Phase 1 Multicenter Open-Label Study of REM-422, an MYB mRNA Degrader, in Relapsed/Refractory Acute Myeloid Leukemia or Higher-Risk Myelodysplastic Syndromes

The primary objective of this study is to evaluate the safety and tolerability of **REM-422**, an MYB mRNA degrader, in patients with **relapsed/refractory acute myeloid leukemia (AML)** or **higher-risk myelodysplastic syndromes (MDS)**. This is clinically relevant as these conditions are associated with poor prognosis and limited treatment options, necessitating the development of novel therapeutic strategies. The study aims to determine the maximum tolerated dose and identify any dose-limiting toxicities, which are critical for establishing a safe dosage regimen for future clinical trials.

The clinical trial involves a total of **80 participants** diagnosed with **relapsed/refractory acute myeloid leukemia (AML)** or higher-risk myelodysplastic syndromes (MDS). The study population includes both male and female subjects, with an age range encompassing adults and older adults. Participants were selected based on specific criteria, although detailed inclusion or exclusion criteria are not provided. The trial includes a vulnerable population, indicating that special considerations are in place for the protection of these individuals. Lifestyle factors such as diet, physical activity, or habits are not specified in the available data. The sponsor has not provided information regarding the main objective of the trial.

The clinical trial is a **Phase 1**, multicenter, open-label study designed to evaluate the safety and efficacy of REM-422, an MYB mRNA degrader, in patients with **relapsed/refractory acute myeloid leukemia (AML)** or higher-risk **myelodysplastic syndromes (MDS)**. The trial is set to commence recruitment on September 1, 2024, and is anticipated to conclude by September 1, 2026. Participants will be involved in the study for the duration of the trial, with specific timelines for individual involvement determined by the study protocol and response to treatment.

The trial will include a sequence of study visits, beginning with an inclusion (screening) visit to assess eligibility based on predefined criteria. Following successful screening, participants will undergo a series of follow-up visits at regular intervals to monitor treatment response, safety, and any adverse events. The end-of-study visit will occur upon completion of the treatment period or if a participant meets criteria for early termination. Conditions that may lead to early termination include significant adverse reactions, disease progression, or withdrawal of consent by the participant.

Throughout the trial, data will be collected and analyzed to assess the primary and secondary endpoints, which are not specified in the provided data. The open-label design allows both investigators and participants to be aware of the treatment being administered, facilitating direct observation of the drug's effects. The study's methodology ensures rigorous monitoring and documentation to uphold the integrity and scientific validity of the trial outcomes.

The clinical trial documentation does not provide specific details regarding the experimental medication, including its **pharmaceutical form**, dosage, route, or frequency of administration. Additionally, there is no information available about any non-experimental treatments, such as standard-of-care therapy, placebo, or comparator treatment, that may be used in the study. Furthermore, the documentation lacks details on drug administration, dosing schedules, and participant compliance monitoring. As such, no further description of the treatments used in this clinical trial can be provided based on the available data.

The clinical trial is designed to assess efficacy in a Phase 3 study. The trial is scheduled to commence recruitment on September 1, 2024, with an estimated completion date of September 1, 2026. The efficacy assessment will be conducted through a series of pre-defined parameters, although specific endpoints and methods for measuring efficacy are not detailed in the provided data. The trial will follow a structured timeline to ensure systematic data collection and analysis. The focus will be on evaluating the treatment's impact on the specified medical condition, with results contributing to the understanding of its therapeutic potential. The trial's design adheres to rigorous standards typical of Phase 3 studies, ensuring that the findings will be robust and clinically relevant.