What side effects are associated with this type of intervention?

Common treatments for Acute Myelogenous Leukemia (AML), such as cytarabine and anthracyclines, which inhibit DNA synthesis and cell proliferation, often lead to side effects including myelosuppression (neutropenia, anemia, thrombocytopenia), gastrointestinal symptoms (nausea, vomiting, diarrhea), mucositis, increased risk of infections, fatigue, hair loss, and organ-specific toxicities like cardiotoxicity with anthracyclines.

What prior FDA approvals exist?

The FDA has approved several treatments for Acute Myelogenous Leukemia (AML), including cytarabine and anthracyclines like daunorubicin and idarubicin, which are commonly used in combination chemotherapy regimens. These drugs work by interfering with DNA synthesis and cell proliferation, similar to the mechanism of action of Fosciclopirox. Additionally, newer targeted therapies such as FLT3 inhibitors (e.g., midostaurin) and IDH inhibitors (e.g., ivosidenib) have been approved for specific genetic mutations in AML patients.

What other types of research exist?

Promising novel alternatives to Fosciclopirox for AML patients include: 1) Venetoclax, a BCL-2 inhibitor that promotes apoptosis in cancer cells. 2) Gilteritinib, a FLT3 inhibitor effective in patients with FLT3 mutations. 3) Ivosidenib, an IDH1 inhibitor for patients with IDH1 mutations. 4) Enasidenib, an IDH2 inhibitor for patients with IDH2 mutations. These agents have shown efficacy in clinical trials and offer targeted mechanisms of action for treating AML.

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Other

Fosciclopirox + Cytarabine for Acute Myeloid Leukemia

Phase 1 & 2

Waitlist Available

Research Sponsored by CicloMed LLC

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patient has relapsed AML after complete remission of any duration as evidenced by presence of neoplastic blasts in the bone marrow confirmed by flow cytometry OR has refractory AML, defined as primary refractory to at least 2 cycles of induction therapy

No other therapy exists or patient has received all standard therapies that would be potentially curative or might provide significant benefit

Must not have

Patient has another active malignancy

Patient has acute promyelocytic leukemia (APL) or Ph+ AML

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from informed consent form through follow up visit (30±5 days after last dose of study drug)

Awards & highlights

Summary

This trial tests a new drug, fosciclopirox, alone and with cytarabine, in patients with a type of blood cancer that hasn't responded to other treatments. The goal is to see if these drugs can stop cancer growth or kill the cancer cells.

Who is the study for?

Adults (≥18 years) with relapsed/refractory Acute Myeloid Leukemia who've exhausted standard treatments, have a life expectancy of ≥3 months, and meet specific health criteria including adequate organ function. They must not be pregnant/breastfeeding, have certain other diseases or infections, or be on conflicting medications.

What is being tested?

The trial is testing Fosciclopirox alone and in combination with Cytarabine to evaluate its effectiveness and safety for treating AML. The study will progress through different cohorts based on the observed disease response rate to determine if further investigation is warranted.

What are the potential side effects?

While specific side effects are not listed here, potential risks may include reactions related to liver or kidney function due to the drug's properties and interactions with other medications. Close monitoring for general drug-related side effects such as nausea, fatigue, blood count changes etc., would also be expected.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My AML has returned after treatment or hasn't responded to at least 2 treatment cycles.

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I have tried all standard treatments without success.

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I can take care of myself and am up and about more than half of my waking hours.

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My kidney function is within the required range.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have another active cancer.

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I have been diagnosed with acute promyelocytic leukemia or Ph+ AML.

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I have a serious heart condition.

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I have chronic liver disease or hepatitis B or C.

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I am currently pregnant or breastfeeding.

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I am currently taking warfarin.

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I am currently on iron replacement therapy.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from informed consent form through follow up visit (30±5 days after last dose of study drug)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from informed consent form through follow up visit (30±5 days after last dose of study drug)

This trial's timeline: 3 weeks for screening, Varies for treatment, and from informed consent form through follow up visit (30±5 days after last dose of study drug) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Acute Myeloid Leukemia (AML) response

Frequency and type of treatment-related AEs

Side effects data

From 2021 Phase 2 trial • 50 Patients • NCT03012672

45%

Febrile Neutropenia

25%

Bacteremia

20%

Hypoxia

20%

Rash Maculo-Papular

15%

Cardiac Troponin I Increased

15%

Hyperglycemia

15%

Tumor Lysis Syndrome

10%

Respiratory Failure

10%

Intracranial Hemorrhage

10%

Delirium

10%

Atrial Fibrillation

10%

Edema Limbs, Volume Overload

10%

Blood Bilirubin Increased

10%

Headache

10%

Pulmonary Edema

10%

Soft Tissue Infection

10%

Urinary Tract Infection

10%

COPD Exacerbation

Hypotension

Multi-organ failure

Lung Infection

Sepsis

Right Ventricular Dysfunction

Vascular Access Complication

Generalized Edema

Acute Kidney Injury

Colonic Pseudo-Obstruction

Nausea

Alkalosis

Anorexia

Dyspnea

Bronchopulmonary Hemorrhage

Hypertension

Hypokalemia

Hyponatremia

Optic Nerve Disorder

Paroxysmal Atrial Tachycardia

100%

80%

60%

40%

20%

Study treatment Arm

ARM I (HIGHER-DOSE):

ARM II (LOWER-DOSE):

Trial Design

2Treatment groups

Experimental Treatment

Group I: Cohort 2 - Fosciclopirox + CytarabineExperimental Treatment1 Intervention

To be implemented if a disease response is not seen in Cohort 1a. Cohort 2a will have an initial 14 study participants treated with fosciclopirox and cytarabine. If a disease response is seen, an additional 14 study participants will be enrolled (Cohort 2b).

Group II: Cohort 1 - Fosciclopirox onlyExperimental Treatment1 Intervention

An initial 14 study participants will be enrolled in Cohort 1a and will be treated with fosciclopirox. If there is a disease response, an additional 14 study participants will be enrolled into Cohort 1 (Cohort 1b).

0 / 11Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Acute Myelogenous Leukemia (AML) often target the rapid proliferation and survival of leukemic cells. Chemotherapy agents like cytarabine and anthracyclines work by interfering with DNA synthesis and inducing cell death. Targeted therapies, such as FLT3 inhibitors, block specific signaling pathways crucial for leukemic cell growth. The relevance of these mechanisms lies in their ability to reduce the leukemic cell burden and achieve remission. Similar to Fosciclopirox, which inhibits ribonucleotide reductase to decrease DNA synthesis and cell proliferation, these treatments aim to disrupt the cellular processes essential for the survival and proliferation of AML cells, thereby improving patient outcomes.

Identification of Deregulated Signaling Pathways in Jurkat Cells in Response to a Novel Acylspermidine Analogue-N4-Erucoyl Spermidine.Molecular targeting in acute myeloid leukemia.