NCT07025564

MiRisten for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia

A Phase 1 Trial of miRisten in Adult Patients With Relapsed/Refractory AML

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: City of Hope Medical Center · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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. Estimate overall survival (OS), progression-free survival (PFS) and duration of response (DOR) rate at 6 months. III. Describe the pharmacokinetics (PK) of miRisten. EXPLORATORY STUDY OBJECTIVES: I. Evaluate pharmacodynamics (PD) of miRisten therapy to identify biomarkers of clinical response and resistance. II. Estimate miR-126 level in the bone marrow and blood pre-, post-therapy. III. Evaluate the impact of miRisten on miR-126 and its targets (e.g., SPRED1, CDK3, PIK3R2) in bone marrow. IV. Estimate leukemia stem cell burden in bone marrow pre-, post-therapy. V. Monitor levels of cytokines and complement over time. VI. Dissect the mechanisms of miR-126-dependent leukemia stem cell (LSC) survival and self-renewal. VII. Estimate the anti-leukemogenic force, therapeutic work induced by miRisten using the microRNA (miRNA)-based state transition model. OUTLINE: This is a dose-escalation study. Patients receive miRisten IV over 30 minutes twice a day (BID) on days 1-5, 8-12 and 15-19 in the absence of disease progression or unacceptable toxicity. Patients also undergo echocardiography (ECHO) during screening and bone marrow and blood sample collection throughout the trial. After completion of study treatment, patients are followed up at 30 days and then at 3 and 6 months.

Conditions

• Recurrent Acute Myeloid Leukemia
• Refractory Acute Myeloid Leukemia

Interventions

Type	Name	Description
PROCEDURE	Biospecimen Collection	Undergo bone marrow and blood sample collection
PROCEDURE	Echocardiography Test	Undergo ECHO
DRUG	miR-126 Inhibitor miRisten	Given IV

Timeline

• Start date: 2025-10-24
• Primary completion: 2027-04-08
• Completion: 2027-04-08
• First posted: 2025-06-17
• Last updated: 2026-04-15

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study