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Study of BTX-A51 for Relapsed/Refractory AML or High-Risk MDS

This study is testing a new oral medication called BTX-A51 for people with acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS) that has returned or not responded to previous treatments. The study aims to find the safest and most effective dose of BTX-A51, sometimes given with Azacitidine (a chemotherapy drug). Researchers will look at side effects and how well the treatment works. You may be able to join if you are 18 or older and have relapsed or refractory AML or high-risk MDS. The study is currently enrolling up to 80 participants.

Study design
This is an open-label study, meaning you and your doctors will know which treatment you are receiving. It involves increasing doses of BTX-A51 to find the safest amount, and plans to enroll up to 80 participants.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Researchers will monitor side effects and laboratory results for up to eight 28-day cycles (approximately 224 days).

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT04243785

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

Recruiting
PHASE1Ages 18+InterventionalTreatment
Edgewood Oncology Inc.
~80 participants
Updated 2024-03-20 on ClinicalTrials.gov
What's tested:BTX-A51Azacitidine

At a glance

Recruiting sites
3 of 3 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence of dose-limiting toxicities (DLTs)
Measured over Up to a total of eight 28-day cycles (approximately 224 days)
+8 more outcomes measured

Conditions

Acute Myeloid Leukemia
Myelodysplastic Syndrome

Explore related trials

Acute Myeloid LeukemiaMyelodysplastic Syndrome

Where it's being run

3 sites across 3 states
California1
New York1
Texas1

Study leadership

  • Zung Thai, MD · STUDY_DIRECTOR · Edgewood Oncology Inc.

Who to contact

Zung Thai, MD
[email protected]
415-225-9338
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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

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

What this trial measures

  • Incidence of dose-limiting toxicities (DLTs)Up to a total of eight 28-day cycles (approximately 224 days)

    A DLT is defined as a severe or clinically significant adverse event (AE) or abnormal laboratory value (Grade 3 or greater, unless otherwise specified) starting with the first dose on Cycle 1 Day 1, unless it is clearly related to disease progression, intercurrent illness, preexisting condition, or concomitant medications. DLTs are based on National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0.

  • Number of participants with non-serious AEs and serious AEs (SAEs)Up to a total of eight 28-day cycles (approximately 224 days)

    The severity/intensity of AEs will be graded based upon the participant's symptoms according to the NCI CTCAE Version 5.0

  • Number of participants with laboratory abnormalities and/or AEsUp to a total of eight 28-day cycles (approximately 224 days)

    Number of participants with potentially clinically significant laboratory values; toxicity will be graded and reported according to the NCI CTCAE Version 5.0

  • Number of participants with 12-lead electrocardiogram (ECG) abnormalities and/or AEsUp to a total of eight 28-day cycles (approximately 224 days)

    Number of participants with potentially clinically significant 12-lead ECG findings; toxicity will be graded and reported according to the NCI CTCAE Version 5.0

  • Number of participants with echocardiogram (ECHO) abnormalities and/or AEsUp to a total of eight 28-day cycles (approximately 224 days)

    Number of participants with potentially clinically significant ECHO abnormalities and/or AEs, such as elevated or abnormal left ventricular ejection fraction (LVEF) or abnormal Global Longitudinal Strain (GLS)

  • Number of participants with vital sign abnormalities and/or AEsUp to a total of eight 28-day cycles (approximately 224 days)

    Number of participants with potentially clinically significant vital sign values; toxicity will be graded and reported according to the NCI CTCAE Version 5.0

  • Number of participants with physical examination abnormalities and/or AEsUp to a total of eight 28-day cycles (approximately 224 days)

    Number of participants with potentially clinically significant physical examination findings; toxicity will be graded and reported according to the NCI CTCAE Version 5.0

  • Maximum tolerated dose (MTD)Up to 28 days (one cycle) for each dosing cohort in Phase 1a

    The DLTs are to be evaluated for determination of the MTD. The MTD will be the dose for which the isotonic estimate of the toxicity rate is closest to the target toxicity rate of 0.3. If there are ties, the higher dose level when the isotonic estimate is lower than the target toxicity rate will be identified and the lower dose level selected when the isotonic estimate is greater than or equal to the target toxicity rate.

  • Recommended Phase 2 dose (RP2D)Up to 28 days (one cycle) for each dosing cohort in Phase 1b

    The DLTs are to be evaluated based on cumulative safety/PK data in participants treated in Phase 1b for determination of the RP2D (which may or may not differ from the MTD)