Clinical Trials | A Pivotal Open-label Phase 3 Clinical Study Evaluating the Efficacy and Safety of QTX-2101 in Combination With All-trans Retinoic Acid in Newly Diagnosed, Low-risk Acute Promyelocytic Leukemia

The University of Virginia is conducting a clinical research study for adults ages 18 and over who are newly diagnosed, low-risk acute promyelocytic leukemia. This study has two parts. The purpose of Part 1 of this study is to find out more about how QTX-2101 moves through your body and how safe it is for patients like you to take QTX-2101 (15 mg) together with ATRA (with food) during the consolidation stage of treatment for APL. The study will also measure how much of the drug is present in your blood. This information will help researchers determine the most appropriate dose to use in future studies. The purpose of Part 2 of this study is to assess if QTX-2101 is as safe and effective as IV ATO for the treatment of APL by measuring the cancer’s response to study treatment and seeing if there are any side effects. For part one of this study, you will have a maximum of 3 cycles of treatment. You will receive QTX-2101 by mouth once daily for 5 days per week (ie, Days 1 to 5 of each week) for 4 weeks of an 8-week cycle (4 weeks on and 4 weeks off). You will also receive ATRA, which is split into 2 doses daily, for 2 weeks (ie, ATRA dosing on Weeks 1 to 2 [Days 1 to 14] and Weeks 5 to 6 [Days 29 to 42] of Consolidation treatment Cycles 2 and 3, and Weeks 1 to 2 [Days 1 to 14] of Consolidation treatment Cycle 4 only). For part two of this study, you will be randomly assigned (like flipping a coin) to one of two study groups, as determined by an electronic system. Group 1 will receive the study drug (oral QTX-2101) with oral ATRA, and Group 2 will receive IV ATO with oral ATRA. Depending on your group assignment, you will receive the following treatment: Group 1: You will take oral QTX-2101 once daily with oral ATRA, which is split into 2 doses daily, until your cancer responds to the treatment or up to a maximum of 60 days. Group 2: You will receive IV ATO once daily with oral ATRA, which is split into 2 doses daily, until your cancer responds to the treatment or up to a maximum of 60 days. If your cancer does not respond to treatment by Day 60, you will stop taking the study treatment. If your cancer responds to the treatment by Day 60, you will continue onto the consolidation part of the study. The consolidation stage of the study is the part of treatment that is designed to kill any remaining APL cells and keep APL cells from coming back. During this stage of the study, you will undergo 4 cycles of treatment. Study-related procedures that are being done beyond your standard of care will be provided at no cost to you or your insurance. [email protected]