A Phase 1, Multicenter, Open-Label, Dose-Escalation, Safety, Pharmacokinetic, Pharmacodynamic, and Clinical Activity Study of Orally Administered AG-221 in Subjects With Advanced Hematologic Malignancies With an IDH2 Mutation

The purpose of this Phase 1, multi-center study is to evaluate the safety, pharmacokinetics, pharmacodynamics and clinical activity of AG-221 in advanced hematologic malignancies that harbor an IDH2 mutation. The first portion of the study is a dose escalation phase where cohorts of patients will receive ascending oral doses of AG-221 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-221 to further evaluate the safety, tolerability, and clinical activity. Anticipated time on study treatment is until disease progression or unacceptable toxicity occurs.

Stanford is not currently accepting new patients for this trial. You may want to check clinicaltrials.gov to see if other locations are recruiting.

Investigator(s):

Intervention(s):

  • drug : AG-221

Phase: Phase 1

Eligibility

Ages Eligible For Study:

18 Years - N/A

Inclusion Criteria

- Subject must be ?18 years of age. - Subjects must have documented IDH2 gene-mutated advanced hematologic malignancy based on local evaluation. - Subjects must be amenable to serial bone marrow biopsies, peripheral blood sampling, and urine sampling during the study. - Subjects must be able and willing to sign an informed consent. - Subjects must have ECOG PS of 0 to 2. - Platelet count ?20,000/L (Transfusions to achieve this level are allowed.) Subjects with a baseline platelet count of <20,000/L due to underlying malignancy are eligible with Medical Monitor approval. - 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 organ involvement, and serum total bilirubin ?1.5 x upper limit of normal (ULN), unless considered due to Gilbert's disease or leukemic organ involvement. - 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. Subjects with reproductive potential are defined as one who is biologically capable of becoming pregnant. Women of childbearing potential as well as fertile men and their partners must agree to abstain from sexual intercourse or to use an effective form of contraception during the study and for 90 days (females and males) following the last dose of AG-221.

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Contact information

Primary Contact:

Leilani Hong Lien 650-725-0437

Stanford University School of Medicine 300 Pasteur Drive Stanford, CA 94305

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