miRisten (miR-126 Inhibitor)

PRIMARY OBJECTIVES: I. Evaluate the safety and tolerability of miR-126 inhibitor miRisten (miRisten) given intravenously (i.v.) twice daily. II. Determine the maximum tolerated dose (MTD) and recommended phase 2 dose (RP2D). SECONDARY OBJECTIVES: I.

Evaluate the anti-leukemic activity, as assessed by complete remission (complete response [CR]+complete response with Incomplete hematopoietic recovery [CRi]+CR with partial hematologic recovery [CRh]), overall response (overall response rate [ORR]: CR+CRi+CRh+partial response [PR]) and minimal residual disease negative (MRD-) rate and duration over the study period. II.

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.

Live record on ClinicalTrials.gov →

• 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.
• 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.

Inclusion criteria as written on the registry, trimmed. The full list, with exclusions, is on the live record.