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RecruitingPhase 1

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

A Phase 1 Study of AUTX-703 in Participants With Relapsed/Refractory Acute Myeloid Leukemia and Myelodysplastic Syndromes

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
18 Years and older
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

Not stated in the study description — ask the study team about visits and procedures.

Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This early-stage study tests AUTX-703, a medicine taken by mouth, in adults with AML or MDS, cancers that affect blood-forming cells. It is for people whose disease has come back or has not responded to treatment. Researchers will study safe doses, side effects, how the body handles the medicine, and early signs that it may work.

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.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • You must be at least 18 years old.
  • You must have confirmed AML or MDS that has come back or resisted treatment. For AML, all approved treatments known to work for your disease must have failed to help enough or caused side effects you could not tolerate. For MDS, more than 10% of your bone marrow cells must be blasts (immature blood cells), and treatment must not have helped enough after at least 4 cycles of a hypomethylating drug regimen or another treatment known to work. You may also qualify if AML or MDS came back after a blood stem cell transplant.
  • You must be willing and able to follow study visits and treatment plans and have all study procedures unless they pose a medical risk.
  • You must be fully active or able to walk and do light work, even if you cannot do hard physical activity.
  • Your liver, kidneys, and heart must work well enough for the study.
  • Your white blood cell count must be 20 billion cells per liter or lower. Stable use of hydroxyurea is allowed.
  • You must meet the required waiting times after earlier treatments and surgery.
  • You must follow birth control rules. Men must use condoms during treatment and for 90 days afterward, even after a vasectomy. Women who can become pregnant must use one highly effective and one effective birth control method during treatment and for 180 days afterward.

Who can't join

  • You cannot give informed consent or follow study rules, or you have another cancer that could interfere with AML or MDS diagnosis or treatment.
  • You had CAR T-cell therapy (treatment using changed immune cells) or a blood stem cell transplant within 60 days before the first dose, or have active, significant graft-versus-host disease, where donor cells attack your body.
  • You have a severe active infection needing treatment, an unexplained fever above 38.5°C at screening or on the first treatment day, active hepatitis B or C with detectable virus, detectable HIV, or an AIDS-related illness within the past 6 months.
  • You are sensitive to AUTX-703 or its ingredients. Certain medicines affecting CYP3A4, P-gp, or BCRP, which help the body handle drugs, are not allowed within 14 days before the first dose. If you take a proton pump inhibitor for stomach acid, you must switch to another acid-reducing medicine.
  • You have an uncontrolled illness or active, severe heart or blood vessel disease (Class III or IV) within 6 months before treatment starts.
  • You cannot take medicine by mouth, or digestive problems would keep your body from properly absorbing, processing, or clearing it.
  • You are pregnant, breastfeeding, or planning to become pregnant within 1 year after study treatment starts.
  • You have a history of interstitial lung disease or pneumonitis, conditions that cause lung damage or swelling.

Simplified for readability. The official criteria below are what the study team uses.

Official 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, 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) 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 0-1 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 15. Have a history of interstitial lung disease or pneumonitis.

Study sites (9)

CA

FL

NC

NY

OH

PA

TN

TX