Actively Recruiting
A First-In-Human, Open-Label Study to Evaluate Safety, Toxicity, and Pharmacokinetics of BTX-A51 Alone and With Azacitidine in Relapsed or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome
Led by Edgewood Oncology Inc. · Updated on 2024-03-20
80
Participants Needed
3
Research Sites
52 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying BTX-A51, an oral capsule medicine, in people with relapsed or refractory acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS). This open-label, phase 1 study aims to evaluate the safety, side effects, how the drug moves through the body, and early signs of effectiveness. The study is sponsored by Edgewood Oncology Inc. and involves participants who have limited treatment options left. The study has three parts. Part 1a involves increasing doses of BTX-A51 alone to find the highest safe dose and dose-limiting toxicities during 28-day cycles of treatment, with 3 weeks of medicine followed by 1 week without. Part 1b expands this dose group to gather more safety and efficacy data. Part 1c explores combining BTX-A51 at the recommended dose with azacitidine, given by injection on days 1 to 7 of each 28-day cycle. Treatment can continue for up to eight 28-day cycles if benefits outweigh risks. Participants will have regular assessments including monitoring for side effects, lab tests, heart checks (ECG and echocardiogram), and physical exams during treatment cycles. After completing treatment, they will be followed by phone every 3 months for up to 2 years to check survival and any further cancer therapies. The main outcomes include the highest tolerated dose, safety, drug levels in the blood, and response to treatment over time.
CONDITIONS
Brief Title
A Study of BTX-A51 in People With Relapsed or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 years or older
- Diagnosis of acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS) with relapsed or refractory disease
- Ineligible for or exhausted standard therapies likely to provide clinical benefit
- Eastern Cooperative Oncology Group performance status 0 to 2
- Life expectancy of at least 6 weeks
- Adequate organ function (Grade 1 serum creatinine; Grade 1 total bilirubin; AST/ALT ≤ 2 times upper limit of normal)
- Females of childbearing potential must not be pregnant and agree to use effective contraception during and for 3 months after treatment
- Males sexually active with females of childbearing potential must agree to use barrier contraception during and for 3 months after treatment
You will not qualify if you...
- Diagnosis of acute promyelocytic leukemia
- White blood cell count greater than 20 x 10^9/L
- Chemotherapy (other than hydroxyurea) within 2 weeks before starting study drug
- Stem cell transplantation within 3 months prior to screening
- Active graft-versus-host disease requiring systemic treatment
- Use of systemic immunosuppressive medications (high-dose steroids, calcineurin inhibitors, or other specified drugs) within specified timeframes before screening
- Life-threatening complications of leukemia such as uncontrolled bleeding or severe infections
- Persistent side effects from prior treatment of Grade 2 or higher
- Active uncontrolled infections (fungal, bacterial, mycobacterial, or viral)
- Significant heart disease
- Conditions limiting oral drug intake or absorption (e.g., dysphagia, short-gut syndrome, gastroparesis)
- Other medical conditions that would interfere with study or pose hazard
- Pregnancy or breastfeeding in females of childbearing potential
AI-Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to eight 28-day cycles (approximately 224 days)
Participants receive BTX-A51 alone or in combination with azacitidine in treatment cycles consisting of 3 weeks of dosing followed by 1 week without study drug, for up to eight 28-day cycles (approximately 224 days). Dosing schedules vary by cohort and phase, with BTX-A51 given orally 3 to 5 days per week and azacitidine administered intravenously or subcutaneously on Days 1-7 of each cycle.
Weekly visits during each 28-day cycle
Duration - Up to 2 years after last treatment dose
Participants are monitored for overall survival and event-free survival for up to 2 years after their last dose of BTX-A51 or until death, whichever occurs first.
Periodic visits for survival assessment
Trial Site Locations
Total: 3 locations
1
City of Hope National Medical Center
Duarte, California, United States, 91010
Actively Recruiting
2
Memorial Sloan-Kettering Cancer Center
New York, New York, United States, 10065
Actively Recruiting
3
The University of Texas MD Anderson Cancer Center
Houston, Texas, United States, 77030
Actively Recruiting
Research Team
Z
Zung Thai, MD
E
Edgar Bautista
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
3
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