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RecruitingPhase 1

A Study of BTX-A51 in People With Relapsed or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

A First-In-Human, Open-Label, Escalating Multiple-Dose Study to Evaluate the Safety, Toxicity, and Pharmacokinetics of BTX-A51 Alone and in Combination With Azacitidine in Patients With Relapsed or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

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

  • Time periods mentioned: 2 years

Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.

Show the study's own words
  • "Once BTX-A51 treatment has completed, participants will be contacted by telephone every 3 months for up to 2 years after their last treatment for survival status and anticancer therapy (Overall Survival Follow-up)."
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 study tests the safety, side effects, and doses of BTX-A51 capsules, alone and with azacitidine. It also checks how the body handles the drug and whether it may help treat the cancer. It is for adults with acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS), a bone marrow disease, that has come back or has not responded to treatment.

This is an open-label, dose escalation study to evaluate the safety, toxicity, and pharmacokinetics (PK) as well as preliminary efficacy of BTX-A51 capsules in participants with relapsed or refractory acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS). The study will be done in three parts. Part 1a (Monotherapy Dose Escalation) of this study is designed to determine the dose-limiting toxicities (DLTs) and maximum tolerated dose (MTD) of orally administered BTX-A51 in up to 35 participants who are evaluable for toxicity. Once the MTD is determined, it is planned that an additional 15 participants will be enrolled in Part 1b (Monotherapy Cohort Expansion) of this study for additional experience with safety and efficacy, and to determine the recommended Phase 2 dose (RP2D) which may or may not be different from the MTD. After determination of MTD and RP2D from Part 1a, Part 1c (Azacitidine Combination Dose Escalation) will enroll up to 30 participants. Continued treatment will be available under this study protocol for up to eight 28-day cycles (Continued Treatment Phase) if the Investigator judges the benefit outweighs the risk. Once BTX-A51 treatment has completed, participants will be contacted by telephone every 3 months for up to 2 years after their last treatment for survival status and anticancer therapy (Overall Survival Follow-up).

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

Eligibility

Who can join

  • Be at least 18 years old.
  • Understand the study and freely sign a consent form.
  • Have AML or high-risk MDS that has come back or has not responded to treatment. Standard treatments must be unsuitable or already used up.
  • Be able to care for yourself and be out of bed for at least half the day. Be expected to live at least 6 weeks.
  • Have kidney and liver blood test results within the study’s limits.
  • If you can become pregnant, you must not be pregnant at screening or when treatment starts. Avoid sex or use highly effective birth control through 3 months after the last dose.
  • If you are a man having sex with a woman who can become pregnant, use a barrier birth control method, such as a condom, through 3 months after the last dose.

Who can't join

  • Have acute promyelocytic leukemia, a type of AML, or a white blood cell count above 20 billion per liter.
  • Had cancer chemotherapy within 2 weeks before starting the study drug, except hydroxyurea.
  • Had a stem cell transplant within 3 months before screening, have certain active problems from donor cells attacking your body, or have used certain immune-suppressing drugs around screening after a transplant.
  • Have life-threatening problems from leukemia, such as uncontrolled bleeding, severe pneumonia, or serious blood-clotting problems.
  • Still have moderate or worse side effects from earlier treatment, or have an active infection that is not controlled.
  • Have serious heart disease or another health problem that could make the study unsafe or affect its procedures or results.
  • Have trouble swallowing or a condition that limits how well your body takes in drugs by mouth.
  • Are pregnant or breastfeeding.

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

Official eligibility criteria

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

Study sites (3)

CA

NY

TX