Study of Orally Administered AG-120 in Subjects With Advanced Hematologic Malignancies With an IDH1 Mutation
Hematopoietic Malignancies
Leukemia
18 Years and older, Male and Female
AG120-C-001 (primary)
NCI-2014-00720
Summary
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.
Eligibility
- Subject must be â?¥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) â?¤3.0 Ã? ULN, unless considered due to leukemic disease and serum total bilirubin â?¤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 â?¤2.0 Ã? 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. Key
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