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IDH1 Inhibitor

Ivosidenib + Azacitidine for Acute Myeloid Leukemia (AGILE Trial)

Phase 3
Waitlist Available
Research Sponsored by Institut de Recherches Internationales Servier
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be ≥ 18 years of age
Have previously untreated AML with ≥ 20% leukemic blasts in the bone marrow
Must not have
Have received a hypomethylating agent for myelodysplastic syndrome (MDS)
Are candidates for and willing to receive intensive induction chemotherapy (IC) for their AML
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to approximately 52 months
Awards & highlights
Pivotal Trial

Summary

This trial is testing a new oral drug, ivosidenib, combined with an injectable drug, azacitidine. It targets adults with a specific type of untreated leukemia who can't undergo intensive treatments. Ivosidenib blocks a faulty enzyme in cancer cells, and azacitidine stops these cells from growing.

Who is the study for?
This trial is for adults with previously untreated Acute Myeloid Leukemia (AML) who have an IDH1 mutation and are not suitable for intensive chemotherapy. They must have good liver and kidney function, agree to regular blood and bone marrow tests, understand the study, consent to it, and if of childbearing potential, use two forms of contraception.
What is being tested?
The trial is testing AG-120 (Ivosidenib) combined with Azacitidine against a placebo plus Azacitidine in patients with AML. It's a global Phase 3 study where participants are randomly assigned to either treatment group. The main goal is to see how long patients live without their disease getting worse.
What are the potential side effects?
Possible side effects include issues affecting the heart rhythm or electrical activity (QT interval changes), allergic reactions to medication components, infection risks due to weakened immune system from leukemia or its treatments, as well as general drug-related side effects like nausea or fatigue.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am 18 years old or older.
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My leukemia has not been treated and my bone marrow shows a high number of cancer cells.
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My cancer has an IDH1 mutation.
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I can take care of myself and am up and about more than half of the day.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have been treated with a drug to modify my DNA for MDS.
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I am willing and able to undergo strong chemotherapy for my AML.
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I have been treated with an IDH1 inhibitor before.
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I am currently pregnant or breastfeeding.
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I have trouble swallowing or absorbing pills.
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I have symptoms or a diagnosis of leukemia in my brain or spinal cord.
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I am experiencing severe, life-threatening complications from leukemia.
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I have been diagnosed with progressive multifocal leukoencephalopathy.
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I have an ongoing infection that hasn't improved with treatment.
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I have a condition that could affect my heart's rhythm.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to approximately 52 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to approximately 52 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Event-Free Survival (EFS)
Secondary study objectives
CR + CRi (Including CRp) Rate
CR + Complete Remission With Partial Hematologic (CRh) Rate
Change From Baseline in the EORTC EQ-5D-5L Questionnaire
+25 more

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: AG-120 + AzacitidineExperimental Treatment2 Interventions
Participants received AG-120 500 mg orally, once daily (QD) in combination with azacitidine 75 milligrams per square meter per day (mg/m\^2/day) subcutaneously (SC) or intravenously (IV), on Days 1-7, or on Days 1-5 and 8-9, of each 28-day cycle for a minimum of 6 cycles until death, disease relapse, disease progression, development of unacceptable toxicity (adverse event), confirmed pregnancy, withdrawal by participant or protocol violation.
Group II: Placebo + AzacitidinePlacebo Group2 Interventions
Participants received AG-120 matching placebo orally, QD in combination with azacitidine 75 mg/m\^2/day SC or IV, on Days 1-7, or on Days 1-5 and 8-9, of each 28-day cycle for a minimum of 6 cycles until death, disease relapse, disease progression, development of unacceptable toxicity (adverse event), confirmed pregnancy, withdrawal by participant or protocol violation .
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
AG-120
2017
Completed Phase 3
~370
Azacitidine
2012
Completed Phase 3
~1440

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
The most common treatments for Acute Myeloid Leukemia (AML) include targeted therapies, hypomethylating agents, and traditional chemotherapy. Ivosidenib, a targeted therapy, inhibits the mutant IDH1 enzyme, which is crucial in halting the production of the oncometabolite 2-hydroxyglutarate, thereby restoring normal cell differentiation. Hypomethylating agents like azacitidine and decitabine work by incorporating into DNA and inhibiting DNA methyltransferase, leading to reactivation of tumor suppressor genes. Traditional chemotherapy, such as cytarabine and daunorubicin, targets rapidly dividing cells by interfering with DNA synthesis and function. These treatments are vital for AML patients as they address the underlying genetic and cellular abnormalities, aiming to induce remission and improve survival rates.
The Molecular Mechanisms of Resistance to IDH Inhibitors in Acute Myeloid Leukemia.New drugs approved for acute myeloid leukaemia in 2018.Molecular targeting in acute myeloid leukemia.

Find a Location

Who is running the clinical trial?

Institut de Recherches Internationales ServierLead Sponsor
89 Previous Clinical Trials
66,968 Total Patients Enrolled

Media Library

AG-120 (Ivosidenib) (IDH1 Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT03173248 — Phase 3
Acute Myeloid Leukemia Research Study Groups: Placebo + Azacitidine, AG-120 + Azacitidine
Acute Myeloid Leukemia Clinical Trial 2023: AG-120 (Ivosidenib) Highlights & Side Effects. Trial Name: NCT03173248 — Phase 3
AG-120 (Ivosidenib) (IDH1 Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03173248 — Phase 3
~18 spots leftby Oct 2025