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Study of Orally Administered AG-120 in Subjects With Advanced Hematologic Malignancies With an IDH1 Mutation | Clinical Trials | Clareo Health

RECRUITINGPHASE1

Study of Orally Administered AG-120 in Subjects With Advanced Hematologic Malignancies With an IDH1 Mutation

A Phase I, Multicenter, Open-Label, Dose-Escalation and Expansion, Safety, Pharmacokinetic, Pharmacodynamic, and Clinical Activity Study of Orally Administered AG-120 in Subjects With Advanced Hematologic Malignancies With an IDH1 Mutation

NCT02074839•Institut de Recherches Internationales Servier

View on ClinicalTrials.gov

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

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•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 Criteria

•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.
+5 more criteria

Locations (30)

University of Alabama at Birmingham

Birmingham, Alabama, United States

Mayo Clinic-AZ

Phoenix, Arizona, United States

City of Hope

Duarte, California, United States

University of California-Los Angeles

Los Angeles, California, United States

University of California-San Francisco

San Francisco, California, United States

University of Colorado Denver

Aurora, Colorado, United States

Mayo Clinic-Jacksonville

Jacksonville, Florida, United States

University of Miami

Miami, Florida, United States

Moffit Cancer Center

Tampa, Florida, United States

Emory University

Atlanta, Georgia, United States

Key Dates

Start Date

March 1, 2014

Primary Completion Date

March 1, 2026

Completion Date

March 1, 2026

Last Updated

June 8, 2025

Enrollment

291

ESTIMATED participants

Conditions

Relapsed or Refractory Acute Myeloid Leukemia (AML)Untreated AMLOther IDH1-mutated Positive Hematologic MalignanciesMyelodysplastic Syndromes

Interventions

AG-120

DRUG

Contacts

Institut de Recherches Internationales Servier Clinical Studies Department

CONTACT

+33 1 55 72 43 66 scientificinformation@servier.com

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: June 8, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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