OncoMatch/Clinical Trials/NCT04243785
A Study of BTX-A51 in People With Relapsed or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome
Is NCT04243785 recruiting? Yes, currently enrolling (May 2026). This Phase 1 trial studies multiple treatments including BTX-A51 and Azacitidine for acute myeloid leukemia.
Treatment: BTX-A51 · Azacitidine — 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).
Check if I qualifyExtracted eligibility criteria
Cancer type
Acute Myeloid Leukemia
Myelodysplastic Syndrome
Performance status
ECOG 0–2(Ambulatory, capable of self-care)
Prior therapy
Must have received: standard therapeutic options
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
Cannot have received: cancer chemotherapy (other than hydroxyurea)
Receipt of cancer chemotherapy (other than hydroxyurea) within 2 weeks prior to the start of study drug
Cannot have received: autologous or allogeneic stem cell transplantation
transplantation within the 3 months prior to Screening
Lab requirements
Kidney function
grade 1 serum creatinine
Liver function
grade 1 total bilirubin; aspartate aminotransferase and/or alanine transaminase ≤ 2 × uln
Structured fields extracted by AI. May contain errors — verify against the official protocol.
US trial sites
- City of Hope National Medical Center · Duarte, California
- Memorial Sloan-Kettering Cancer Center · New York, New York
- The University of Texas MD Anderson Cancer Center · Houston, Texas
Showing up to 5 US sites. See all sites on ClinicalTrials.gov →
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