ASC4START

Chronic myeloid leukemia (CML) is a malignant disease affecting a subtype of white blood cells. “Chronic” means that the disease progresses over a long period of time, in contrast to acute leukemia. The course of the disease is divided into several phases. All participants in this study should be in the first phase, known as the chronic phase. In this phase, the leukemia is still stable and responds well to treatment. In 95% of CML cases, the so-called Philadelphia chromosome can be detected. This is a disease-related mutation in the genetic material of the blood cells. As a result, based on the abnormal genetic material, an abnormal protein (known as tyrosine kinase) is produced in the cell, which drives uncontrolled cell proliferation and is a key factor in the development of CML. In general, therapeutic options have improved significantly since the discovery of the Philadelphia chromosome, and CML is now, unlike in the past, highly treatable in most cases. The new CML medications target the “abnormal” tyrosine kinase, including nilotinib and asciminib. They differ only in the mechanisms by which they inhibit the enzyme. Nilotinib belongs to the second generation of tyrosine kinase inhibitors and is already used as the standard treatment, whereas asciminib belongs to the newly developed third generation, which is said to be better tolerated but may be slightly less effective. The goal of the ASC4START study is to compare the current standard of care (nilotinib) with the new tyrosine kinase inhibitor asciminib, with a particular focus on tolerability. The study will measure the length of time until treatment must be interrupted due to a side effect. The study will last a maximum of 4.5 years. There are two study arms; assignment to these arms is random and the study is open-label, meaning all participants are aware of which arm they are in. Asciminib is administered once daily, and nilotinib twice daily. Eligible participants are adults who have recently been diagnosed with Philadelphia chromosome-positive CML in the chronic phase (no more than 3 months after diagnosis) and have not yet received treatment. Facts: Disease: chronic myeloid leukemia (CML) Cancer characteristics: newly diagnosed, Philadelphia chromosome-positive (Ph+), untreated, chronic phase (CP) What the study is investigating: Asciminib 80 mg once daily vs. Nilotinib 300 mg twice daily Study objective: To evaluate the tolerability of asciminib (3rd-generation tyrosine kinase inhibitor) compared to the current standard of care (nilotinib, 2nd-generation TKI) Study duration: 4.5 years Study characteristics: 2 study arms, random assignment (Phase 3: randomization), open-label (assignment known)