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A Study of BTX-A51 in People With Relapsed or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome
NCT04243785 · View on ClinicalTrials.gov ↗
Study Summary
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).
Conditions Studied
Interventions
- DRUG Azacitidine
- DRUG BTX-A51
Study Locations (3)
California
- City of Hope National Medical Center — Duarte
New York
- Memorial Sloan-Kettering Cancer Center — New York
Texas
- The University of Texas MD Anderson Cancer Center — Houston
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 80 participants |
| Start Date | 2020-01-06 |
| Est. Completion | 2027-03 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04243785
The ClinicalTrials.gov registry entry for NCT04243785 describes a study currently listed as recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 80 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Edgewood Oncology, which has 2 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 2 conditions, with Acute Myeloid Leukemia appearing as the primary indexed condition, and to 2 interventions — of which Azacitidine is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT04243785 reports 3 study locations spanning 3 distinct geographic areas — top geographies include California, New York, Texas. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT04243785 about?
NCT04243785 is a clinical study titled "A Study of BTX-A51 in People With Relapsed or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome". 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 i...
What is the current status of trial NCT04243785?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 80 participants. The study started on 2020-01-06. Estimated completion is 2027-03.
What conditions does trial NCT04243785 study?
This clinical trial studies the following conditions: Acute Myeloid Leukemia, Myelodysplastic Syndrome. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT04243785?
The interventions under investigation include: Azacitidine (DRUG), BTX-A51 (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT04243785?
This trial is sponsored by Edgewood Oncology, which has 2 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT04243785 being conducted?
This trial has 3 study locations across California, New York, Texas. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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