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IDH1 Inhibitor

AG-120 for Blood Cancers

Phase 1
Recruiting
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
Subjects must have adequate hepatic function as evidenced by: Aspartate aminotransferase (AST), alanine aminotransferase (ALT), and alkaline phosphatase (ALP) ≤3.0 × ULN, unless considered due to leukemic disease and serum total bilirubin ≤1.5 x upper limit of normal (ULN), unless considered due to Gilbert's disease or leukemic disease
Subjects must have adequate renal function as evidenced by a serum creatinine ≤2.0 × ULN or creatinine clearance >40mL/min based on Cockroft-Gault glomerular filtration rate (GFR)
Must not have
Subjects with a known history of severe and/or uncontrolled ventricular arrhythmias
Subjects who have undergone hematopoietic stem cell transplant (HSCT) within 60 days of the first dose of AG-120, or subjects on immunosuppressive therapy post HSCT at the time of screening, or with clinically significant graft-versus-host disease (GVHD)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 26 weeks, on average
Awards & highlights

Summary

This trial is testing a new drug, AG-120, to see if it's safe and effective in treating people with advanced cancers that have a mutation in the IDH1 gene. The trial has two parts: first, they'll test different doses of the drug to see what the maximum tolerated dose is; then, they'll expand the trial to a larger group of people to see if the drug is effective. There's also a substudy testing AG-120 in people with a different but related cancer, myelodysplastic syndrome.

Who is the study for?
Adults (18+) with advanced blood cancers like AML or MDS that have a specific IDH1 mutation can join. They must be able to undergo regular bone marrow, blood, and urine tests, not be pregnant, and have an ECOG performance status of 0-2. Adequate liver and kidney function is required. Those with severe heart problems, recent cancer treatments within 14 days (except hydroxyurea), active infections including HIV or hepatitis B/C, CNS leukemia symptoms or history of it without clinical suspicion are excluded.Check my eligibility
What is being tested?
AG-120 is being tested in patients with certain mutations in their blood cancer cells. The trial has two parts: first finding the highest dose people can take without too many side effects (dose escalation) and then giving more people this dose to see how well it works and if it's safe (dose expansion). There's also a special part for those whose myelodysplastic syndrome came back after treatment.See study design
What are the potential side effects?
Specific side effects aren't listed here but generally could include issues from taking oral AG-120 such as nausea, fatigue, liver problems based on its impact on metabolism enzymes; participants will stop treatment if they experience disease progression or unacceptable toxicity.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My liver functions, including AST, ALT, and ALP levels, are within normal limits.
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My kidney function is within the required range.
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My blood cancer has a specific IDH1 R132 gene mutation.
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I am a woman who can have children and have a recent negative pregnancy test.
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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 am 18 years old or older.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have a history of severe heart rhythm problems.
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I had a stem cell transplant less than 60 days ago, or I am on immunosuppressants, or I have significant GVHD.
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I have unstable or uncontrolled chest pain.
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I have unstable or uncontrolled chest pain.
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My heart's electrical activity (QTc interval) is normal or I don't have factors that risk abnormal heart rhythms.
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I am taking medication that affects my heart's rhythm.
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I do not have HIV or active hepatitis B or C.
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I am experiencing severe, life-threatening complications from leukemia.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 26 weeks, on average
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 26 weeks, on average for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Assess clinical activity of AG-120 in subjects with relapsed or refractory AML who are enrolled in the Expansion Phase.
Assess clinical activity of AG-120 in subjects with relapsed or refractory myelodysplastic syndrome.
Maximum Tolerated Dose and/or the recommended Phase II dose of AG-120 in subjects with advanced hematologic malignancies.
+2 more
Secondary outcome measures
Blood and bone marrow sampling at specified time points for determination of 2-HG levels to characterize the percent of 2-HG inhibition of AG-120 in plasma and bone marrow.
Clinical Activity of AG-120 in advanced hematologic malignancies according to the 2003 revised IWG criteria for AML or the 2006 modified IWG criteria for MDS or MDS/myeloproliferative neoplasms (MPN).
Dose Limiting Toxicities of AG-120 in subjects with advanced hematologic malignancies.
+5 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%
Hypokalaemia
5%
Rash
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%
Electrocardiogram QT prolonged
2%
Chills
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

Trial Design

1Treatment groups
Experimental Treatment
Group I: AG-120Experimental Treatment1 Intervention
AG-120 administered continuously as a single agent dosed orally every day of a 28-day cycle.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
AG-120
2017
Completed Phase 3
~370

Find a Location

Who is running the clinical trial?

Institut de Recherches Internationales ServierLead Sponsor
87 Previous Clinical Trials
66,841 Total Patients Enrolled

Media Library

AG-120 (IDH1 Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT02074839 — Phase 1
Acute Myeloid Leukemia Research Study Groups: AG-120
Acute Myeloid Leukemia Clinical Trial 2023: AG-120 Highlights & Side Effects. Trial Name: NCT02074839 — Phase 1
AG-120 (IDH1 Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02074839 — Phase 1
~13 spots leftby Feb 2025
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