Asciminib as Single Agent or Combined With Nilotinib in Newly Diagnosed BCR-ABL1+ Chronic Myeloid Leukemia A Randomized Phase II PEARL Study

What this Trial Is About

Researchers are evaluating the effectiveness and safety of asciminib alone or combined with nilotinib in patients newly diagnosed with chronic phase chronic myeloid leukemia (CP CML). This phase 2, randomized, unblinded study aims to compare asciminib given as a single agent or alongside nilotinib to understand their impact on treatment response and remission. The trial includes a core treatment phase, consolidation phase, and a treatment-free remission phase to assess long-term outcomes. The study has two treatment groups: one receives asciminib 80 mg once daily alone, and the other starts with asciminib 80 mg once daily for 90 days, then switches to asciminib 40 mg twice daily combined with nilotinib 300 mg twice daily or once daily depending on side effects. The core phase lasts two years, followed by a two-year consolidation phase where asciminib continues at the same dose and nilotinib dose is reduced in the combination group. Participants maintaining deep molecular remission (MR4) by the fourth year may stop treatment and enter a treatment-free remission phase lasting one year. During the study, participants will undergo regular monitoring of disease markers using quantitative PCR to track residual disease and treatment response. Researchers will evaluate the response at two years as the primary outcome. Safety and side effects will be closely monitored, with dose adjustments or treatment discontinuation allowed for toxicity or treatment failure. Follow-up continues through the treatment-free remission phase to assess remission stability and relapse rates, with the total study duration extending up to approximately five years.

PEARL Study: PotEntial of Asciminib in the eaRly Treatment of CML