This is a two-arm, open-label study to determine the maximum tolerated dose (MTD) and assess the safety, pharmacokinetics, pharmacodynamics, and preliminary efficacy of BMN 673 in patients with Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS), Chronic Lymphocytic Leukemia (CLL) and Mantle Cell Lymphoma (MCL). Arm 1 will enroll patients with either AML or MDS; Arm 2 will enroll patients with either CLL or MCL.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
33
Oral capsule with multiple dosage forms given once daily
Indiana University Simon Cancer Center
Indianapolis, Indiana, United States
Seattle Cancer Care Alliance
Seattle, Washington, United States
University of Wisconsin
Madison, Wisconsin, United States
University College London
London, United Kingdom
The primary outcome of this study is to determine the MTD of daily oral BMN 673 in patients with AML and MDS (Arm 1) and patients with CLL and MCL (Arm 2).
Time frame: Assessed after each visit until completion (Estimated duration is 12-18 months)
Number of participants with adverse events
Time frame: Assessed after each visit until completion of the study (Estimated duration is 24-30 months)
Determine the pharmacokinetic (PK) profile of BMN 673
Sample collection times vary per visit. PK parameters that will be evaluated include: maximum concentration (Cmax), minimum concentration (Cmin), time to maximum plasma concentration (Tmax), area under the curve from 0 to last quantifiable sampling point postdose (AUC0-inf), area under the curve extrapolated to infinity (AUC0-last), half life (t1/2), systemic clearance (CL/f) and volume of ditribution (VZ/f)
Time frame: Assessed at each visit in cycle 1 - 5 (Estimated duration is 24 months)
Determine the Recommended Phase 2 Dose (RP2D) of oral daily BMN 673
Time frame: Assessed after each visit until completion of the study (Estimated duration is 24-30 months)
Assess preliminary efficacy of BMN 673 by evaluating per response publications
Time frame: Assessed approximately every 4-12 weeks (Estimated duration is 24-30 months)
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King's College Hospital
London, United Kingdom
The Christie NHS Foundation
Manchester, United Kingdom
University of Newcastle Upon Tyne, NHS Foundation Trust
Newcastle upon Tyne, United Kingdom