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Study for Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML)

Phase 2
Terminated
Conditions
Leukemia, Myeloid, Acute
Registration Number
NCT00129948
Lead Sponsor
SGX Pharmaceuticals, Inc.
Brief Summary

This is a phase 2, single-arm, open-label, multi-center study to establish the safety and efficacy of Troxatyl™ (troxacitabine) administered as a continuous infusion for 5 days to subjects with AML.

Detailed Description

This is a phase 2, single-arm, open-label, multi-center study to establish the safety and efficacy of Troxatyl™ (troxacitabine) administered as a continuous infusion for 5 days to subjects with AML. The study will primarily assess the complete response (CR) rate of a 5-day continuous infusion of troxacitabine at 12 mg/m2/day given as second salvage therapy in adult patients with AML, with secondary objectives to determine the overall, relapse-free and event-free survival and remission duration; to determine the duration of response; to determine the complete response with incomplete platelet recovery (CRp) rate; to evaluate the tolerability and safety of 5-day continuous intravenous (IV) infusion of troxacitabine; and to determine the relationship between troxacitabine plasma concentrations, anti-leukemic activity and adverse events. Additional cycles of treatment may be given at the investigator's discretion, provided that the subject does not have progressive disease or experience a dose limiting toxicity. Bone marrow transplantation in responding subjects will be allowed.

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
211
Inclusion Criteria
  • Confirmed diagnosis of acute myeloid leukemia (AML) in the second salvage setting: refractory to two prior courses of therapy or primary refractory without response to two previous courses of leukemia therapy.

  • Patients must have received at least two previous courses of induction chemotherapy to be considered in the second salvage setting.

  • Patients who are in second relapse, must have had a duration of their second CR or CRp of less than six months.

  • Subjects must have adequate organ and immune function as indicated by the following laboratory values:

    • Creatinine clearance ≥ 45 mL/min and ≤ 125 mL/min;
    • Total bilirubin ≤ 2.0 mg/dL (≤ 34.2 µmol/L);
    • AST (SGOT) and ALT (SGPT) ≤ 3 x upper limit of normal (ULN).
    • Subjects must have an Eastern Cooperative Oncology Group (ECOG) performance status of < 2, and an estimated life expectancy of at least eight weeks.
Exclusion Criteria
  • Clinical evidence of active central nervous system (CNS) leukemic involvement
  • Active and uncontrolled infection
  • Uncontrolled medical problems unrelated to the malignancy that impair patient ability to give informed consent or unacceptably reduce the safety of the proposed treatment
  • Neurologic or psychiatric disorders that would interfere with informed consent or study follow-up
  • Known or suspected intolerance or hypersensitivity to Troxatyl® or closely related compounds such as lamivudine or any of the stated ingredients
  • A recent history of alcohol or other substance abuse
  • Subjects who have used another investigational agent or participated in a clinical trial within the last 14 days prior to enrolment
  • Females with a positive pregnancy test at screening
  • Subjects who have previously been enrolled into this study and subsequently withdrew

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Primary Outcome Measures
NameTimeMethod
To determine complete response (CR) rate
Secondary Outcome Measures
NameTimeMethod
Efficacy and safety, preliminary evidence of the anti-tumor activity, determination of Troxatyl™ infusion pharmacokinetic parameters
To determine the complete response with incomplete platelet recovery (CRp) rate, and recurrent disease (RD) rates

Trial Locations

Locations (20)

New York Presbyterian Hospital-Cornell Campus

🇺🇸

New York, New York, United States

Univ. of Florida, Baptist Cancer Center

🇺🇸

Jacksonville, Florida, United States

USC-Norris Neuro-Oncology Program

🇺🇸

Los Angeles, California, United States

Loyola University Medical Center

🇺🇸

Maywood, Illinois, United States

Roswell Park Cancer Institute

🇺🇸

Buffalo, New York, United States

Winship Cancer Institute, Emory University Hosp.

🇺🇸

Atlanta, Georgia, United States

Scripps Clinic

🇺🇸

LaJolla, California, United States

University of Chicago

🇺🇸

Chicago, Illinois, United States

Washington University School of Medicine

🇺🇸

St. Louis, Missouri, United States

Morgantown Internal Medicine Group

🇺🇸

Morgantown, West Virginia, United States

UCSD Moores Cancer Center

🇺🇸

San Diego, California, United States

Dana Farber Cancer Institute

🇺🇸

Boston, Massachusetts, United States

Harper Hospital - Karmanos Cancer Center

🇺🇸

Detroit, Michigan, United States

Univ. of Minnesota Medical Center

🇺🇸

Minneapolis, Minnesota, United States

Wake Forest Univ. School of Medicine

🇺🇸

Winston-Salem, North Carolina, United States

Cleveland Clinic Foundation

🇺🇸

Cleveland, Ohio, United States

Univ. of Texas, MD Anderson Cancer Center

🇺🇸

Houston, Texas, United States

University of Utah Huntsman Cancer Institute

🇺🇸

Salt Lake City, Utah, United States

Memorial Sloan-Kettering Cancer Center

🇺🇸

New York, New York, United States

Univ. of South Carolina, Hematology/Oncology Division

🇺🇸

Charleston, South Carolina, United States

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