Actively Recruiting

Phase 1
Age: 18Years +
All Genders
NCT02074839

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

Led by Institut de Recherches Internationales Servier · Updated on 2025-06-08

291

Participants Needed

30

Research Sites

626 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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.

CONDITIONS

Official Title

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

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Age 18 years or older
  • Documented IDH1 R132 gene-mutated advanced hematologic malignancy
  • Able to undergo serial bone marrow biopsies, peripheral blood sampling, and urine sampling during the study
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2
  • Platelet count of at least 20,000/µL (transfusions allowed to reach this level)
  • Adequate liver function with AST, ALT, and ALP less than or equal to 3 times the upper limit of normal unless due to leukemia, and total bilirubin less than or equal to 1.5 times the upper limit of normal unless due to Gilbert's disease or leukemia
  • Adequate kidney function with serum creatinine less than or equal to 2 times the upper limit of normal or creatinine clearance greater than 40 mL/min
  • Recovered from any significant side effects of prior cancer treatments
  • Female participants with reproductive potential must have a negative pregnancy test within 7 days before starting study drug
Not Eligible

You will not qualify if you...

  • Hematopoietic stem cell transplant within 60 days before first dose or ongoing immunosuppressive therapy post-transplant, or clinically significant graft-versus-host disease (except stable oral steroids or topical treatment for skin GVHD)
  • Received systemic anticancer therapy or radiotherapy less than 14 days before first dose (hydroxyurea allowed)
  • Received an investigational agent less than 14 days before first dose
  • Pregnant or breastfeeding
  • Active severe infection or unexplained fever over 38.5°C at screening or first dose (tumor fever may be allowed)
  • New York Heart Association Class III or IV heart failure or left ventricular ejection fraction less than 40% within 28 days before first dose
  • History of heart attack within 6 months before screening
  • Known unstable or uncontrolled angina or severe ventricular arrhythmias
  • QT interval corrected for heart rate (QTc) of 450 ms or longer or other risk factors for arrhythmia
  • Taking medications known to prolong QT interval
  • Known infection with HIV or active hepatitis B or C
  • Symptoms or diagnosis of active central nervous system leukemia
  • Life-threatening severe complications of leukemia such as uncontrolled bleeding, pneumonia with hypoxia or shock, or disseminated intravascular coagulation

AI-Screening

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Trial Site Locations

Total: 30 locations

1

University of Alabama at Birmingham

Birmingham, Alabama, United States, 35294

Active, Not Recruiting

2

Mayo Clinic-AZ

Phoenix, Arizona, United States, 85259

Terminated

3

City of Hope

Duarte, California, United States, 91010

Active, Not Recruiting

4

University of California-Los Angeles

Los Angeles, California, United States, 90095

Terminated

5

University of California-San Francisco

San Francisco, California, United States, 94143

Terminated

6

University of Colorado Denver

Aurora, Colorado, United States, 80045

Terminated

7

Mayo Clinic-Jacksonville

Jacksonville, Florida, United States, 32224

Terminated

8

University of Miami

Miami, Florida, United States, 33136

Terminated

9

Moffit Cancer Center

Tampa, Florida, United States, 33612

Active, Not Recruiting

10

Emory University

Atlanta, Georgia, United States, 30322

Active, Not Recruiting

11

Northwestern University Medical Hospital

Chicago, Illinois, United States, 60611

Terminated

12

John Hopkins Cancer Center

Baltimore, Maryland, United States, 21287

Active, Not Recruiting

13

Massachusetts General Hospital

Boston, Massachusetts, United States, 02214

Terminated

14

Dana Farber Cancer Institute

Boston, Massachusetts, United States, 02215

Terminated

15

Karmanos Cancer Center

Detroit, Michigan, United States, 48201

Terminated

16

Washington University

St Louis, Missouri, United States, 63110

Terminated

17

Memorial Sloan Kettering Cancer Center

New York, New York, United States, 10021

Active, Not Recruiting

18

Cornell Cancer Center

New York, New York, United States, 10065

Terminated

19

Duke Cancer Center

Durham, North Carolina, United States, 27705

Terminated

20

Cleveland Clinic

Cleveland, Ohio, United States, 44124

Withdrawn

21

Ohio State University

Columbus, Ohio, United States, 43210

Terminated

22

Oregon Health and Science University

Portland, Oregon, United States, 97239

Terminated

23

Medical University of South Carolina

Charleston, South Carolina, United States, 29425

Actively Recruiting

24

Sarah Cannon Research Institute

Nashville, Tennessee, United States, 37203

Terminated

25

UT Southwestern Medical Center

Dallas, Texas, United States, 75390

Actively Recruiting

26

MD Anderson Cancer Center

Houston, Texas, United States, 77030

Actively Recruiting

27

Hopital La Timone

Marseille, France

Terminated

28

Hopital Haut-Leveque

Pessac, France, 33600

Actively Recruiting

29

Central Lyon Sud

Pierre-Bénite, France, 69310

Actively Recruiting

30

Institute Gustave Roussly (IGR)

Villejuif, France, 94800

Actively Recruiting

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Research Team

I

Institut de Recherches Internationales Servier Clinical Studies Department

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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