Evaluating BTX-A51 for patients with relapsed or refractory acute myeloid leukemia or high-risk myelodysplastic syndrome
A First-In-Human, Open-Label, Escalating Multiple-Dose Study to Evaluate the Safety, Toxicity, and Pharmacokinetics of BTX-A51 Alone and in Combination With Azacitidine in Patients With Relapsed or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome
This study is testing a new drug called BTX-A51 to see if it can help people with relapsed or hard-to-treat acute myeloid leukemia or high-risk myelodysplastic syndrome feel better and improve their treatment options.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 80 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Edgewood Oncology Inc. Industry-sponsored |
| Drugs / interventions | ruxolitinib, chemotherapy |
| Locations | 3 sites (Duarte, California and 2 other locations) |
| Trial ID | NCT04243785 on ClinicalTrials.gov |
What this trial studies
This open-label, dose escalation study aims to assess the safety, toxicity, and pharmacokinetics of BTX-A51 capsules in individuals with relapsed or refractory acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS). The study is divided into three parts: the first part focuses on determining the maximum tolerated dose (MTD) through monotherapy dose escalation, followed by a cohort expansion to gather more data on safety and efficacy. The final part will explore the combination of BTX-A51 with Azacitidine to further evaluate its effects. Up to 80 participants will be enrolled throughout the study phases.
Who should consider this trial
Good fit: Ideal candidates include adults aged 18 and older with relapsed or refractory AML or high-risk MDS who have exhausted standard treatment options.
Not a fit: Patients with early-stage disease or those who are not refractory to standard therapies may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide a new treatment option for patients with difficult-to-treat forms of leukemia and myelodysplastic syndrome.
How similar studies have performed: While this approach is novel, similar studies targeting AML and MDS have shown promise in exploring new therapeutic options.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Demonstration of understanding and voluntarily signing of an informed consent form * Age ≥ 18 years * 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 * Eastern Cooperative Oncology Group performance status ≤ 2 and life expectancy of ≥ 6 weeks * Adequate organ function (Grade 1 serum creatinine; Grade 1 total bilirubin; aspartate aminotransferase and/or alanine transaminase ≤ 2 × ULN) * Females of childbearing age must not be pregnant at time of Screening/beginning of treatment and agree to either abstain from sexual intercourse or use highly effective methods of contraception (for up to 3 months after last dose of study drug) * Males sexually active with a woman of childbearing age must agree to use barrier method of birth control during and after the study (up to 3 months after last dose of study drug) Exclusion Criteria: * Diagnosis of acute promyelocytic leukemia * White blood cell count \> 20 x 10\^9/L * Receipt of cancer chemotherapy (other than hydroxyurea) within 2 weeks prior to the start of study drug * In participants who have undergone autologous or allogeneic stem cell transplantation: transplantation within the 3 months prior to Screening; active graft-versus-host disease requiring anything other than topical corticosteroids and budesonide; treatment with systemic immunosuppressive medications including high-dose steroids (≥ 20 mg prednisolone or equivalent per day), or calcineurin inhibitors (e.g., cyclosporine, tacrolimus) for at least 1 week prior to Screening, and sirolimus, mycophenylate mofetil, azathioprine, or ruxolitinib for at least 2 weeks prior to Screening * Immediate life-threatening severe complications of leukemia such as uncontrolled bleeding, pneumonia with hypoxia or shock, and/or disseminated intravascular coagulation * Persistent toxicities from prior treatment of Grade 2 or higher * Active uncontrolled systemic fungal, bacterial, mycobacterial, or viral infection * Clinically significant cardiac disease * Known dysphagia, short-gut syndrome, gastroparesis, or other conditions that limit the ingestion or gastrointestinal absorption of drugs administered orally * Any other concurrent medical condition or disease that is likely to interfere with study procedures or results, or that, in the opinion of the Investigator, would constitute a hazard for participating in this study * If female, pregnant or breastfeeding
Where this trial is running
Duarte, California and 2 other locations
- City of Hope National Medical Center — Duarte, California, United States (Recruiting)
- Memorial Sloan-Kettering Cancer Center — New York, New York, United States (Recruiting)
- The University of Texas MD Anderson Cancer Center — Houston, Texas, United States (Recruiting)
Study contacts
- Study coordinator: Zung Thai, MD
- Email: zung@edgewoodonc.com
- Phone: 415-225-9338
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.