Teprotumumab shows promising results in integrated trials for thyroid eye disease
Results from the European Union (EU) study sites of the clinical trial program evaluating teprotumumab for the treatment of thyroid eye disease (TED) have shown significant improvements in patient outcomes, according to a presentation at ENDO 2023.
The study analyzed data from the integrated randomized, double-masked, placebo-controlled phase 2 and 3 trials, as well as the open-label OPTIC-X trial, involving EU patients. The primary outcome assessed was the reduction of proptosis by at least 2 millimeters from baseline. Secondary outcomes included diplopia response (reduction in double vision), Clinical Activity Score (CAS), overall response (combination of CAS reduction and proptosis reduction), changes in proptosis measurements, and improvements in the Graves’ ophthalmopathy-specific quality-of-life (GO-QOL) score.
A total of 35 patients were assigned to the teprotumumab group, while 39 patients received the placebo in the integrated EU trials. The study observed a higher prevalence of tobacco use among EU patients compared to US patients (44.6% vs 13.4%). The EU patients also had a longer duration since the diagnosis of Graves’ disease compared to their US counterparts (48.8 months vs 26.3 months). At baseline, fewer EU patients experienced constant diplopia (grade 3) compared to US patients (13.5% vs 25.8%).
After 24 weeks of treatment, the percentage of EU patients in the teprotumumab group who experienced a proptosis response was significantly higher compared to the placebo group (77% vs 13%). All secondary outcomes, including diplopia response, CAS 0/1, overall response, changes in proptosis measurements, and improvements in GO-QOL scores, showed significantly better results in the teprotumumab group compared to the placebo group.
In the open-label OPTIC-X trial, 19 EU patients who had previously received the placebo were administered teprotumumab for the first time. After 24 weeks of treatment, 95% of patients experienced a proptosis response, 54% showed a diplopia response, 69% achieved a CAS 0/1, and 81% demonstrated an overall response. Additionally, there were significant improvements in proptosis measurements and GO-QOL scores.
Kahaly GJ, Fu Q, MA, Holt RJ. Teprotumumab Pooled Efficacy from the European Study Sites Participating in the Phase 2, OPTIC (Phase 3) and OPTIC-X Pivotal trials. Presented at: ENDO 2023.