OncoMatch/Clinical Trials/NCT02074839
Study of Orally Administered AG-120 in Subjects With Advanced Hematologic Malignancies With an IDH1 Mutation
Is NCT02074839 recruiting? Yes, currently enrolling (May 2026). This Phase 1 trial studies AG-120 for relapsed or refractory acute myeloid leukemia (aml).
Treatment: AG-120 — The purpose of this Phase I, multicenter study is to evaluate the safety, pharmacokinetics, pharmacodynamics and clinical activity of AG-120 in advanced hematologic malignancies that harbor an IDH1 mutation. The first portion of the study is a dose escalation phase where cohorts of patients will receive ascending oral doses of AG-120 to determine maximum tolerated dose (MTD) and/or the recommended Phase II dose. The second portion of the study is a dose expansion phase where four cohorts of patients will receive AG-120 to further evaluate the safety, tolerability, and clinical activity of the recommended Phase II dose. Additionally, the study includes a substudy evaluating the safety and tolerability, clinical activity, pharmacokinetics, and pharmacodynamics of AG-120 in subjects with relapsed or refractory myelodysplastic syndrome with an IDH1 mutation. Anticipated time on study treatment is until disease progression or unacceptable toxicity occurs.
Check if I qualifyExtracted eligibility criteria
Cancer type
Acute Myeloid Leukemia
Acute Lymphoblastic Leukemia
Non-Hodgkin Lymphoma
Multiple Myeloma
Myelodysplastic Syndrome
Chronic Lymphocytic Leukemia
Biomarker criteria
Required: IDH1 r132 mutation
Performance status
ECOG 0–2(Ambulatory, capable of self-care)
Prior therapy
Cannot have received: hematopoietic stem cell transplant
Exception: HSCT >60 days prior to first dose, stable oral steroids/topical for skin GVHD permitted
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.)
Cannot have received: systemic anticancer therapy or radiotherapy
Exception: Hydroxyurea is allowed prior to enrollment and after the start of AG-120
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).
Cannot have received: investigational agent
Subjects who received an investigational agent <14 days prior to their first day of study drug administration.
Lab requirements
Blood counts
Platelet count ≥20,000/µL (Transfusions to achieve this level are allowed)
Kidney function
serum creatinine ≤2.0 × ULN or creatinine clearance >40mL/min based on Cockroft-Gault GFR
Liver function
AST, ALT, and ALP ≤3.0 × ULN unless considered due to leukemic disease; serum total bilirubin ≤1.5 x ULN unless considered due to Gilbert's disease or leukemic disease
Cardiac function
NYHA Class III or IV CHF or LVEF <40% by ECHO or MUGA within ~28 days of C1D1 excluded; history of MI within last 6 months excluded; known unstable or uncontrolled angina pectoris excluded; severe/uncontrolled ventricular arrhythmias excluded; QTc interval ≥450 ms or other factors that increase risk of QT prolongation or arrhythmic events excluded
adequate hepatic function as evidenced by: AST, ALT, and ALP ≤3.0 × ULN, unless considered due to leukemic disease and serum total bilirubin ≤1.5 x ULN, unless considered due to Gilbert's disease or leukemic disease; adequate renal function as evidenced by a serum creatinine ≤2.0 × ULN or creatinine clearance >40mL/min based on Cockroft-Gault GFR; Platelet count ≥20,000/µL (Transfusions to achieve this level are allowed); NYHA Class III or IV CHF or LVEF <40% by ECHO or MUGA within ~28 days of C1D1 excluded; history of MI within last 6 months excluded; known unstable or uncontrolled angina pectoris excluded; severe/uncontrolled ventricular arrhythmias excluded; QTc interval ≥450 ms or other factors that increase risk of QT prolongation or arrhythmic events excluded
Structured fields extracted by AI. May contain errors — verify against the official protocol.
US trial sites
- University of Alabama at Birmingham · Birmingham, Alabama
- Mayo Clinic-AZ · Phoenix, Arizona
- City of Hope · Duarte, California
- University of California-Los Angeles · Los Angeles, California
- University of California-San Francisco · San Francisco, California
Showing up to 5 US sites. See all sites on ClinicalTrials.gov →
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