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MiRisten for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia | Clinical Trials | Clareo Health

RECRUITINGPHASE1

MiRisten for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia

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

NCT07025564•City of Hope Medical Center

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.

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•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).
+12 more criteria

Exclusion Criteria

•Allogeneic hematopoietic stem cell transplant within 3 months prior to day 1 of protocol therapy. Patients must be off calcineurin inhibitors for at least 2 weeks prior to day 1 of protocol therapy.
•Chemotherapy, radiation therapy, biological therapy, immunotherapy within 14 days prior to day 1 of protocol therapy, with the exception of hydroxyurea.
•Strong and moderate CYP3A4 inducers and strong CYP3A inhibitors (with the exception of azole antifungals) within 7 days prior to day 1 of protocol therapy.
•Foods/supplements that are strong inhibitors or strong or moderate inducers of CYP3A (such as St. John's wort) within 3 days prior to initiation of and during study treatment.
•Systemic steroid therapy \> 10 mg/day (≤ 10mg/day prednisone equivalent ok) or any other form of immunosuppressive medication within 14 days. Inhaled or topical steroids, and adrenal replacement steroid doses ≤10 mg daily prednisone equivalent, are permitted. Steroids given for study drug infusion reaction prophylaxis or infusion reactions should not count towards this maximum.
•Must not have received or planning to receive live vaccine while being on study or 28 days before and after completion of treatment.
•Acute promyelocytic leukemia (APL).
•History of allergic or infusion reactions attributed to compounds of similar chemical or biologic composition to study agent.
•Inability to tolerate dexamethasone at the doses prescribed in this protocol.
•Unstable cardiac disease as defined by the following:
+8 more criteria

Locations (1)

City of Hope Medical Center

Duarte, California, United States

Key Dates

Start Date

February 28, 2026

Primary Completion Date

April 8, 2027

Completion Date

April 8, 2027

Last Updated

October 10, 2025

Enrollment

ESTIMATED participants

Conditions

Recurrent Acute Myeloid LeukemiaRefractory Acute Myeloid Leukemia

Interventions

Biospecimen Collection

PROCEDURE

Echocardiography Test

PROCEDURE

miR-126 Inhibitor miRisten

DRUG

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A Phase 1 Study of AOH1996 in Patients With Relapsed/Refractory Acute Myeloid Leukemia

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: October 10, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

MiRisten for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia

Inclusion Criteria

Exclusion Criteria

Testing the Anti-cancer Drug, Cirtuvivint, and Its Combination With ASTX727 to Improve Outcomes in Patients With Acute Myeloid Leukemia and Myelodysplastic Syndromes

A Phase 1 Study of AOH1996 in Patients With Relapsed/Refractory Acute Myeloid Leukemia

SNDX-5613 and Gilteritinib for the Treatment of Relapsed or Refractory FLT3-Mutated Acute Myeloid Leukemia and Concurrent MLL-Rearrangement or NPM1 Mutation

Study of Revumenib, Azacitidine, and Venetoclax in Pediatric and Young Adult Patients With Refractory or Relapsed Acute Myeloid Leukemia

BCL2i CLAG-M in R/R Acute Myeloid Leukemia

CLN-049 in Patients With Relapsed/Refractory Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)