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IDH Mutant Targeted Therapy

IDH Mutant Targeted Therapies + Azacitidine for Acute Myeloid Leukemia

Phase 1 & 2
Waitlist Available
Research Sponsored by Celgene
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Newly diagnosed, primary (ie, de novo) or secondary (progression of Myelodysplastic syndrome [MDS] or myeloproliferative neoplasms [MPN], or therapy-related) acute myeloid leukemia (AML) according to the WHO classification with ≥ 20% leukemic blasts in the bone marrow
Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2
Must not have
AML secondary to chronic myelogenous leukemia (CML)
Suspected or proven to have acute promyelocytic leukemia based on morphology, immunophenotype, molecular assay, or karyotype
Timeline
Screening 3 weeks
Treatment Varies
Follow Up from first dose up to approximately 26 months
Awards & highlights
No Placebo-Only Group

Summary

This trial will study two drugs, AG-120 and AG-221, in combination with azacitidine, to see if they are safe and effective in treating AML with IDH1 or IDH2 mutations.

Who is the study for?
This trial is for adults with a new diagnosis of acute myeloid leukemia (AML) that has IDH mutations and can't handle strong chemotherapy. They should be able to undergo regular bone marrow tests, have an ECOG performance status of 0-2, and not have had previous IDH-targeted treatments or certain types of AML.
What is being tested?
The study is testing the safety and effectiveness of two drug combinations: AG-120 plus Azacitidine, and AG-221 plus Azacitidine. It aims to find the best doses when used together and compare them against using Azacitidine alone in treating AML with specific genetic changes.
What are the potential side effects?
Possible side effects from these drugs may include nausea, vomiting, fatigue, liver problems like jaundice or elevated enzymes levels, nerve damage symptoms such as numbness or tingling sensations, diarrhea or constipation.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been diagnosed with AML with more than 20% cancer cells in my bone marrow.
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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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I agree to have multiple bone marrow tests.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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My AML developed from chronic myelogenous leukemia.
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I am diagnosed or suspected to have a specific type of leukemia.
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I have been treated with a drug targeting IDH1 or IDH2 mutation.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~from randomization to 1 year after randomization
This trial's timeline: 3 weeks for screening, Varies for treatment, and from randomization to 1 year after randomization for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Overall Response Rate: Phase 2 (Randomized Stage)
The Number of Participants Experiencing Adverse Events: Phase 1B (Dose Finding and Expansion Stage)
The Number of Participants Experiencing Dose-limiting Toxicities (DLTs): Phase 1B (Dose Finding Stage)
Secondary study objectives
AUC (0-24)- Area Under the Plasma Concentration-Time Curve: Phase 2 (Randomized Stage)
AUC (0-8)- Area Under the Plasma Concentration-Time Curve: Phase 1B (Expansion Stage)
AUC (0-8)- Area Under the Plasma Concentration-Time Curve: Phase 2 (Randomized Stage)
+21 more

Side effects data

From 2021 Phase 3 trial • 187 Patients • NCT02989857
28%
Nausea
28%
Diarrhoea
23%
Fatigue
21%
Oedema peripheral
16%
Abdominal pain
16%
Anaemia
14%
Decreased appetite
14%
Weight decreased
12%
Vomiting
12%
Asthenia
12%
Cough
12%
Ascites
12%
Constipation
12%
Arthralgia
9%
Hypertension
9%
Abdominal pain upper
9%
Dizziness
9%
Muscle spasms
9%
Muscular weakness
9%
Dyspnoea
9%
Blood alkaline phosphatase increased
7%
Upper respiratory tract infection
7%
Hypoalbuminaemia
7%
Pruritus
7%
Hypophosphataemia
7%
Aspartate aminotransferase increased
7%
Alanine aminotransferase increased
7%
Insomnia
7%
Abdominal discomfort
5%
Rash
5%
Hypokalaemia
5%
Back pain
5%
White blood cell count decreased
5%
Hyperglycaemia
5%
Hyperkalaemia
5%
Pyrexia
5%
Headache
5%
Abdominal distension
5%
Blood bilirubin increased
5%
Confusional state
5%
Platelet count decreased
2%
Chills
2%
Electrocardiogram QT prolonged
2%
Cholangitis
2%
Gastrointestinal haemorrhage
2%
Intestinal pseudo-obstruction
2%
Biliary obstruction
2%
Bacteraemia
2%
Clostridium difficile colitis
2%
Escherichia bacteraemia
2%
Hip fracture
2%
Hypercalcaemia
2%
Encephalopathy
2%
Acute kidney injury
2%
Hypotension
2%
Gastrooesophageal reflux disease
2%
Hypomagnesaemia
2%
Blood creatinine increased
2%
Dyspepsia
2%
Urinary tract infection
2%
Rash maculo-papular
2%
Dry mouth
2%
Multiple sclerosis relapse
2%
Spinal cord compression
2%
Syncope
2%
Hyponatraemia
2%
Hepatic cirrhosis
2%
Oesophageal varices haemorrhage
2%
Upper gastrointestinal haemorrhage
100%
80%
60%
40%
20%
0%
Study treatment Arm
After Cross Over to AG-120
AG-120
Placebo

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

3Treatment groups
Experimental Treatment
Group I: AzacitidineExperimental Treatment1 Intervention
Group II: AG-221 + AzacitidineExperimental Treatment2 Interventions
Group III: AG-120 + AzacitidineExperimental Treatment2 Interventions
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
AG-221
2015
Completed Phase 3
~390

Find a Location

Who is running the clinical trial?

CelgeneLead Sponsor
645 Previous Clinical Trials
130,053 Total Patients Enrolled
Ira Gupta, MDStudy DirectorCelgene Corporation
Bristol-Myers SquibbStudy DirectorBristol-Myers Squibb
1,579 Previous Clinical Trials
3,387,490 Total Patients Enrolled

Media Library

AG-120 (IDH Mutant Targeted Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT02677922 — Phase 1 & 2
Acute Myeloid Leukemia Research Study Groups: AG-120 + Azacitidine, AG-221 + Azacitidine, Azacitidine
Acute Myeloid Leukemia Clinical Trial 2023: AG-120 Highlights & Side Effects. Trial Name: NCT02677922 — Phase 1 & 2
AG-120 (IDH Mutant Targeted Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02677922 — Phase 1 & 2
~14 spots leftby Dec 2025
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