Treatment of Graves' Orbitopathy to Reduce Proptosis With Teprotumumab Infusions in an Open-Label Clinical Extension Study (OPTIC-X)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03461211|
Recruitment Status : Enrolling by invitation
First Posted : March 9, 2018
Last Update Posted : March 8, 2019
|Condition or disease||Intervention/treatment||Phase|
|Thyroid Eye Disease||Biological: Teprotumumab||Phase 3|
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 were 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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||50 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Multicenter, Safety and Efficacy, Open-Label Extension Study Evaluating Teprotumumab (HZN-001) Treatment in Subjects With Thyroid Eye Disease|
|Actual Study Start Date :||April 16, 2018|
|Estimated Primary Completion Date :||September 2020|
|Estimated Study Completion Date :||March 2022|
Experimental: Teprotumumab 20 mg/kg
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).
Approximately 50 participants will receive 8 infusions of Teprotumumab q3W for a total of 21 weeks. Teprotumumab 10 mg/kg will be administered on Day 1 and Teprotumumab 20 mg/kg will be administered q3W for the remaining 7 infusions.
- Effect of teprotumumab on the proptosis responder rate at the end of treatment at Week 24 [ Time Frame: Week 24 ]The percentage of participants with a ≥ 2 mm reduction from Baseline in the study eye without deterioration [≥ 2 mm increase] of proptosis in the fellow eye.
- Percentage of participants with a Clinical Activity Score (CAS) value of 0 or 1 at Week 24 in the study eye [ Time Frame: Week 24 ]
- Mean change from Baseline to Week 24 in proptosis measurement in the study eye [ Time Frame: Week 24 ]
- Effect of teprotumumab on the diplopia responder rate at Week 24 [ Time Frame: Week 24 ]The percentage of participants with baseline diplopia > 0 in study eye who have a reduction of ≥ 1 grade with no corresponding deterioration [≥ 1 grade worsening] in the fellow eye) at Week 24.
- Mean change from Baseline to Week 24 in the Graves' Ophthalmopathy Quality of Life (GO-QoL) questionnaire overall score [ Time Frame: Week 24 ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03461211
|United States, California|
|Beverly Hills, California, United States, 90212|
|Cedars-Sinai Medical Center|
|Los Angeles, California, United States, 90078|
|United States, Florida|
|The Lennar Foundation Medical|
|Coral Gables, Florida, United States, 33146|
|Bascom Palmer Eye Institute|
|Miami, Florida, United States, 33135|
|United States, Michigan|
|Kellogg Eye Center at University of Michigan|
|Ann Arbor, Michigan, United States, 48105|
|United States, Oregon|
|Casey Eye Institute at Oregon Health and Science|
|Portland, Oregon, United States, 97239|
|United States, Tennessee|
|Hamilton Eye Institute at University of Tennessee Health|
|Memphis, Tennessee, United States, 38163|
|United States, Texas|
|Eye Wellness Center|
|Houston, Texas, United States, 77005|
|United States, Wisconsin|
|Medical College of Wisconsin, The Eye Institute|
|Milwaukee, Wisconsin, United States, 53226|
|University Hospital Essen, Department of Ophthalmology|
|Essen, Germany, 45147|
|Johannes Gutenberg University Medical Center|
|Mainz, Germany, 55131|
|Fondazione IRCCS Ca Granda Ospedale Maggiore|
|Milan, Italy, 20122|
|University of Pisa, Department of Clinical and Experimental Medicine|
|Pisa, Italy, 56100|
|University of Pisa, Department of Clinical and Experimental Medicine, Endocrinology Unit|
|Pisa, Italy, 56124|
|Principal Investigator:||Raymond Douglas, MD, PhD||Cedars-Sinai Medical Center|
|Principal Investigator:||George Kahaly, MD, PhD||Johannes Gutenberg University Medical Center|