What side effects are associated with this type of intervention?

Common treatments for Acute Myeloid Leukemia (AML) such as hypomethylating agents (azacitidine and decitabine) and combinations like pomalidomide with daunorubicin and cytarabine liposome are associated with several side effects. The most frequent adverse events include neutropenia, anemia, thrombocytopenia, and fatigue. Neutropenia, a significant reduction in neutrophils, increases infection risk. Anemia and thrombocytopenia can lead to fatigue, weakness, and increased bleeding risk. Fatigue is a common side effect impacting the patient's quality of life. These treatments aim to balance efficacy with manageable toxicity.

What prior FDA approvals exist?

The FDA has approved several drugs for the treatment of Acute Myeloid Leukemia (AML) that share similar mechanisms to pomalidomide, such as anti-angiogenic properties, immune stimulation, and cytotoxicity. Azacitidine, an anti-cancer drug with hypomethylating properties, has been approved for post-remission maintenance therapy of AML. Decitabine, another hypomethylating agent, is approved by the European Medicines Agency (EMA) for AML treatment but not by the FDA. These drugs work by modifying the DNA of cancer cells to inhibit their growth and stimulate the immune system to target the cancer cells. While pomalidomide is being studied for its potential benefits in AML, these existing treatments provide a foundation for understanding the therapeutic landscape of AML.

What other types of research exist?

Promising novel alternatives to Pomalidomide for Acute Myeloid Leukemia patients include hypomethylating agents (azacitidine and decitabine), and combination therapies such as low-dose cytarabine (LoDAC) with venetoclax or glasdegib. These treatments have demonstrated efficacy in clinical trials and offer potential benefits in terms of anti-angiogenic properties, immune stimulation, and cytotoxicity.

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Anti-tumor antibiotic, Anti-metabolites

Pomalidomide + Chemotherapy for Acute Myeloid Leukemia

Phase 2

Recruiting

Led By Joshua F Zeidner

Research Sponsored by National Cancer Institute (NCI)

Eligibility Criteria Checklist

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

Must have

Must meet criteria for t-AML or AML with MRC as defined by the 5th Edition of the World Health Organization (WHO) Classification of Myeloid Neoplasms or the International Consensus Classification (ICC) of Myeloid Neoplasms. Patients must meet one of the following criteria: Therapy-related AML (AML derived from prior chemotherapy or radiation therapy) AML originating from prior hematologic malignancy (MDS, CMML, or MPN)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

Study Summary

This trial is testing the addition of pomalidomide to usual chemotherapy in treating patients with newly diagnosed acute leukemia with myelodysplastic syndrome-related changes.

Who is the study for?

Adults aged 18-75 with newly diagnosed acute myeloid leukemia (AML) and specific genetic mutations or changes related to myelodysplastic syndrome, who have good organ function and performance status. They must not have had previous AML treatment except hydroxyurea or leukapheresis, no Wilson's Disease, uncontrolled illnesses, prior allogeneic stem cell transplant, or certain cumulative doses of daunorubicin. Women of childbearing potential must test negative for pregnancy and use two forms of birth control.Check my eligibility

What is being tested?

The trial is testing the addition of Pomalidomide—an immunomodulatory drug—to standard chemotherapy (Daunorubicin and Cytarabine Liposome) in patients with AML that has characteristics similar to myelodysplastic syndrome. The study aims to see if this combination improves outcomes compared to the usual treatment alone.See study design

What are the potential side effects?

Pomalidomide may cause blood vessel growth inhibition, immune system stimulation leading to cancer cell death but also risks like blood clots and fetal harm if taken during pregnancy. Standard chemo can cause side effects such as hair loss, nausea, vomiting, fatigue, infection risk increase due to low blood cell counts.

Eligibility Criteria

Inclusion Criteria

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

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My leukemia is classified as therapy-related or stems from a previous blood disorder.

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My leukemia diagnosis was confirmed with a specific test showing more than 20% immature blood cells.

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My cancer has a specific genetic change known as del(11q).

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My cancer has a specific genetic feature (-13/del(13q)).

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My cancer has a specific genetic change in chromosome 12.

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My cancer has a specific genetic change known as idicX(q13).

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My hepatitis B virus load is undetectable with treatment.

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My cancer has a STAG2 mutation.

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My leukemia is classified as t-AML or AML with MRC by WHO or ICC standards.

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My AML was caused by previous cancer treatments.

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My AML is linked to specific genetic changes.

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My AML has specific mutations and I've only had hydroxyurea or leukapheresis.

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My kidneys are functioning well enough to clear waste.

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My AML developed from a previous blood disorder.

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My cancer has a specific genetic abnormality.

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My AML is linked to certain genetic changes.

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My cancer has a specific genetic change (-17/add(17p) or del(17p)).

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My cancer has a specific genetic change known as del(20q).

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I had hepatitis C but am cured, or I'm being treated with no detectable virus.

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I am between 18 and 75 years old and ready for intensive chemotherapy.

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My cancer has a complex genetic profile with multiple chromosomal abnormalities.

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My AML has mutations related to myelodysplasia.

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I have AML and have only been treated with hydroxyurea or leukapheresis.

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My condition involves a -7/del(7q) chromosome abnormality.

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My cancer has a specific genetic change involving chromosome 5.

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I can take care of myself but may not be able to do heavy physical work.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Rate of complete response (CR)/complete response with incomplete hematologic recovery (CRi)

Secondary outcome measures

CR with full hematologic recovery (absolute neutrophil count > 1 x 10^9/L and platelets > 100 x 10^9/L)

Complete response (CR) without minimal residual disease (MRD)

Disease-free survival

+4 more

Side effects data

From 2015 Phase 2 trial • 36 Patients • NCT02011113

72%

NEUTROPENIA

47%

ANAEMIA

44%

THROMBOCYTOPENIA

25%

NASOPHARYNGITIS

25%

PYREXIA

25%

CONSTIPATION

22%

LYMPHOPENIA

19%

DIARRHOEA

19%

OEDEMA PERIPHERAL

19%

NAUSEA

19%

RASH

17%

LEUKOPENIA

17%

INSOMNIA

17%

MALAISE

14%

PNEUMONIA

14%

DYSGEUSIA

11%

EPISTAXIS

11%

FATIGUE

11%

DECREASED APPETITE

11%

HYPERURICAEMIA

11%

HYPOALBUMINAEMIA

11%

UPPER RESPIRATORY TRACT INFECTION

11%

HYPOKALAEMIA

RASH MACULO-PAPULAR

Pharyngitis

HYPERGLYCAEMIA

HYPOPHOSPHATAEMIA

HYPOXIA

ANXIETY

MYALGIA

HEPATIC FUNCTION ABNORMAL

HERPES ZOSTER

WEIGHT INCREASED

GASTROENTERITIS

HEADACHE

DECUBITUS ULCER

NEUROPATHY PERIPHERAL

CYSTITIS

DIABETES MELLITUS

PERIPHERAL SENSORY NEUROPATHY

CANCER PAIN

INCREASED APPETITE

RESTLESSNESS

HYPOTENSION

HYPOGAMMAGLOBULINAEMIA

ABDOMINAL PAIN UPPER

HAEMORRHOIDS

ASTHMA

VOMITING

BRONCHITIS

HYPERCALCAEMIA

HYPOCALCAEMIA

HYPONATRAEMIA

HYPERSOMNIA

DYSPHONIA

PLEURAL EFFUSION

HICCUPS

ALANINE AMINOTRANSFERASE INCREASED

ASPARTATE AMINOTRANSFERASE INCREASED

BLOOD ALKALINE PHOSPHATASE INCREASED

MUSCLE SPASMS

WEIGHT DECREASED

TREMOR

HYPOTHYROIDISM

SOMNOLENCE

PROCTALGIA

DYSPNOEA

SEPSIS

PNEUMOCYSTIS JIROVECII PNEUMONIA

PNEUMONIA PNEUMOCOCCAL

MULTI-ORGAN FAILURE

INTERSTITIAL LUNG DISEASE

C-REACTIVE PROTEIN INCREASED

CARDIAC FAILURE

CHRONIC OBSTRUCTIVE PULMONARY DISEASE

URINARY RETENTION

MENINGITIS

SPINAL COMPRESSION FRACTURE

BLOOD FIBRINOGEN DECREASED

BACK PAIN

SHOCK HAEMORRHAGIC

100%

80%

60%

40%

20%

Study treatment Arm

Pomalidomide Plus Dexamethasone

Trial Design

2Treatment groups

Experimental Treatment

Active Control

Group I: Arm A (daunorubicin and cytarabine liposome, pomalidomide)Experimental Treatment4 Interventions

INDUCTION: Patients receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1, 3, and 5 and then pomalidomide PO QD beginning between days 21-30 for 14 days in the absence of disease progression or unacceptable toxicity. Patients who do not respond, may receive a second cycle of liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3 in the absence of disease progression or unacceptable toxicity. CONSOLIDATION: Patients who achieve CR/CRi receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3. Treatment repeats every 21 days for 2 cycles in the absence of disease progression and unacceptable toxicity. Patients also undergo bone marrow aspirate and biopsy and collection of blood samples throughout all phases of the trial.

Group II: Arm B (daunorubicin and cytarabine liposome)Active Control3 Interventions

INDUCTION: Patients receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1, 3, and 5 in the absence of disease progression or unacceptable toxicity. Patients who do not respond, may receive a second cycle of liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3 in the absence of disease progression or unacceptable toxicity. CONSOLIDATION: Patients who achieve CR/CRi receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3. Treatment repeats every 21 days for 2 cycles in the absence of disease progression and unacceptable toxicity. Patients also undergo bone marrow aspirate and biopsy and collection of blood samples throughout all phases of the trial.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Liposome-encapsulated Daunorubicin-Cytarabine

2016

Completed Phase 2

~100

Biospecimen Collection

2004

Completed Phase 2

~1720

Bone Marrow Aspiration and Biopsy

2016

Completed Phase 1

~40

Pomalidomide

2011

Completed Phase 2

~1020

0 / 27Eligibility 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 Myeloid Leukemia (AML) include chemotherapy agents like daunorubicin and cytarabine, which work by killing rapidly dividing cancer cells (cytotoxicity). Hypomethylating agents such as azacitidine and decitabine inhibit DNA methylation, leading to the reactivation of tumor suppressor genes. Targeted therapies like IDH inhibitors block specific mutations driving the cancer. Treatments similar to Pomalidomide, which include anti-angiogenic properties (inhibiting blood vessel growth to the tumor), immune stimulation (enhancing the body's immune response against cancer cells), and cytotoxic effects, are crucial as they offer a multi-faceted approach to combat AML. These mechanisms are important because they not only directly target cancer cells but also modify the tumor environment and enhance the body's natural defenses, potentially leading to better treatment outcomes.

Role of epigenetic in leukemia: From mechanism to therapy.Progress in the problem of relapsed or refractory acute myeloid leukemia.Molecular targeting in acute myeloid leukemia.