NCT06846606 · Auron Therapeutics, Inc.
Phase 1 Study of AUTX-703 in Relapsed/Refractory AML and MDS
What this study is about
This Phase 1, conducted at multiple hospitals, where both patients and doctors know the treatment given, gradually increasing doses and dose optimization study is designed to assess the safety, tolerability, how the drug moves through the body (PK), how the drug affects the body (PD), and preliminary clinical activity of AUTX-703 administered taken by mouth in subjects with advanced hematologic malignancies.
View original scientific 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.
Interventions
DRUG
AUTX-703
AUTX-703 administered orally
Primary outcome measures
Incidence of Adverse Events (AEs), Dose-Limiting Toxicities (DLTs), and Serious Adverse Events (SAEs)
Time frame: From the first dose through 28 days after the last dose of study drug.
To assess the safety and tolerability of AUTX-703 by evaluating the incidence and severity of AEs, DLTs, SAEs, and AEs leading to treatment discontinuation.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 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. Key
Exclusion criteria
- Participant is unable to provide informed consent and/or to follow protocol requirements.
- 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 has a known sensitivity to AUTX-703 or any of its components.
- 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 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
- 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 is pregnant or breastfeeding or is planning to become pregnant within 1 year of the start of study treatment
Where
- Duarte, California
- Tampa, Florida
- Buffalo, New York
- New York, New York
- Chapel Hill, North Carolina
- Columbus, Ohio
- Philadelphia, Pennsylvania
- Nashville, Tennessee
- Houston, Texas
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Sep 2, 2026 · Source of record for eligibility and locations