Myelodysplastic Syndromes
Conditions
Keywords
Low or Intermediate-1 Risk Myelodysplastic Syndrome
Brief summary
This is a Phase 1 study during which patients with low or intermediate-1 risk myelodysplastic syndrome (MDS) will receive investigational study drug ARRY-614. This study has 2 parts. In the first part, patients will receive increasing doses of study drug, given either with food or without food, in order to achieve the highest dose of the study drug possible that will not cause unacceptable side effects. Approximately 60 patients from the US will be enrolled in Part 1 (Completed). In the second part of this study, patients will receive the best dose of study drug, given either with food or without food, determined from the first part of the study and will be followed to see what side effects and effectiveness the study drug has, if any, in treating the cancer. Approximately 40 patients from the US will be enrolled in Part 2 (Completed).
Interventions
Part 1: multiple dose, escalating; Part 2: multiple dose, single schedule.
Sponsors
Array Biopharma, now a wholly owned subsidiary of Pfizer
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria (Part 1 and Part 2): * Diagnosis of MDS by bone marrow biopsy. * International Prognostic Scoring System (IPSS) score of low or intermediate-1 risk MDS. * May have received prior therapy for MDS. * Eastern Cooperative Oncology Group (ECOG) performance status (PS) between 0 and 2. * Adequate liver and renal function. * Additional criteria exist. Key
Exclusion criteria
(Part 1 and Part 2): * History of bone marrow transplant. * Concomitant malignancies or previous malignancies with less than a 2-year disease-free interval at the time of enrollment. * Treatment with an investigational medicinal product that is not expected to be cleared by the first dose of study drug or that has demonstrated to have late side effects. * Known positive serology for the human immunodeficiency virus (HIV), hepatitis C and/or active hepatitis B. * Additional criteria exist.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Characterize the safety profile of the study drug, in either the fasted or fed state, in terms of adverse events, clinical laboratory tests and electrocardiograms. | Part 1, one year; Part 2, one year |
| Establish the maximum tolerated dose (MTD) of the study drug. | Part 1, one year |
| Characterize the pharmacokinetics of the study drug and a metabolite under either fasted or fed conditions. | Part 1, one year; Part 2, one year |
Secondary
| Measure | Time frame |
|---|---|
| Assess the efficacy of study drug in terms of response, duration of response and hematologic improvement. | Part 1, one year; Part 2, one year |
Countries
United States
Outcome results
None listed