Trial results for the Phase 1/2 study (NCT04383938) investigating pembrolizumab in combination with APR-246 in subjects with solid tumor malignancies were posted on ClinicalTrials.gov on 2025-05-13. The study evaluated the safety of the combination and confirmed the recommended Phase 2 dose for APR-246 at 4.5 g/d.
Background
The study investigated the combination of APR-246 with pembrolizumab in various solid tumor malignancies, including bladder cancer, gastric cancer, non-small cell lung cancer (NSCLC), and urothelial carcinoma. Pembrolizumab is an established immunotherapy, and this trial explored its safety in combination with APR-246.
Trial design
The study (NCT04383938) was a Phase 1/2, open-label trial designed to determine the safety and preliminary efficacy of APR-246 in combination with pembrolizumab. It enrolled a total of 40 participants with solid tumor malignancies, specifically including bladder cancer, gastric cancer, non-small cell lung cancer, and urothelial carcinoma. The trial included a safety lead-in portion followed by a Phase 2 expansion portion in specific disease groups.
Key results
The trial evaluated the safety of APR-246 in combination with pembrolizumab in subjects with solid tumors. The safety evaluation included:
- 6 participants in the Safety Lead In group.
- 6 participants in Expansion 1.
- 5 participants in Expansion 2.
- 20 participants in Expansion 3.
The study also confirmed the recommended Phase 2 dose (RP2D) for APR-246 in combination with pembrolizumab at 4.5 g/d, based on the Safety Lead In group.
What this means
The results from this Phase 1/2 study provide initial safety data for the combination of APR-246 and pembrolizumab in patients with various solid tumor malignancies. The confirmation of a recommended Phase 2 dose for APR-246 at 4.5 g/d is a crucial step for potential further development of this combination therapy. These findings support the continued investigation of this regimen in future clinical trials, focusing on both safety and efficacy in specific tumor types.
Source
The information regarding these trial results was obtained from ClinicalTrials.gov, a public database of clinical studies. The results for study NCT04383938, titled "Phase 1/2 Study of APR-246 in Combination With Pembrolizumab in Subjects With Solid Tumor Malignancies," were posted on 2025-05-13 on clinicaltrials.gov.