Evaluating BTX-A51 for patients with relapsed or refractory acute myeloid leukemia or high-risk myelodysplastic syndrome

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This open-label, dose escalation study aims to assess the safety, toxicity, and pharmacokinetics of BTX-A51 capsules in individuals with relapsed or refractory acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS). The study is divided into three parts: the first part focuses on determining the maximum tolerated dose (MTD) through monotherapy dose escalation, followed by a cohort expansion to gather more data on safety and efficacy. The final part will explore the combination of BTX-A51 with Azacitidine to further evaluate its effects. Up to 80 participants will be enrolled throughout the study phases.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Demonstration of understanding and voluntarily signing of an informed consent form
* Age ≥ 18 years
* Diagnosis of acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) according to the World Health Organization classification and, with respect to MDS, that is high risk; participants must have refractory or relapsed disease and be ineligible for or have exhausted standard therapeutic options that would otherwise be likely to provide clinical benefit
* Eastern Cooperative Oncology Group performance status ≤ 2 and life expectancy of ≥ 6 weeks
* Adequate organ function (Grade 1 serum creatinine; Grade 1 total bilirubin; aspartate aminotransferase and/or alanine transaminase ≤ 2 × ULN)
* Females of childbearing age must not be pregnant at time of Screening/beginning of treatment and agree to either abstain from sexual intercourse or use highly effective methods of contraception (for up to 3 months after last dose of study drug)
* Males sexually active with a woman of childbearing age must agree to use barrier method of birth control during and after the study (up to 3 months after last dose of study drug)

Exclusion Criteria:

* Diagnosis of acute promyelocytic leukemia
* White blood cell count \> 20 x 10\^9/L
* Receipt of cancer chemotherapy (other than hydroxyurea) within 2 weeks prior to the start of study drug
* In participants who have undergone autologous or allogeneic stem cell transplantation: transplantation within the 3 months prior to Screening; active graft-versus-host disease requiring anything other than topical corticosteroids and budesonide; treatment with systemic immunosuppressive medications including high-dose steroids (≥ 20 mg prednisolone or equivalent per day), or calcineurin inhibitors (e.g., cyclosporine, tacrolimus) for at least 1 week prior to Screening, and sirolimus, mycophenylate mofetil, azathioprine, or ruxolitinib for at least 2 weeks prior to Screening
* Immediate life-threatening severe complications of leukemia such as uncontrolled bleeding, pneumonia with hypoxia or shock, and/or disseminated intravascular coagulation
* Persistent toxicities from prior treatment of Grade 2 or higher
* Active uncontrolled systemic fungal, bacterial, mycobacterial, or viral infection
* Clinically significant cardiac disease
* Known dysphagia, short-gut syndrome, gastroparesis, or other conditions that limit the ingestion or gastrointestinal absorption of drugs administered orally
* Any other concurrent medical condition or disease that is likely to interfere with study procedures or results, or that, in the opinion of the Investigator, would constitute a hazard for participating in this study
* If female, pregnant or breastfeeding

Where it's running

• City of Hope National Medical Center
  Duarte, California, United States Recruiting
• Memorial Sloan-Kettering Cancer Center
  New York, New York, United States Recruiting
• The University of Texas MD Anderson Cancer Center
  Houston, Texas, United States Recruiting

Study contacts

• Study coordinator: Zung Thai, MD
• Email: zung@edgewoodonc.com
• Phone: 415-225-9338

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.