The AZALOX Study (PXS5505-MDS-101) is a multicenter, open-label Phase Ib/II study evaluating the safety and pharmacokinetics of escalating doses of the pan-LOX/LOXL inhibitor PXS-5505 in combination with 5-azacitidine. The study targets patients diagnosed with Myelodysplastic Neoplasms (MDS) or Chronic Myelomonocytic Leukemia (CMML). Primary endpoints for Phase Ib include dose-limiting toxicity (DLT), determination of the recommended Phase II dose (RP2D), and the overall safety profile. The trial enrolls adult patients with MDS or CMML in the first-line setting or following prior therapy, who are eligible for azacitidine treatment. Participants receive oral PXS-5505 in combination with 5-azacitidine. Key secondary endpoints include the assessment of overall response rate (ORR), duration of response (DOR), and hematological improvement (HI).
This international study is testing a new drug called PXS-5505 in combination with the standard treatment azacitidine for adults with blood cancers, specifically Myelodysplastic Neoplasms (MDS) or Chronic Myelomonocytic Leukemia (CMML). PXS-5505 is designed to reduce scarring (fibrosis) in the bone marrow to help improve blood cell production. First, the safest and best dose of PXS-5505 will be determined, followed by an investigation of its effectiveness. Participants include patients who are newly diagnosed with these conditions or whose disease has returned after initial therapy. All participants will receive the study drug along with azacitidine. The main goals are to assess the safety and tolerability of the combination treatment and to look for early signs of improvement in blood counts.
