Phase 1 Study of AUTX-703 in Relapsed/Refractory AML and MDS

Launched by AURON THERAPEUTICS, INC. · Feb 20, 2025

Keywords

ClinConnect Summary

This clinical trial is testing a new medication called AUTX-703 for patients with advanced blood cancers, specifically relapsed or refractory acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). The goal of the study is to see how safe the medication is, how well it is tolerated, and if it shows any signs of effectiveness in treating these conditions. The trial is currently open for participants aged 18 and older who have not responded well to existing treatments or have certain other specific health conditions.

To be eligible for the trial, participants must have a confirmed diagnosis of relapsed or refractory AML or MDS and must meet specific health criteria, such as having stable organ function and being able to follow the study requirements. Participants will take the medication by mouth and will have regular visits for assessments throughout the study. It's also important to note that both men and women who can become pregnant will need to use effective contraception during the trial and for a period afterward. This study is a chance to contribute to important research that may help improve treatment options for others facing these challenging cancers.

Gender

ALL

Eligibility criteria

• Key Inclusion Criteria:
• • 1. Participant must be ≥18 years of age
• 2. 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)
• • 3. Participant must be willing and able to comply with scheduled study visits and treatment plans.
• • 4. Participant must be willing to undergo all study procedures unless contraindicated due to medical risk.
• • 5. Participant must have an Eastern Cooperative Oncology Group (ECOG) performance status (PS) of ≤2
• • 6. Participant must have adequate hepatic function
• • 7. Participant must have adequate renal function
• • 8. Participant must have adequate cardiovascular function
• • 9. Participant must have a white blood cell (WBC) count ≤20 × 10⁹/L (with stable hydroxyurea use allowed)
• • 10. Participant must meet timing requirements with respect to prior therapy and surgery
• • 11. 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.
• Key Exclusion Criteria:
• • 1. Participant is unable to provide informed consent and/or to follow protocol requirements.
• • 2. Participant has undergone chimeric antigen receptor T cell therapy or HSCT within 60 days of the first dose of study treatment or has active clinically significant graft-versus-host disease (GVHD)
• • 3. Participant has another malignancy that may interfere with diagnosis and treatment of R/R AML or R/R MDS.
• • 4. Participant has an active severe infection that requires anti-infective therapy or has an unexplained temperature of \>38.5°C during screening visits or on their first day of study treatment.
• • 5. Participant has a known sensitivity to AUTX-703 or any of its components.
• • 6. Participant is taking systemic strong CYP3A4 inhibitors or inducers within 14 days of the first dose of study treatment.
• • 7. Participant who are taking proton pump inhibitors should be switched to another acid-reducing agent such as an antacid or H2 blocker
• • 8. Participant is taking P-gp and breast cancer resistance protein (BCRP) inhibitors or inducers within 14 days of first dose of study treatment.
• • 9. Participant has active hepatitis B virus (HBV) or hepatitis C virus (HCV) infections with detectable viral load
• • 10. Participant has experienced AIDS related illness within the past 6 months or have detectable HIV viral load.
• • 11. Participant has an uncontrolled intercurrent illness
• • 12. Participant has active Class III or IV cardiovascular disease within 6 months prior to the start of study treatment
• • 13. Participant is unable to tolerate the administration of oral medication or has GI dysfunction that would preclude adequate absorption, distribution, metabolism, or excretion of an oral medication
• • 14. Participant is pregnant or breastfeeding or is planning to become pregnant within 1 year of the start of study treatment