Acute Myeloid Leukemia, Advanced MDS
Conditions
Keywords
preleukemia, dysplasia of myeloid blood cells
Brief summary
This is a 2-phase study during which patients with select myeloid leukemias or advanced myelodysplastic syndrome (MDS), who have failed, refused or are not eligible for standard treatment, will receive investigational study drug ARRY-520. The study has 3 parts. The first phase of the study, Phase 1, has 2 parts. In the first part of Phase 1, patients with select myeloid leukemias or advanced MDS will receive increasing doses of study drug on different schedules in order to achieve the highest dose possible that will not cause unacceptable side effects. Approximately 30 patients (per schedule) from the US will be enrolled in Part 1 (Completed). In the second part of Phase 1, patients with advanced MDS will receive the best dose of study drug and schedule determined from the first part of the study. Approximately 10 patients from the US will be enrolled in Part 2 (Completed). In the third part of the study, Phase 2, patients with acute myeloid leukemia (AML) or advanced MDS will receive the best dose of study drug and schedule determined from the first part of the study and will be followed to see what side effects the study drug causes and to see what effectiveness it has, if any, in treating the cancer. Approximately 40 patients from the US will be enrolled in Part 3 (Withdrawn).
Interventions
Part 1: multiple dose, escalating; Part 2: multiple dose, single schedule; Part 3: multiple dose, single schedule.
Sponsors
Pfizer
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Eligibility
Sex/Gender
ALL
Age
17 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria (Part 2): * Patients with either Intermediate-2 or High risk MDS or with AML (\>20% bone marrow blasts) with stable low or normal white blood cell count (WBC). Patients should have failed one prior chemotherapy regimen which should have included a hypomethylating agent. * Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0, 1 or 2. * Discontinuation of prior treatment at least 2 weeks prior to the start of the study. * Adequate hepatic and renal function. * Additional criteria exist. Key
Exclusion criteria
(Part 2): * Concurrent cytotoxic therapy, or biological, endocrine and immunological response modifiers. * Previous radiation to \>25% of bone marrow. * Other active malignancies. * Known positive serology for the human immunodeficiency virus (HIV). * Central nervous system involvement as documented by spinal fluid cytology. * Active, uncontrolled infection. * Additional criteria exist
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Establish the maximum tolerated dose (MTD) of the study drug. | Part 1 |
| Characterize the pharmacokinetics (PK) of the study drug. | Part 1 |
| Characterize the safety profile of the study drug in terms of adverse events, dose limiting toxicity, clinical laboratory tests, weight, electrocardiograms and physical examinations. | Part 1 and Part 2 |
| Assess the efficacy of the study drug in terms of incidence of complete remission (CR) and hematologic improvement (CRp). | Part 3 |
Secondary
| Measure | Time frame |
|---|---|
| Assess the efficacy of the study drug in terms of incidence of CR and CRp. | Part 1 and Part 2 |
| Characterize the safety profile of the study drug in terms of adverse events, dose limiting toxicity, clinical laboratory tests, weight, electrocardiograms and physical examinations. | Part 3 |
Countries
United States
Outcome results
None listed