Articles related to CLINICAL TRIALS

A 12-year odyssey of a cancer drug that works in a subcellular organelle: From discovery of a unique and promising tumor target – featured on the cover of the Journal of Clinical Investigation – through startup company, grant-writing, DoD funding, Wall Street disinterest, perseverance, and finally, approval to initiate clinical trials.

According to the final results from the phase 3 PALLAS trial, palbociclib offers no benefit over endocrine therapy alone in women with early HR+ HER2- breast cancer. The primary endpoint — 4-year invasive DFS — was nearly equivalent in the test and control arms of the study. None of the secondary endpoints showed a significant difference either. There was a high rate of palbociclib discontinuation (42%), mostly due to grade 3 or 4 neutropenia. But even in those who completed the two-year planned course showed no benefit over endocrine therapy alone. The results were not just disappointing, but surprising given that CDK 4/6 inhibitors plus endocrine therapy is the standard of care for advanced breast cancer.

This single-arm study of 40 patients with advanced neuroendocrine tumors (NETs) sought to evaluate the potential benefit of a VEGF inhibitor plus a PD-L1 inhibitor. The primary endpoint was objective radiographic response. Responses were seen in 15% to 20% of patients.

The phase 3 KEYNOTE-048 trial compares pembrolizumab with or without concurrent chemotherapy to cetuximab-chemotherapy in patients with recurrent or metastatic head and neck SCC. Patients were evaluated in subgroups based on PD-L1 expression. In median survival, pembrolizumab + chemotherapy outperformed cetuximab + chemotherapy in all patient subgroups regardless of PD-L1 expression. In the PD-L1 CPS 1-19 subgroup, pembrolizumab alone slightly outperformed cetuximab + chemotherapy. In the PD-L1 CPS < 1 subgroup median survival was significantly lower with pembrolizumab alone.

Data from a large phase III trial (Myeloma XI) were examined to determine the relationship between MRD status, PFS, and OS in post-ASCT patients randomly assigned to lenalidomide maintenance or no maintenance at 3 months after ASCT. At ASCT + 3 and ASCT + 9, MRD-negative status was associated with improved PFS and OS. Sustained MRD negativity from ASCT + 3 to ASCT + 9 or the conversion to MRD negativity by ASCT + 9 was associated with the longest PFS/OS.

An analysis by an Australian physician is making waves by suggesting that an infamous study of vitamin C treatments for sepsis may be fraudulent. The study author alleges that the pre- and post-comparison groups involved in the 94-patient study were too similar to be realistic and that the data were fabricated.