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Compensation: Varies

Number of Visits: 31

Duration: 3 weeks

12 Participants Needed

miRisten for Acute Myeloid Leukemia

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: City of Hope Medical Center

Must not be taking: CYP3A4 inducers, CYP3A inhibitors

Disqualifiers: Acute promyelocytic leukemia, Unstable cardiac disease, Uncontrolled infection, others

No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new drug, miRisten, for individuals with acute myeloid leukemia (AML) that has relapsed or not improved with previous treatments. The main goal is to determine miRisten's safety and identify the optimal dose. MiRisten aims to inhibit the growth of cancer cells. Suitable candidates for this trial have AML that hasn't responded to other treatments or has relapsed, and they do not have other serious health issues. As a Phase 1 trial, participants will be among the first to receive this new treatment, aiding researchers in understanding its effects in people.

Do I need to stop my current medications to join the trial?

The trial requires stopping certain medications before starting, such as strong and moderate CYP3A4 inducers and strong CYP3A inhibitors, except for azole antifungals, within 7 days before the trial. Also, systemic steroid therapy over 10 mg/day must be stopped 14 days prior, except for specific cases. Check with the trial team for details on your specific medications.

Is there any evidence suggesting that miRisten is likely to be safe for humans?

Research has shown that miRisten might be a safe option for treating acute myeloid leukemia (AML). In animal studies, researchers administered miRisten through an IV, and it appeared effective without causing major issues. It also worked well with other drugs already approved for AML, suggesting miRisten could be a promising treatment.

However, since the current trial is in its early phase, it primarily focuses on understanding the drug's safety and determining the right dose for humans. While there is hope for the drug's safety, it is still under testing to confirm this. Prospective participants should know that the trial aims to assess how people handle the treatment and to identify any side effects.¹ ² ³ ⁴ ⁵

Why do researchers think this study treatment might be promising?

Researchers are excited about miRisten for acute myeloid leukemia (AML) because it offers a fresh approach compared to the standard chemotherapy options. Unlike traditional treatments that target rapidly dividing cells, miRisten works by using microRNA to specifically target and regulate cancer cell growth at the genetic level. This precision could mean fewer side effects and a more effective attack on leukemia cells. Plus, miRisten’s unique delivery method, administered intravenously over short, repeated cycles, aims to maximize its impact while minimizing harm to healthy cells.

What evidence suggests that miRisten might be an effective treatment for acute myeloid leukemia?

Research has shown that miRisten, the investigational treatment in this trial, might help treat acute myeloid leukemia (AML), particularly when the disease recurs or resists treatment. Studies have found that miRisten targets and reduces a molecule called miR-126, which aids cancer cell survival. In lab tests with mice, miRisten slowed the growth and activity of leukemia cells and reduced the overall number of cancer cells. These promising early results suggest miRisten could be a helpful treatment option for AML.¹ ⁶ ⁷ ⁸ ⁹

Who Is on the Research Team?

Amanda Blackmon

Principal Investigator

City of Hope Medical Center

Are You a Good Fit for This Trial?

This trial is for individuals with Acute Myeloid Leukemia (AML) that has either returned after getting better or hasn't improved with past treatments. Specific eligibility details are not provided, but typically include health status and prior therapy specifics.

Inclusion Criteria

I am 18 years old or older.

Women of childbearing potential (WOCBP) must have negative pregnancy test and agree to use effective birth control or abstain from heterosexual activity

Documented informed consent of the participant and/or legally authorized representative

See 7 more

Exclusion Criteria

I cannot tolerate the standard doses of dexamethasone.

I have recently used specific medications or therapies.

I might not be able to follow the study's required procedures.

See 4 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Treatment

Participants receive miRisten IV over 30 minutes twice a day on days 1-5, 8-12, and 15-19

3 weeks

Multiple visits for IV administration

Follow-up

Participants are monitored for safety and effectiveness after treatment

6 months

Follow-up visits at 30 days, 3 months, and 6 months

What Are the Treatments Tested in This Trial?

Interventions

miRisten

Trial Overview The trial is evaluating the safety, side effects, and optimal dosage of a new treatment called miRisten for AML patients. It includes biospecimen collection and echocardiography tests to monitor heart function during the study.

How Is the Trial Designed?

1Treatment groups

Experimental Treatment

Group I: Treatment (miRisten)Experimental Treatment3 Interventions

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.

Find a Clinic Near You

Who Is Running the Clinical Trial?

City of Hope Medical Center

Lead Sponsor

Trials

614

Recruited

1,924,000+

National Cancer Institute (NCI)

Collaborator

Trials

14,080

Recruited

41,180,000+

Citations

1.clinicaltrials.govclinicaltrials.gov/study/NCT07025564

MiRisten for the Treatment of Relapsed or Refractory Acute ...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 ...

2.nature.comnature.com/articles/s41467-021-26420-7

Targeting miR-126 in inv(16) acute myeloid leukemia ...Therapeutic depletion of miR-126 with an anti-miR-126 (miRisten) inhibits AML cell survival, reduces leukemia burden and leukemia stem cell (LSC) activity.

3.cityofhope.orgcityofhope.org/research/hematologic-malignancies-research-institute/gehr-family-center-for-leukemia-research/research-highlights

Leukemia Research HighlightsPreclinical research shows that miRisten is highly effective against relapsed or treatment-resistant AML This work is published (Zhang et al. Nature ...

4.researchgate.netresearchgate.net/figure/Administration-of-miRisten-effectively-inhibits-AML-propagation-and-leukemia-initiating_fig7_355481038

Administration of miRisten effectively inhibits AML ...Administration of miRisten effectively inhibits AML propagation and leukemia-initiating activity in inv(16) AML PDX model a Schematic of experimental design ...

5.pmc.ncbi.nlm.nih.govpmc.ncbi.nlm.nih.gov/articles/PMC8516361/

Targeting miR‐126 disrupts maintenance of ...In vivo miRisten administration partially reversed pancytopenia in NHD13 mice and blocked the leukemic transformation (combination group vs DAC ...

6.ashpublications.orgashpublications.org/blood/article/142/Supplement%201/5754/505226/Pharmacology-of-a-CpG-Linked-Anti-Mir-126

Pharmacology of a CpG-Linked Anti-Mir-126 Oligonucleotide ...These data are consistent with the safety of miRisten given IV with a dose ramping regimen combined with solumedrol. The greater AUC in NHP may ...

7.sciencedirect.comsciencedirect.com/science/article/abs/pii/S0006497123123548

Pharmacology of a CpG-Linked Anti-Mir-126 ...miRisten eradicates LSCs in mouse models of AML and synergizes with FDA-approved AML drugs (PMID: 29505034, 34372909).

8.pmc.ncbi.nlm.nih.govpmc.ncbi.nlm.nih.gov/articles/PMC8536759/

9.withpower.comwithpower.com/trial/phase-1-leukemia-myeloid-acute-1-2026-24d1c

miRisten for Acute Myeloid LeukemiaThis 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 ...

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