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NCT04243785 · ClinicalTrials.gov registry record · Phase 1

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

A Phase 1 study of Acute Myeloid Leukemia and Myelodysplastic Syndrome, sponsored by Edgewood Oncology.

Recruiting
Registry status
Phase 1
Development phase
80
Enrollment target
3
Study locations

NCT04243785: Recruiting Phase 1 study of Acute Myeloid Leukemia and Myelodysplastic Syndrome, sponsored by Edgewood Oncology.

NCT04243785 is a Phase 1 study of Acute Myeloid Leukemia and Myelodysplastic Syndrome that is actively recruiting participants, run by Edgewood Oncology. The registered enrollment target is 80 participants, below the 116-participant average among 306 other Acute Myeloid Leukemia trials with a reported enrollment target (31% lower). The trial reports 3 study locations across 3 states. According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT04243785, a Phase 1 study of Acute Myeloid Leukemia and Myelodysplastic Syndrome, is actively recruiting participants, sponsored by Edgewood Oncology.

RECRUITING
Registry status
Phase 1
Development phase
80 participants
Enrollment target
3
Study locations

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).

Primary Outcome

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.

Conditions Studied

Acute Myeloid Leukemia Myelodysplastic Syndrome

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

FieldValue
Enrollment Target 80 participants
Start Date 2020-01-06
Est. Completion 2027-03
Phase Phase 1
Edgewood Oncology

2 total trials

What NCT04243785 shows while recruiting

NCT04243785 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 80 participants, a relatively small participant target, below the 116-participant average among 306 other Acute Myeloid Leukemia trials with a reported enrollment target (31% lower).

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.

NCT04243785 reports a single indexed study location in California, New York, Texas.

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.

What interventions are being tested in trial NCT04243785?

The interventions under investigation include: Azacitidine (DRUG), BTX-A51 (DRUG).

Who is sponsoring clinical trial NCT04243785?

This trial is sponsored by Edgewood Oncology, which has 2 total clinical trials registered on ClinicalTrials.gov.

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.

Learn More About Clinical Trials

Similar trials for Acute Myeloid Leukemia

Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.

Where NCT04243785's enrollment target sits among peer trials

80 97th of 306 higher than 206 of 306 other Acute Myeloid Leukemia trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Acute Myeloid Leukemia trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.

Source ClinicalTrials.gov registry export · 2026-08-08

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More recruiting context for NCT04243785

Source: ClinicalTrials.gov NCT04243785, the US trial registry maintained by the National Library of Medicine. NCT04243785 (small enrollment · single site footprint · recruiting) retrieved and formatted by PlainTrial, see methodology.