Clinical trial NCT00129948: Study for Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML)
Official title: An Open-Label, Single-Arm, Multi-Center, Phase II Study of Troxatyl™ (Troxacitabine) Administered by Continuous Infusion in Patients With Acute Myeloid Leukemia (AML) in Second Salvage
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Leukemia, Myeloid, Acute
Keywords: Acute myeloid leukemia
AML
refractory
relapsed
Lead sponsor: SGX Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2005-08-12
Enrollment: 211
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: To determine complete response (CR) rate
Secondary outcomes:
measure: To determine the complete response with incomplete platelet recovery (CRp) rate, and recurrent disease (RD) rates
measure: Efficacy and safety, preliminary evidence of the anti-tumor activity, determination of Troxatyl™ infusion pharmacokinetic parameters
Other outcomes:
Locations:
city: LaJolla
country: United States
facility: Scripps Clinic
state: California
city: Los Angeles
country: United States
facility: USC-Norris Neuro-Oncology Program
state: California
city: San Diego
country: United States
facility: UCSD Moores Cancer Center
state: California
city: Jacksonville
country: United States
facility: Univ. of Florida, Baptist Cancer Center
state: Florida
city: Atlanta
country: United States
facility: Winship Cancer Institute, Emory University Hosp.
state: Georgia
city: Chicago
country: United States
facility: University of Chicago
state: Illinois
city: Maywood
country: United States
facility: Loyola University Medical Center
state: Illinois
city: Boston
country: United States
facility: Dana Farber Cancer Institute
state: Massachusetts
city: Detroit
country: United States
facility: Harper Hospital - Karmanos Cancer Center
state: Michigan
city: Minneapolis
country: United States
facility: Univ. of Minnesota Medical Center
state: Minnesota
city: St. Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: Buffalo
country: United States
facility: Roswell Park Cancer Institute
state: New York
city: New York
country: United States
facility: Memorial Sloan-Kettering Cancer Center
state: New York
city: New York
country: United States
facility: New York Presbyterian Hospital-Cornell Campus
state: New York
city: Winston-Salem
country: United States
facility: Wake Forest Univ. School of Medicine
state: North Carolina
city: Cleveland
country: United States
facility: Cleveland Clinic Foundation
state: Ohio
city: Charleston
country: United States
facility: Univ. of South Carolina, Hematology/Oncology Division
state: South Carolina
city: Houston
country: United States
facility: Univ. of Texas, MD Anderson Cancer Center
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah Huntsman Cancer Institute
state: Utah
city: Morgantown
country: United States
facility: Morgantown Internal Medicine Group
state: West Virginia
Study officials:
affiliation: M.D. Anderson Cancer Center
name: Francis Giles, MD
role: PRINCIPAL_INVESTIGATOR
References: