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

Study Overview
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Brief Summary
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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
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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.

Official Title
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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

Conditions
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Leukemia, Myeloid, Acute

Intervention / Treatment
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* Drug: Troxatyl™ (troxacitabine)


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Intervention/Treatment |
| --- |
|Drug: Troxatyl™ (troxacitabine)|nan|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To determine complete response (CR) rate |  |  | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To determine the complete response with incomplete platelet recovery (CRp) rate, and recurrent disease (RD) rates |  |  | 
| Efficacy and safety, preliminary evidence of the anti-tumor activity, determination of Troxatyl™ infusion pharmacokinetic parameters |  |  | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Acute myeloid leukemia, AML, refractory, relapsed