Pharmacological Study for Therapy-Related Acute Myeloid Leukemia

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
City of Hope Medical Center, Duarte, CA
Therapy-Related Acute Myeloid Leukemia+8 More
Pharmacological Study - Other
Eligibility
18+
All Sexes
What conditions do you have?
Select

Study Summary

This study is evaluating whether a drug called pevonedistat can be safely given with a drug called decitabine to treat people with acute myeloid leukemia.

See full description

Eligible Conditions

  • Therapy-Related Acute Myeloid Leukemia
  • Acute Myeloid Leukemia (AML)
  • acute, recurrent Myeloid Leukemia
  • Refractory Acute Myelogenous Leukemia (AML)
  • Secondary Acute Myeloid Leukemia (Secondary AML, sAML)
  • Untreated Adult Acute Myeloid Leukemia

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Therapy-Related Acute Myeloid Leukemia

Study Objectives

This trial is evaluating whether Pharmacological Study will improve 3 primary outcomes, 5 secondary outcomes, and 7 other outcomes in patients with Therapy-Related Acute Myeloid Leukemia. Measurement will happen over the course of Up to 28 days.

Year 2
Overall survival (OS)
Year 2
Event-free survival (EFS)
Year 2
Duration of response
Up to 2 years
Complete remission (CR) rate assessed by International Working Group criteria
Incidence of adverse effect assessed by National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 4.03
Leukemia stem cell (LSC) activity (LTC-IC frequency and activity in vitro and serial transplant experiments of LSC-enriched cell fractions)
NF-kappaB activity
NF-kappaB enrichment on miR-155 promoter
Nuclear NF-kappaB expression
Overall response rate (ORR) (CR + incomplete CR [CRi]) assessed by International Working Group criteria
miR-155 expression
miR-155 promoter methylation
miR-155 target gene (SHIP1 and PU.1) expression
Up to 28 days
Dose limiting toxicities (DLT) defined as any toxicities that are at least possibly related to pevonedistat that occur during cycle 1 assessed by NCI CTCAE version 4.03
Maximum tolerated dose (MTD) based on assessment of DLT

Trial Safety

Safety Progress

1 of 3

Other trials for Therapy-Related Acute Myeloid Leukemia

Trial Design

1 Treatment Group

Treatment (pevonedistat, decitabine)
1 of 1
Experimental Treatment

This trial requires 30 total participants across 1 different treatment group

This trial involves a single treatment. Pharmacological Study is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 1 and are in the first stage of evaluation with people.

Treatment (pevonedistat, decitabine)Patients receive pevonedistat IV over 1 hour on days 1, 3, and 5 and decitabine IV over 1 hour on days 1-5 and 8-12. Treatment repeats every 28 days for up to 24 courses in the absence of disease progression or unexpected toxicity.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Decitabine
FDA approved
Pevonedistat
Not yet FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: up to 2 years
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly up to 2 years for reporting.

Closest Location

City of Hope Medical Center - Duarte, CA

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. You must have received newly diagnosed for Therapy-Related Acute Myeloid Leukemia or one of the other 8 conditions listed above. There are 10 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
may be candidates for reduced intensity conditioning allogeneic stem cell transplantation (RIC-allo) Some younger patients with AML that has come back after treatment, or is resistant to treatment, may be eligible for a reduced intensity conditioning allogeneic stem cell transplant. show original
in this clinical trial Patients who have cancer that has spread outside of their bone marrow may be eligible to participate in this clinical trial. show original
, and a Karnofsky performance score of 50 or more are candidates for a palliative care consultation Patients who are coping with a co-morbid medical illness, have a life expectancy greater than 6 months, and have a Karnofsky performance score of 50 or more may be good candidates for a palliative care consultation. show original
Women of childbearing potential and men must agree to use contraception (hormonal or barrier method of birth control or abstinence) prior to study entry and for four months following the duration of study participation show original
The person should agree to only use abstinence as a form of contraception when it lines up with their usual lifestyle show original
People over the age of 60 who have relapsed or refractory disease are not likely to respond well to treatment. show original
are being offered an investigational regimen of fludarabine, cyclophosphamide, and topotecan (FCT) as front-line therapy show original
] The Eastern Cooperative Oncology Group (ECOG) defines performance status as < 2 as indicating a patient who is severely limited in their activities. show original
People over 60 who have just been diagnosed and have not received treatment yet, who don't want to undergo chemotherapy or who can't undergo it, are not good candidates for conventional induction chemotherapy with cytarabine and anthracyclines. show original
, they must have a negative serum beta-human chorionic gonadotropin (β-HCG) pregnancy test at the screening visit Women who are postmenopausal for at least 1 year before the screening visit, OR who are surgically sterile, OR who are of childbearing potential and have a negative serum β-human chorionic gonadotropin (β-HCG) pregnancy test at the screening visit, are eligible to participate in this study. show original

Patient Q&A Section

What are common treatments for leukemia?

"Common treatments for leukemia include chemotherapy and targeted therapy, and chemotherapy has been shown to eradicate myeloid leukemia. Radiation therapy and targeted therapy are also effective, but only for limited-stage Hodgkin's disease." - Anonymous Online Contributor

Unverified Answer

How many people get leukemia a year in the United States?

"In the United States, the peak age of onset for acute myeloid leukemia is 38 years, and the age at peak incidence is 44 years; the average age of diagnosis is 62 years. The majority of people with acute myeloid leukemia are in the second decade of life when they are diagnosed with the disease, suggesting an age association with exposure to the environment and/or genetic factors. The median overall survival for first-time acute myeloid leukemia is only 3-4 months; the five-year survival rate is only 14%." - Anonymous Online Contributor

Unverified Answer

What are the signs of leukemia?

"The signs of leukemia include fever, weight loss, easy bruising, and pale or bluish skin. Bone pain, numbness or tingling also may be reported by patients with leukemias. In addition, diarrhea, constipation and easy bruising may be the first manifestation of a bone marrow disorder. If leukemia is suspected based on the symptoms, leukocyte counts and marrow aspirate and biopsy are strongly indicated." - Anonymous Online Contributor

Unverified Answer

What is leukemia?

"Leukemia is a blood disease that results when abnormal white blood cells grow and multiply at an abnormal rate. This disease usually follows an indolent but rapid course with symptoms ranging from only mild or absent, to severe, debilitating and sometimes fatal, symptoms. The disease will typically strike adults between the ages of 20-30 and will typically cause a bone marrow infection with leukemic cells. The disease is frequently treated with cytotoxic therapy with or without high-dose chemotherapy, a chemotherapeutic drug that selectively targets rapidly dividing cells." - Anonymous Online Contributor

Unverified Answer

Can leukemia be cured?

"This patient was diagnosed with AML, a disease fatal in 3% of people, in our study. Our patient had not been treated with cytotoxic agents, and he did not survive. He died after a prolonged, severe and painful battle with leukemia, leaving his wife struggling with his disease and death while he was never aware of the severity of his disease or its ultimate outcome. His autopsy revealed several severe problems with most of the body, including sepsis, leukemia, and many severe infections. This case demonstrates the ultimate failure to live with the diagnosis of AML and to survive with therapy." - Anonymous Online Contributor

Unverified Answer

What causes leukemia?

"The development of leukemia is likely the result of an error in the cellular formation in the bone marrow. This could be due to a blood disorder called imatinibs syndrome, or it may be due to genetics. When combined with a toxin, the likelihood that a child will develop leukemia can be increased. All of this is dependent on the development of the child and the amount and type of toxins he or she is exposed to.\n" - Anonymous Online Contributor

Unverified Answer

What are the latest developments in pharmacological study for therapeutic use?

"The data presented is for three major drugs: etoposide, topotecan and mitoxantrone. Etoposide and topotecan are known to increase cell turnover in tumor cells. Mitoxantrone is a synthetic anthranilic acid antibiotic. It inhibits DNA replication by crosslinking the DNA of dividing cells and interfering with their mitosis. There has been recent development in these drugs use and they are being used more commonly. This is one of the newest drugs for hematological and malignancy purposes. Since its use in the past 15 years this has become a medication. The dosage of Topotecan has lately been changed; once a day was being used instead." - Anonymous Online Contributor

Unverified Answer

How serious can leukemia be?

"This article aims to help people manage their leukemic condition for better care. The treatment depends on where the leukemic cells originate from, and how fast the leukemia gets worse, also, depends on the type of leukemia you are having. If the leukemia gets worse too fast, it may need to be treated quickly before the leukemia turns into acute leukemia (a form of chronic leukemia). If the leukemia is present for a while or doesn't rapidly get worse it is treated once per month for the first time, then once per month but the last treatment of the month should be given 4 weeks later and the next month and so on until it finishes." - Anonymous Online Contributor

Unverified Answer

Has pharmacological study proven to be more effective than a placebo?

"A meta-analysis of seven randomized controlled trials comparing the effectiveness of anakinra with placebo in the treatment of rheumatoid arthritis showed a significant increase in the likelihood of pain resolution with the anakinra treatment, which was not seen at either the early or mid to late end of follow up. Based on these data anakinra is advocated for patients with rheumatoid arthritis in order to improve their pain." - Anonymous Online Contributor

Unverified Answer

What is the average age someone gets leukemia?

"The average age of people getting leukemia is 49.8, and the typical age of death is 69.5 in 2016. There have been 2 major studies on leukemia mortality – the USA and UK, and there is another study from Sweden which gives the exact numbers of deaths from leukemia in Sweden. The study shows that leukemia as a cause of death is slightly lower in Sweden than in the US and UK. The US has on average 9 deaths per month per 100,000 population while in Sweden it is about 5 and in UK it is 3. If we see this situation in statistics, I think it is easy to explain why leukemia is still a major problem in the United States and UK." - Anonymous Online Contributor

Unverified Answer

Does pharmacological study improve quality of life for those with leukemia?

"Results from a recent paper did not demonstrate an improvement in patients with the addition of a systematic follow-up program compared with routine care or a routine follow-up programme." - Anonymous Online Contributor

Unverified Answer

What is the primary cause of leukemia?

"It was concluded that only a subset with chronic or chronic-intermittent leukemias has primary causation of leukemia. Patients with secondary causation for leukemia need prompt and appropriate therapy. If in the last five or ten years this cause is noticed then patients are typically diagnosed with secondary causation of leukemia. \n1. Chronic leukemias (also termed chronic myelogenous leukemia (CML)), e.g. Ph+CML\n2. Acute leukemias (also termed acute myelogenous leukemia (AML))\n3. Acquired leukemias (also termed AML)\n4." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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