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BTX-A51 for Leukemia

Phase 1
Recruiting
Research Sponsored by Edgewood Oncology Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Eastern Cooperative Oncology Group performance status ≤ 2 and life expectancy of ≥ 6 weeks
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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 2 years after participant's last dose of btx-a51 or upon death, whichever occurs first
Awards & highlights

Study Summary

This trial is testing a new drug, BTX-A51, to see if it is safe and effective in people with relapsed or refractory acute myeloid leukemia or high-risk myelodysplastic syndrome. The study will be done in two phases. Phase 1a will determine the dose-limiting toxicities and maximum tolerated dose of orally administered BTX-A51 in up to 35 participants who are evaluable for toxicity. Once the maximum tolerated dose is determined, it is planned that an additional 15 participants will be enrolled in Phase 1b for additional experience with safety and efficacy, and to determine the recommended Phase

Who is the study for?
Adults (18+) with relapsed or refractory acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS), who have no other standard treatment options. Participants must be able to understand the study and consent, have a life expectancy of at least 6 weeks, and adequate organ function. Women must not be pregnant and agree to contraception; men must use barrier birth control.Check my eligibility
What is being tested?
The trial is testing BTX-A51 capsules for safety, toxicity, pharmacokinetics, and preliminary efficacy in two phases: dose escalation to find the maximum tolerated dose and a continuation phase for further safety and efficacy data. Patients may receive up to eight cycles of treatment.See study design
What are the potential side effects?
While specific side effects are not listed here, participants will be monitored for drug-related toxicities such as potential organ damage or dysfunction due to medication intake which could manifest in various ways depending on individual tolerance.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I can care for myself and doctors expect me to live more than 6 weeks.
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I have AML or high-risk MDS that is not responding to standard treatments.
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My kidney and liver functions are within normal limits.
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I am 18 years old or older.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 2 years after participant's last dose of btx-a51 or upon death, whichever occurs first
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 2 years after participant's last dose of btx-a51 or upon death, whichever occurs first for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Incidence of dose-limiting toxicities (DLTs)
Maximum tolerated dose (MTD)
Number of participants with 12-lead electrocardiogram (ECG) abnormalities and/or AEs
+6 more
Secondary outcome measures
Complete remission (CR) for participants with acute myeloid leukemia (AML)
Muscular Dystrophy
Complete remission with incomplete blood count recovery (CRi) for participants with AML
+14 more

Side effects data

From 2023 Phase 1 trial • 48 Patients • NCT02038777
67%
Pyrexia
67%
Pneumonia
33%
Enteritis infectious
33%
Hyperaesthesia teeth
33%
Nausea
33%
Infective tenosynovitis
33%
Bronchitis
33%
Bacteraemia
33%
Rhinitis
33%
Blood creatine phosphokinase increased
33%
Prothrombin time prolonged
33%
Dysgeusia
33%
Muscle spasms
33%
Auricular chondritis
33%
Small intestinal haemorrhage
33%
Malaise
33%
Oedema
33%
Influenza
33%
Nasopharyngitis
33%
Phlebitis
100%
80%
60%
40%
20%
0%
Study treatment Arm
Monotherapy Cohort: PF-04449913 25 mg
Monotherapy Cohort: PF-04449913 50 mg
Monotherapy Cohort: PF-04449913 100 mg
Combination Cohort 1 (Unfit Participants): PF-04449913 100 mg + LDAC 20 mg
Combination Cohort 2 (Fit Participants): PF-04449913 100 mg + Cytarabine + Daunorubicin
Combination Cohort 3: PF-04449913 100 mg + Azacitidine
Continuation Cohort (Monotherapy Cohort): PF-04449913 100 mg
Expansion Cohort (Unfit Participants): PF-04449913 100 mg+ LDAC 20 mg

Trial Design

3Treatment groups
Experimental Treatment
Group I: Part 1c (Azacitidine Combination Dose Escalation)Experimental Treatment2 Interventions
After determination of MTD and RP2D from Part 1a, combination dose escalation in Part 1c may begin. Patients with AML will receive BTX-A51 combined with azacitidine in escalating BTX-A51 dose cohorts. Dosing in this stage of the study consists of the first cycle of therapy (i.e., 28 days). The starting dose of BTX-A51 will be RP2D. Part 1c will follow a BOIN design as described for Part 1a. The numbers of patients and actual doses administered will be determined in response to DLTs a. There will be at least 3 patients per cohort.
Group II: Part 1b (Monotherapy Cohort Expansion)Experimental Treatment1 Intervention
Dosing in this phase of the study consists of the first cycle of therapy (i.e., 28 days consisting of 3 weeks of treatment followed by 1 week with no study drug). Part 1b will continue at the MTD or the highest dose achieved in Phase 1a.
Group III: Part 1a (Monotherapy Cohort Escalation)Experimental Treatment1 Intervention
Dosing in this phase of the study consists of the first cycle of therapy (i.e., 28 days consisting of 3 weeks of treatment followed by 1 week with no study drug). The BTX-A51 starting dose for Cohort 1 is 1 mg, to be given 5 days per week (maximum weekly dose of 5 mg). Beginning with Cohort 2, doses are intended to be administered 3 days per week. Barring dose-limiting toxicity (DLT), sequential dose escalation of BTX-A51 is planned with up to a total of eight dose levels to a maximum of 21 mg (63 mg/week); on the basis of these an MTD will be identified. The numbers of participants and actual doses administered will be determined using a Bayesian optimal interval (BOIN) design to determine the DLTs and MTD of BTX-A51.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Azacitidine
2012
Completed Phase 3
~1440

Find a Location

Who is running the clinical trial?

Edgewood Oncology Inc.Lead Sponsor
1 Previous Clinical Trials
112 Total Patients Enrolled
BioTheryX, Inc.Lead Sponsor
2 Previous Clinical Trials
120 Total Patients Enrolled
Zung Thai, MDStudy DirectorEdgewood Oncology Inc.
5 Previous Clinical Trials
700 Total Patients Enrolled

Media Library

BTX-A51 (Other) Clinical Trial Eligibility Overview. Trial Name: NCT04243785 — Phase 1
Acute Myeloid Leukemia Research Study Groups: Part 1b (Monotherapy Cohort Expansion), Part 1c (Azacitidine Combination Dose Escalation), Part 1a (Monotherapy Cohort Escalation)
Acute Myeloid Leukemia Clinical Trial 2023: BTX-A51 Highlights & Side Effects. Trial Name: NCT04243785 — Phase 1
BTX-A51 (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04243785 — Phase 1

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is recruitment underway for this experiment?

"According to the records on clinicaltrials.gov, this medical trial is actively searching for participants and was initially posted on January 6th 2020, with the last update occurring on September 8th 2022."

Answered by AI

What is the current enrollment count for this trial?

"This trial requires 50 suitable candidates, who meet the eligibility requirements. They can be recruited from various locations including the Memorial Sloan-Kettering Cancer Center in New york City and the City of Hope National Medical Centre situated in Duarte, California."

Answered by AI

What potential adverse effects could result from the utilization of BTX-A51?

"As this is an experimental Phase 1 trial, our team at Power assessed BTX-A51's safety to be a low score of 1 due to the limited evidence backing its efficacy or safety."

Answered by AI

What results is the research team seeking to attain through this trial?

"This clinical trial will span 224 days and evaluate the maximum tolerated dose of a new drug. Secondary objectives include measuring the maximum observed plasma concentration, complete remission in participants with acute myeloid leukemia (AML) through analysis of absolute neutrophil count, platelet count, bone marrow blasts and circulating blast cells; as well as estimating terminal elimination phase half-life on Day 5 after initial dose administration."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
A Study of BTX-A51 in People With Relapsed or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome
2020. "A Study of BTX-A51 in People With Relapsed or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04243785.
~24 spots leftby Mar 2026
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