Trial results for the Phase 2 study (NCT03414229) investigating pembrolizumab in combination with epacadostat for patients with metastatic and/or locally advanced sarcoma were posted on ClinicalTrials.gov on 2025-05-02. The study reported a limited objective response rate, with only 1 out of 30 enrolled participants achieving an objective response across all sarcoma subtypes.
Background
The study investigated the combination of epacadostat, an IDO1 inhibitor, and pembrolizumab (Keytruda) in patients diagnosed with metastatic and/or locally advanced sarcoma. The trial aimed to assess the effects and efficacy of this combination therapy.
Trial design
The study (NCT03414229) was a Phase 2 trial that enrolled 30 participants with metastatic and/or locally advanced sarcoma. The trial evaluated the combination of epacadostat and pembrolizumab. The primary objective included assessing the objective response rate to the combination therapy.
Key results
The trial reported the "Number of Patients With Best Objective Response Rate" across different sarcoma subtypes:
- In the UPS, Liposarcoma or Pleomorphic Liposarcoma group, 0 out of 10 participants achieved an objective response.
- In the Leiomyosarcoma group, 1 out of 4 participants achieved an objective response.
- In the Vascular Sarcoma Subtypes group, 0 out of 5 participants achieved an objective response.
- In the Other sarcoma group, 0 out of 10 participants achieved an objective response.
Across all enrolled participants, a total of 1 objective response was observed among the 30 participants in the study.
What this means
The results from this Phase 2 trial indicate that the combination of pembrolizumab and epacadostat demonstrated a very limited objective response rate in patients with metastatic and/or locally advanced sarcoma. With only 1 objective response observed among 30 participants, these findings suggest that this particular combination therapy may not be highly effective for this patient population under the conditions studied. Further research would be needed to explore potential efficacy in specific sarcoma subtypes or patient subgroups, or to evaluate alternative treatment strategies.
Source
The information regarding these trial results was obtained from ClinicalTrials.gov, a public database of clinical studies. The results for study NCT03414229, titled "A Study of Epacadostat, an IDO1 Inhibitor, in Combination With Pembrolizumab in Patients With Metastatic and/or Locally Advanced Sarcoma," were posted on 2025-05-02 on clinicaltrials.gov.