Yakushima

Researchers are studying bleximenib, an oral drug, in participants with acute leukemia to find the best dose and evaluate its safety and effectiveness. The study includes Phase 1 dose escalation to find recommended doses and Phase 1 dose expansion and Phase 2 to assess safety, tolerability, and anti-leukemia activity. Participants include both pediatric and adult patients with relapsed or refractory acute leukemia, especially those with specific genetic alterations. In Phase 1 Part 1, participants receive increasing doses of bleximenib orally to identify recommended doses based on tolerance. In Phase 1 Part 2, participants receive bleximenib at these doses to further evaluate safety. Phase 2 participants take the recommended dose to study the drugs effect on leukemia. The study monitors participants up to 4 years and 9 months for safety and treatment response. Participants will undergo assessments including monitoring adverse events, dose-limiting toxicities, and treatment responses. Blood tests will measure drug levels and leukemia remission rates. Safety and efficacy are tracked throughout treatment and follow-up, with a focus on remission rates and survival outcomes. The total study duration extends to September 2030, allowing long-term evaluation of bleximenib.

Researchers are evaluating the combination of bleximenib, venetoclax VEN, and azacitidine AZA compared to placebo with VEN and AZA in treating adults with newly diagnosed Acute Myeloid Leukemia AML who have mutations in the NPM1 or KMT2A genes. This Phase 3 study focuses on participants who are not eligible for intensive chemotherapy due to age or other health conditions. The goal is to understand how these treatments work in this specific AML population. Participants receive treatment in 28-day cycles, either with bleximenib plus VEN and AZA or placebo plus VEN and AZA. Bleximenib, VEN, and placebo are taken orally, while AZA is given intravenously or under the skin. Treatment continues until disease progression or unacceptable side effects occur. During the study, participants will be monitored for response to treatment including complete remission and overall survival for up to over four years. Researchers will track event-free survival, duration and timing of remission, transfusion independence, and other health outcomes. Safety is also closely observed through adverse events and lab tests. Participation involves regular visits for treatment and assessments over the study period.

Researchers are evaluating the addition of bleximenib to standard chemotherapy for adults with newly diagnosed acute myeloid leukemia AML who have specific genetic mutations in the NPM1 or KMT2A genes. This phase 3, randomized, double-blind, placebo-controlled trial aims to see if bleximenib can improve treatment outcomes compared to chemotherapy alone. The study includes patients eligible for intensive chemotherapy and focuses on those with DNA changes in their leukemia cells. Participants will receive standard chemotherapy combined with either bleximenib or a placebo. Treatment includes remission induction and consolidation therapy, followed by maintenance therapy with either bleximenib or placebo depending on the group. Treatment will continue until disease progression, unacceptable side effects, or other criteria require stopping. The study involves three groups one receiving bleximenib throughout, one receiving bleximenib during initial therapy and placebo for maintenance, and one receiving placebo throughout. Participants will be monitored for up to 4 years and 5 months to assess event-free survival, with additional follow-up up to nearly 8 years to evaluate overall survival and remission rates. Researchers will collect data on treatment response, remission duration, and the number of participants undergoing stem cell transplants. Safety and long-term outcomes will be observed to understand the effects of adding bleximenib to standard therapy.