A Phase 1 Study of AUTX-703 in Participants With Relapsed/Refractory Acute Myeloid Leukemia and Myelodysplastic Syndromes
This Phase 1, multicenter, open-label, dose escalation and dose optimization study is designed to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity of AUTX-703 administered orally in subjects with advanced hematologic malignancies.
• Participant must be ≥18 years of age
• Participant must have confirmed diagnosis as follows:
• R/R AML and has not achieved adequate response to, cannot tolerate, or refused all approved therapies known to be active for treatment of their disease OR R/R MDS with over 10% blasts in the bone marrow and has not achieved an adequate response to at least 4 cycles of a hypomethylating agent (HMA)- containing regimen or other treatment known to be active for their disease OR R/R AML or R/R MDS that has relapsed after a hematopoietic stem cell transplant (HSCT)
• Participant must be willing and able to comply with scheduled study visits and treatment plans.
• Participant must be willing to undergo all study procedures unless contraindicated due to medical risk.
• Participant must have an Eastern Cooperative Oncology Group (ECOG) performance status (PS) of ≤2
• Participant must have adequate hepatic function
• Participant must have adequate renal function
• Participant must have adequate cardiovascular function
• Participant must have a white blood cell (WBC) count ≤20 × 10⁹/L (with stable hydroxyurea use allowed)
⁃ Participant must meet timing requirements with respect to prior therapy and surgery
⁃ Participant must agree to use effective contraception during the study and for the required post-treatment period: Males: Use condoms (even if vasectomized) during the study and for 90 days post-treatment. Females of childbearing potential: Use a combination of 1 highly effective and 1 effective method of contraception during the study and for 180 days post-treatment.