Study of AG-120 for Advanced Blood Cancers with IDH1 Mutation
This study is testing an oral drug called AG-120 for people with advanced blood cancers (hematologic malignancies) that have a specific genetic change called an IDH1 mutation. This includes certain types of acute myeloid leukemia (AML) and myelodysplastic syndromes. The main goals are to find a safe and effective dose of AG-120, understand how it works in the body, and see if it helps improve your condition. You would take AG-120 daily in 28-day cycles. To join, you must be at least 18 years old and have a confirmed IDH1 mutation. The study is currently ongoing, but its exact status is unclear.
- Study design
- This is a Phase 1, multi-center study with an estimated 291 participants. It has two parts: a dose escalation phase to find the best dose, and a dose expansion phase to further evaluate that dose.
- What's involved
- You would take AG-120 orally every day in 28-day cycles. You would also need to have serial bone marrow biopsies, peripheral blood (blood from a vein) sampling, and urine sampling.
- Compensation
- Not stated in the trial record.
- Follow-up
- Safety, tolerability, and clinical activity will be measured for up to 26 weeks on average.
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NCT02074839
Study of Orally Administered AG-120 in Subjects With Advanced Hematologic Malignancies With an IDH1 Mutation
Recruiting
PHASE1Ages 18+InterventionalTreatmentInstitut de Recherches Internationales ServierInvestigator-initiated
~291 participants
Updated 2026-06-15 on ClinicalTrials.gov
What's tested:AG-120
At a glance
Recruiting sites
6 of 30 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Safety/tolerability: incidence of adverse events.
Measured over up to 26 weeks, on average
+4 more outcomes measured
Conditions
Where it's being run
30 sites across 19 statesFrance4
California3
Florida3
Massachusetts2
New York2
Ohio2
Texas2
Alabama1
Who to contact
Institut de Recherches Internationales Servier Clinical Studies Department
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Eligibility criteria
Inclusion
Subject must be ≥18 years of age.
Subjects must have documented IDH1 R132 gene-mutated advanced hematologic malignancy based on local or central evaluation.
Subjects must be amenable to serial bone marrow biopsies, peripheral blood sampling, and urine sampling during the study.
Subjects must have ECOG PS of 0 to 2.
Platelet count ≥20,000/µL (Transfusions to achieve this level are allowed).
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)
Subjects must be recovered from any clinically relevant toxic effects of any prior surgery, radiotherapy, or other therapy intended for the treatment of cancer.
Female subjects with reproductive potential must have a negative serum pregnancy test within 7 days prior to the start of therapy and on the first day of study drug administration.
Exclusion
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). (The use of a stable dose of oral steroids post HSCT and/or topical for ongoing skin GVHD is permitted.)
Subjects who received systemic anticancer therapy or radiotherapy \<14 days prior to their first day of study drug administration. (Hydroxyurea is allowed prior to enrollment and after the start of AG-120).
Subjects who received an investigational agent \<14 days prior to their first day of study drug administration.
Subjects who are pregnant or breastfeeding.
Subjects with an active severe infection or with an unexplained fever \>38.5°C during screening visits or on their first day of study drug administration (at the discretion of the Investigator, subjects with tumor fever may be enrolled).
Subjects with New York Heart Association (NYHA) Class III or IV congestive heart failure or LVEF \<40% by echocardiogram (ECHO) or multi-gated acquisition (MUGA) scan within approximately 28 days of C1D1.
Subjects with a history of myocardial infarction within the last 6 months of screening.
Subjects with a known unstable or uncontrolled angina pectoris.
Subjects with a known history of severe and/or uncontrolled ventricular arrhythmias.
Subjects with known unstable or uncontrolled angina pectoris.
Subjects with heart-rate corrected QT (QTc) interval ≥450 ms or other factors that increase the risk of QT prolongation or arrhythmic events.
Patients taking medications that are known to prolong the QT interval
Subjects with known infection with human immunodeficiency virus (HIV) or active hepatitis B or C.
Subjects with clinical symptoms suggesting active central nervous system (CNS) leukemia or known CNS leukemia. Evaluation of cerebrospinal fluid is only required if there is a clinical suspicion of CNS involvement by leukemia during screening.
Subjects with immediately life-threatening, severe complications of leukemia such as uncontrolled bleeding, pneumonia with hypoxia or shock, and/or disseminated intravascular coagulation.
What this trial measures
- Safety/tolerability: incidence of adverse events.up to 26 weeks, on average
- Maximum Tolerated Dose and/or the recommended Phase II dose of AG-120 in subjects with advanced hematologic malignancies.up to 26 weeks, on average
- Assess clinical activity of AG-120 in subjects with relapsed or refractory AML who are enrolled in the Expansion Phase.up to 26 weeks, on average
- Safety/tolerability of treatment with AG-120 in subjects with relapsed or refractory myelodysplastic syndrome.up to 26 weeks, on average
- Assess clinical activity of AG-120 in subjects with relapsed or refractory myelodysplastic syndrome.up to 26 weeks, on average