A Phase 1 Trial of miRisten in Adult Patients With Relapsed/Refractory AML
This phase I trial tests the safety, side effects, and best dose of miRisten in treating patients with acute myeloid leukemia (AML) that has come back after a period of improvement (relapsed) or that has not responded to previous treatment (refractory). MiRisten may stop the growth of cancer cells by blocking some of the molecules needed for cell growth. Giving miRisten may be safe, tolerable and/or effective in treating patients with relapsed or refractory AML.
• Documented informed consent of the participant and/or legally authorized representative.
• Agreement to allow the use of archival tissue from diagnostic tumor biopsies
⁃ If unavailable, exceptions may be granted with study principal investigator (PI) approval.
• ≥ 18 years.
• Eastern Cooperative Oncology Group (ECOG) ≤ 2.
• Patients with histologically confirmed AML, according to International Consensus Classification (ICC) or World Health Organization (WHO) criteria, with relapsed or refractory (R/R) disease who have failed treatment with, or are ineligible for, available therapies known to be active for treatment of AML.
⁃ Patients with extramedullary disease may be included if they also have concurrent marrow disease.
• Fully recovered from the acute toxic effects (except alopecia) to ≤ grade 1 to prior anti-cancer therapy.
• Life expectancy of ≥ 3 months.
• Total bilirubin ≤ 1.5 x upper limit of normal (ULN) (performed within 28 days prior to day 1 of protocol therapy unless otherwise stated).
• Aspartate aminotransferase (AST) ≤ 3 x ULN (performed within 28 days prior to day 1 of protocol therapy unless otherwise stated).
• Alanine aminotransferase (ALT) ≤ 3 x ULN (performed within 28 days prior to day 1 of protocol therapy unless otherwise stated).
• Creatinine clearance of ≥ 60 mL/min per 24-hour urine test or the Cockcroft-Gault formula (performed within 28 days prior to day 1 of protocol therapy unless otherwise stated).
• International normalized ratio (INR) or prothrombin (PT) ≤ 1.5 x ULN (performed within 28 days prior to day 1 of protocol therapy unless otherwise stated).
• Activated partial thromboplastin time (aPTT) ≤ 1.5 x ULN (performed within 28 days prior to day 1 of protocol therapy unless otherwise stated).
• Left ventricular ejection fraction (LVEF) ≥ 45% (performed within 28 days prior to day 1 of protocol therapy unless otherwise stated).
• Corrected QT interval (QTcF) ≤ 480 ms based on Fridericia's formula
⁃ Note: To be performed within 28 days prior to day 1 of protocol therapy.
• Women of childbearing potential (WOCBP): negative urine or serum pregnancy test (performed within 28 days prior to day 1 of protocol therapy unless otherwise stated).
⁃ If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required.
• Agreement by females and males of childbearing potential to use an effective method of birth control or abstain from heterosexual activity for the course of the study through at least 3 months after the last dose of protocol therapy.
⁃ Childbearing potential defined as not being surgically sterilized (men and women) or have not been free from menses for \> 1 year (women only).