP-TEP-301
      and are proptosis non-responders or are proptosis responders at Week 24 but meet the criteria
      for re-treatment due to relapse during the Follow-Up Period of HZNP-TEP-301 will be eligible
      for enrollment.

      All participants who choose to participate will receive 8 infusions of teprotumumab (10 mg/kg
      for the first infusion followed by 20 mg/kg for the remaining 7 infusions) in an open-label
      fashion. The Baseline (Day 1) Visit of this extension study will occur within 14 days after
      the final visit of Study HZNP-TEP-301 (Week 24 for proptosis non-responders and up to Week 72
      for participants who relapse). During the open-label Treatment Period, study drug infusions
      are scheduled for Day 1 (Baseline), and Weeks 3, 6, 9, 12, 15, 18, and 21, (with the final
      visit at Week 24). After completion of the Treatment Period, subjects who were proptosis
      non-responders in Study HZNP-TEP-301 will enter a 24-week Follow-Up Period during which study
      drug will not be administered and clinic visits are scheduled for 1, 3, and 6 months (Visits
      Month 7, 9, and 12) after the Week 24 visit. Subjects will be contacted 6 and 12 months later
      by phone or email to enquire if any treatment for TED had been received since the last study
      contact.

      Participants who relapse during the Follow-Up Period of HZNP-TEP-301 and choose to enter this
      extension study will not participate in the Follow-Up Period of this study but will be
      contacted by phone or email 6 and 12 months after the Week 24 visit.
    

Intervention name(s): Teprotumumab
Intervention type(s): Biological
Intervention description(s): Teprotumumab is a fully human anti-IGF-1R mAb. Teprotumumab will be provided in single-dose 20 mL glass vials as a freeze-dried powder. Each vial of teprotumumab must be reconstituted with 10 mL of water for injection. Reconstituted teprotumumab solution must be further diluted in 0.9% (w/v) sodium chloride (NaCl) solution prior to administration. Teprotumumab will be administered in 100 mL or 250 mL infusion bags (100 mL infusion bags for doses up to 1800 mg and 250 mL infusion bags for doses > 1800 mg).
Other intervention name(s): HZN-001
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment