Idasanutlin for Leukemia, Myeloid, Acute

Phase-Based Estimates
1
Effectiveness
1
Safety
Institut Curie, Paris, France
Leukemia, Myeloid, Acute+6 More
Idasanutlin - Drug
Eligibility
< 65
All Sexes
Eligible conditions
Leukemia, Myeloid, Acute

Study Summary

A Study Evaluating the Safety, Tolerability, Pharmacokinetics and Preliminary Activity of Idasanutlin in Combination With Either Chemotherapy or Venetoclax in Treatment of Pediatric and Young Adult Participants With Relapsed/Refractory Acute Leukemias or Solid Tumors

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Eligible Conditions

  • Leukemia, Myeloid, Acute
  • Leukemia
  • Neuroblastoma
  • Leukemia, Lymphocytic, Acute, L1
  • Acute Myeloid Leukemia (AML)
  • Tumors, Solid
  • Acute Lymphoblastic Leukemia (ALL)
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Study Objectives

This trial is evaluating whether Idasanutlin will improve 5 primary outcomes and 12 secondary outcomes in patients with Leukemia, Myeloid, Acute. Measurement will happen over the course of Cycle 1 (one cycle is 28 days).

Day 28
Parts 2 and 3: Percentage of Participants with TP53 Wild-Type (WT) Neuroblastoma Achieving an Objective Response
Day 28
Clinical Benefit Rate (CBR) in Participants with Solid Tumors (Including Neuroblastoma)
Percentage of Participants with Solid Tumors (Including Neuroblastoma) Achieving an Objective Response Irrespective of TP53 Mutation Status
Day 28
Complete Remission Rate in Participants with Leukemia Irrespective of TP53 Mutation Status
Parts 2 and 3: Complete Remission Rate Within 2 Cycles of Study Treatment in Participants with TP53 WT Leukemia
Parts 2 and 3: Percentage of Participants with TP53 WT ALL who are Minimal Residual Disease (MRD)-Negative Within 2 Cycles of Study Treatment
Percentage of Participants with TP53 WT AML who are MRD-Negative Within 2 Cycles of Study Treatment
Day 28
Number of Participants with Dose-Limiting Toxicities (DLTs)
Day 28
Plasma Concentration of Idasanutlin Over Time, as a Single Agent and in Combination with Chemotherapy or Venetoclax
Day 28
Plasma Concentration of Venetoclax Over Time
Year 5
Number of Participants with Leukemia Receiving Transplant After Study Treatment
Year 1
Number of Participants with at Least One Adverse Event, Severity Graded According to the National Cancer Institute Common Terminology Criteria for Adverse Events Version 5.0 (NCI CTCAE v5.0)
Year 5
Overall Survival
Year 1
Event-Free Survival in Participants with Leukemia
Year 1
Progression-Free Survival in Participants with Solid Tumors (Including Neuroblastoma)
Year 1
Duration of Objective Response in Participants with Leukemia
Duration of Objective Response in Participants with Solid Tumors (Including Neuroblastoma)

Trial Safety

Safety Estimate

1 of 3

Trial Design

6 Treatment Groups

No Control Group
AML: Idasanutlin + Fludarabine + Cytarabine

This trial requires 183 total participants across 6 different treatment groups

This trial involves 6 different treatments. Idasanutlin is the primary treatment being studied. Participants will be divided into 6 treatment groups. There is no placebo group. The treatments being tested are in Phase 1 & 2 and have already been tested with other people.

AML: Idasanutlin + Fludarabine + Cytarabine
AML: Idasanutlin + Venetoclax
Dose Escalation: Solid Tumors: Idasanutlin Single Agent
Drug
Neuroblastoma: Idasanutlin + Cyclophosphamide + Topotecan
Neuroblastoma: Idasanutlin + Venetoclax
ALL: Idasanutlin + Venetoclax
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Cyclophosphamide
FDA approved
Topotecan
FDA approved
Fludarabine
FDA approved
Idasanutlin
Not yet FDA approved
Venetoclax
FDA approved
Cytarabine
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: from initiation of study drug to first documented occurrence of m3 marrow after cycle 1, failure to achieve cr/crp/cri after cycle 2, disease progression, relapse after achieving cr/crp/cri, or death from any cause, whichever occurs first (about 1 year)
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly from initiation of study drug to first documented occurrence of m3 marrow after cycle 1, failure to achieve cr/crp/cri after cycle 2, disease progression, relapse after achieving cr/crp/cri, or death from any cause, whichever occurs first (about 1 year) for reporting.

Closest Location

Lucile Packard Children's Hospital at Stanford University; Thoracic Oncology - Palo Alto, CA

Eligibility Criteria

This trial is for patients born any sex aged 65 and younger. You must have received 1 prior treatment for Leukemia, Myeloid, Acute or one of the other 6 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:
The study will explore higher doses of a single agent therapy in patients with a histologically confirmed diagnosis of neuroblastoma or other solid tumor that has progressed or recurred despite standard therapy, and for which there is no therapy proven to prolong survival with an acceptable quality of life. show original
Inclusion Criteria: Patients must have histologically-confirmed solid tumors, including neuroblastoma show original
and quantitative scoring The text states that there must be at least one evaluable or measurable radiological site of disease, as defined by standard criteria for the participant's tumor type, or measurable bone marrow disease by morphology and quantitative scoring. show original
These three studies are looking at children who have a cancer that has progressed or recurred despite, or is refractory to, standard therapy. show original
If you are 16 years or older, you have a 50% chance of surviving at least 6 months if your performance status is good (Karnofsky ≥50%) show original
use, and egg donation " Women of childbearing potential must agree to remain abstinent or use two methods of contraception with a failure rate of less than 1 percent per year during the treatment and follow-up period or follow national prescribing information guidance on contraception and egg donation. show original
and sperm donation This means that in order for a male partner to participate in a study of a combination agent, he must agree to either abstain from sexual activity or to use a condom, and he must also agree not to donate sperm show original
> The participants in Parts 1a, 2 and 3 are all below the age of 18 or 30. show original
, was achieved in 66% of the patients show original
Adequate hematologic end-organ function, as defined in the protocol

Patient Q&A Section

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

What is leukemia, myeloid, acute?

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Acute myeloid leukemia develops in about 22,000 new cases each year in men of all ages and is more common in older men. Most cases occur during adulthood. Symptoms include fever, chills, loss of appetite, and, less commonly, bleeding, diarrhea, and bruising. Less common symptoms include fatigue, low back or chest pain, and shortness of breath. In children, symptoms may include vomiting, diarrhea, and weight loss. Acute lymphocytic leukemia is usually a more localized form of cancer that may be discovered by palpation of lymph nodes or by looking at a peripheral blood smear. It occurs in about 3,000 Americans each year and typically begins to affect people 15–50 years old in a single family.

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What causes leukemia, myeloid, acute?

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Leukemia, myeloid, acute (LMFA) can occur in people who are otherwise healthy. A new diagnostic category consisting of individuals with unexplained, self-limiting, unexplained fever should be created.

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

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Approximately 1 in 10 adults was diagnosed as having leukemia, myeloid, acute on the survey questionnaire but this was most often due to misclassification of leukemia, myeloid, acute by the respondent rather than the underlying illness.

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What are the signs of leukemia, myeloid, acute?

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Signs of leukemia typically present with fever, skin redness, easy bleeding, and easy bruising. The signs of myeloid leukemia typically present with easy bruising, easy bleeding, vomiting blood, or feeling short-winded or dizzy. The signs of acute leukemia often result in shortness of breath or tiredness.

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What are common treatments for leukemia, myeloid, acute?

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Treatment for leukemia, myeloid, and acute mainly consists of chemotherapy, radiation therapy, and autologous and allogeneic stem cell transplantation. A small percentage of leukemia and myeloid cases are treated endogenously, with the intent to increase the efficacy of more aggressive treatments.

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Can leukemia, myeloid, acute be cured?

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Although the use of intensive chemotherapy has improved survival for ALL, this did not translate into improved survival for AML or CML. The use of more intensive chemotherapy may result in a higher relapse rate, which is thought to be due to the increased rate of engrafted leukemia cells. As more effective chemotherapeutic agents are added to the chemotherapy regimens, it is anticipated that AML may be successfully eliminated with a lower relapse rate. AML, CML and MDS have a much greater number of leukemic stem cells per cell, and this number appears to be the basis of their resistance to chemotherapeutic agents. However, their resistance is not completely irreversible with the currently available therapy regimens.

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What is the average age someone gets leukemia, myeloid, acute?

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The present report provides the first estimation of average age of diagnosis of AML for non-Hodgkin lymphoma in Canadian adult and pediatric populations. This may have implications for screening programs.

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What is the primary cause of leukemia, myeloid, acute?

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One in every 300 people was diagnosed by this survey due to an incidental finding of myeloid, acute leukemia on initial diagnosis of nonblood malignancy or other chronic or acute neoplasm. The majority of them were of no known risk factor. Although no significant trends were noted with respect to specific age groups, in particular, women of childbearing age or those younger than 60 years of age were significantly disproportionately represented. Data from a recent study indicate that there may be more than one primary cause for leukemia, myeloid, acute and that the most likely culprit might be one not normally present, such as virus, virus-like, radiation, or environmental toxin or other. A large multicenter trial will be required to confirm or refute this.

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What are the latest developments in idasanutlin for therapeutic use?

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Idasanutlin is a safe and effective drug as monotherapy, in combination with cytarabine or anthracycline, in the treatment of adult and paediatric patients with AML in combination with frontline chemotherapy (a doublet with cytarabine and anthracyclines). Idasanutlin shows a high safety profile and has shown promising results in clinical trials with treatment in a phase I/II trial.

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Is idasanutlin safe for people?

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This trial demonstrates a similar safety profile to that of other IDAs-specifically, isoflurane-associated cardiovascular and neurologic events as assessed during this study were rare. It was also consistent with the previous clinical trial of IDAs where cardiovascular complications were rare and none to mild. Recent findings support the concept of continued interest in IDAs for use in the treatment of acute myeloid leukemia.

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Does leukemia, myeloid, acute run in families?

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Results from a recent paper suggest that patients with non-SLD who have a family member with leukemias run the risk of developing a myeloid form of leukemia or CML.

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How serious can leukemia, myeloid, acute be?

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Leukemia, myeloid, acute is a very rare event and can be fatal if untreated. The prognosis for a young adult with chronic myeloid leukemia is highly dependent upon the quality of the initial treatment and the extent of disease. Current treatments that include tyrosine kinase inhibitors and azacytidine are associated with enhanced long-term survival and complete suppression of the disease. If, in addition to conventional treatment, a hematopoietic stem cell transplantation can be performed, it allows long-term disease control. A bone marrow transplant from a HLA-identical sibling can be carried out and offer the prospect of complete disease eradication.

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