MiRisten for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia

NCT07025564 | Suspended Phase 1 DMC FDA Drug

• 3 other identifiers: 24764NCI-2025-03912P30CA033572
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Recurrent Acute Myeloid Leukemia; Refractory Acute Myeloid Leukemia

Outcome Measures

Primary Outcomes (1)

• Incidence of adverse events (AEs)

  Toxicity data collection will include incidence and nature of adverse events (AEs) (including grade 3 and 4 AEs, treatment-related AEs, serious adverse events, dose limiting and moderate toxicities, AEs requiring discontinuation of miRisten, and changes in laboratory values.

  Up to 30 days after completion of study treatment

Secondary Outcomes (3)

• Disease response

  Up to 6 months after completion of study treatment

• Duration of response

  From the date of first documented response to documented disease relapse, progression or death whichever occurs first, assessed p to 6 months after completion of study treatment

• Progression-free survival

  From date of first dose of study drug to first documented evidence of disease progression or death from any cause, whichever occurs first, assessed up to 6 months after completion of study treatment

Study Arms (1)

Treatment (miRisten)

EXPERIMENTAL

Patients receive miRisten IV over 30 minutes BID on days 1-5, 8-12 and 15-19 in the absence of disease progression or unacceptable toxicity. Patients also undergo ECHO during screening and bone marrow and blood sample collection throughout the trial.

Procedure: Biospecimen Collection; Procedure: Echocardiography Test; Drug: miR-126 Inhibitor miRisten

Interventions

Biospecimen Collection PROCEDURE

Undergo bone marrow and blood sample collection

Also known as: Biological Sample Collection, Biospecimen Collected, Specimen Collection

Treatment (miRisten)

miR-126 Inhibitor miRisten DRUG

Given IV

Also known as: CpG-linked Anti-Mir-126 Oligonucleotide, miRisten; Oligodeoxynucleotide Anti-miR-126 Inhibitor miRisten; MicroRNA-126 Inhibitor miRisten; CpG(D19)-Anti-miR-126; ODN-based CpG-Anti-miRNA-126;

Treatment (miRisten)

Echocardiography Test PROCEDURE

Undergo ECHO

Also known as: EC, Echocardiography

Treatment (miRisten)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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).

You may not qualify if:

• 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:
• Unstable angina
• Uncontrolled atrial fibrillation or hypertension
• Acute coronary syndrome and/or revascularization (e.g., coronary artery bypass graft, stent) within 6 months of first dose of study drug.
• Clinically significant uncontrolled illness.
• Uncontrolled active infection.

Sponsors & Collaborators

• City of Hope Medical Center: lead
• National Cancer Institute (NCI): collaborator

Study Sites (1)

City of Hope Medical Center

Duarte, California, 91010, United States

Location

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Interventions

Specimen Handling

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Investigative Techniques

Study Officials

• Amanda Blackmon

  City of Hope Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 6, 2025
• First Posted: June 17, 2025
• Study Start: October 24, 2025
• Primary Completion (Estimated): March 1, 2028
• Study Completion (Estimated): August 1, 2028
• Last Updated: June 11, 2026

Record last verified: 2026-06

Locations

US (1)