Study of XL999 in Patients With Acute Myeloid Leukemia (AML)

Study Overview
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Brief Summary
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This clinical study is being conducted at multiple sites to determine the activity, safety and tolerability of XL999 when given weekly to patients with relapsed or newly-diagnosed AML. XL999 is a small molecule inhibitor against Flk1/kinase insert domain receptor (KDR), PDGFR, c-Kit, FLT3 and SRC. c-Kit and FLT3 are receptors commonly expressed on AML blasts.

Official Title
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A Phase 2 Study of XL999 Administered Intravenously to Subjects With Acute Myeloid Leukemia

Conditions
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Acute Myeloid Leukemia, AML

Intervention / Treatment
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* Drug: XL999


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of acute myeloid leukemia (except AML FAB-M3 or acute promyelocytic leukemia [APL]) based on the World Health Organization (WHO) classification of ≥ 20% blasts in the bone marrow or peripheral blood at initial diagnosis (prior to start of standard chemotherapy) ECOG performance status of 0 or 1 Subjects with newly-diagnosed AML or subjects with relapsed AML after at least 2 chemotherapy regimens. Adequate liver and renal function Signed informed consent Exclusion Criteria: Anticancer therapy including chemotherapeutic, biologic, or investigative agents within 30 days of XL999 treatment Hematopoietic stem cell transplantation within the previous 6 weeks Immunosuppressive therapy (eg, cyclosporine, steroids, tacrolimus) for graft-versus-host disease (GvHD) within 30 days prior to the start of XL999 The subject has not recovered to grade ≤ 1 or to within 10% of baseline from adverse events due to investigational or chemotherapeutic drugs or stem cell transplantation which were administered > 4 weeks prior to study enrollment Uncontrolled and/or concomitant illness Pregnant or breastfeeding females Known HIV

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: XL999|XL999 was administered at a dose of 2.4 mg/kg given as a 4-hour IV infusion weekly for 4 weeks. In the absence of progressive disease and unacceptable toxicity, subjects were to receive XL999 treatment weekly for up to 1 year on this study|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Hematologic and cytogenetic response rate |  | Inclusion until disease progression | 
| Safety and tolerability |  | Inclusion until 30 dyas post last treatment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Duration of hematologic response and transfusion independence |  | Inclusion until disease progression | 
| Progression-free survival |  | Inclusion until disease progression | 
| Overall survival |  | Inclusion until 180-day Follow-up post last treatment or death | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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acute myeloid leukemia, AML