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.

Registro actualizado en ClinicalTrials.gov →

• Consentimiento informado documentado del participante y/o de su representante legal autorizado.
• Acuerdo para permitir el uso de tejido de archivo de las biopsias tumorales diagnósticas
• 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.

Criterios de inclusión tal como figuran en el registro, recortados. La lista completa, con las exclusiones, está en el registro actualizado.