PHASE 3B STUDY DESIGN: COMPARING TREATMENT PREFERENCE BETWEEN ORAL DECITABINE/CEDAZURIDINE AND AZACITIDINE IN PATIENTS WITH MYELODYSPLASTIC SYNDROME, LOW-BLAST ACUTE MYELOID LEUKAEMIA, OR CHRONIC MYELOMONOCYTIC LEUKAEMIA

Abstract

Introduction: There is uncertainty about how choice of therapy in myelodysplasia (MDS), low-blast count AML (LB-AML) or chronic myelomonocytic leukaemia (CMML) is made by patients, physicians and carers when >1 treatment type is available. This study's primary objective is to compare patients’ treatment preference using the patient ‘treatment preference in myelodysplasia questionnaire’ (pTPMQ). Secondary objectives include evaluation of preference by carers (cTPMQ), and clinicians (mTPMQ). Methods: This phase 3b, open-label, multi-centre study (NCT05883956), with sites in Australia and New Zealand, will compare preference between oral decitabine/cedazuridine (Treatment A) and SC AZA (Treatment B). The study design includes 28 days of screening, four continuous 28-day cycles of study treatment, and a follow-up period with two 28-day cycles of continued therapy. Patients (N=42) will be randomised to two balanced treatment sequences: ABBA or BAAB (Figure 1). Patients will express a preference twice in the study, first after completing Cycle 2, then after completing Cycle 4. This will be assessed via the pTPMQ. Clinician and carer preference will also be assessed. Results: Trial to be activated in Australia and New Zealand, with first patient recruited in December 2023. We will report the primary and secondary objectives. Exploratory objectives including treatment discontinuation rates, quality of life and safety of SC AZA and oral decitabine/cedazuridine will also be reported. Conclusions: This study aims to address an evidence gap in the comparison of patients’, carers’ and clinician's preference between an oral and a parenteral treatment, preference strength, and the reasons for it.