Testing AUTX-703 in adults with relapsed or hard-to-treat leukemia or myelodysplastic syndromes

Trial details

Who runs it

Auron Therapeutics, Inc.

Trial code

NCT06846606EU 2025-521394-13-00

Record updated

16 October 2026

Original description

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.Read full original descriptionShow less

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.

Inclusion: Participant must be ≥18 years of age; Participant must have confirmed diagnosis as follows:; R/R AML and has not achieved adequate response to, or cannot tolerate, 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 0-1; 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.

Exclusion: Participant is taking systemic strong CYP3A4 inhibitors or inducers within 14 days of the first dose of study treatment; Participant who are taking proton pump inhibitors should be switched to another acid-reducing agent such as an antacid or H2 blocker; Participant has active Class III or IV cardiovascular disease within 6 months prior to the start of study treatment; 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; 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); Participant has another malignancy that may interfere with diagnosis and treatment of R/R AML or R/R MDS; 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; Participant is taking P-gp and breast cancer resistance protein (BCRP) inhibitors or inducers within 14 days of first dose of study treatment; Participant has active hepatitis B virus (HBV) or hepatitis C virus (HCV) infections with detectable viral load; Participant has experienced AIDS related illness within the past 6 months or have detectable HIV viral load; Participant has an uncontrolled intercurrent illness; Have a history of interstitial lung disease or pneumonitis; Participant is unable to provide informed consent and/or to follow protocol requirements; Participant has a known sensitivity to AUTX-703 or any of its components; Participant is pregnant or breastfeeding or is planning to become pregnant within 1 year of the start of study treatment.

ClinicalTrials.gov NCT06846606 · EU CTIS 2025-521394-13-00